transfer center (mttc) and the massachusetts association...

March 24, 2008

Welcome to the Fourth Early Stage Life Sciences Technology Conference – a showcase for platform technologies and recently formed companies that are based on research developed at Massachusetts

research institutions, hospitals and universities.

The Conference is one of the most important events co-hosted by the Massachusetts Technology Transfer Center (MTTC) and the Massachusetts Association for Technology Transfer Offices (MATTO). The program is designed to provide an introduction for the investment and corporate community to some of the earliest stage life science start-ups as well as a review of some available platform technologies that are in the process of spinning-out of local institutions.

Among our presenters today are several research teams who have received proof of concept funding from the Massachusetts Technology Transfer Center. This funding enables these researchers to perform pivotal early development work that will provide the initial results that can demonstrate the viability of their technologies to potential investors and licensors. We congratulate these teams on their success in receiving this very competitive funding and wish them well as they continue to commercialize their technologies.

Today’s conference will also feature a panel discussion on “A New Translational Medicine Player - The New Role of Patients and Philanthropists in Pushing Technologies into the Marketplace.” This panel will examine how foundations see their role in supporting the translation of basic research into new products: how and when they decide to invest their time and money; what returns they expect; how they fund their efforts; and what they can contribute to the process in addition to funding. Our panelists include the leaders and founders of innovative philanthropic organizations that are leading this approach and is moderated by Glenn E. Mangurian, Founding Partner, Frontier Works who is also a Patient Advocate.

We hope you will enjoy today’s program and look forward to seeing you among the attendees of this event in 2009. Please stay for this evening’s closing networking reception, which will include 22 poster presentations and take advantage of this opportunity to meet individually with the presenting companies and technology transfer representatives from participating research institutions.

The Massachusetts Life Sciences Cluster continues to be vibrant. By supporting these early stage companies and researchers we will ensure that the cluster continues to grow and diversify within the Commonwealth. Thank you for participating in the conference.

Dr. Abigail A. Barrow, Conference Co-ChairDirector, MTTC

The 4th Annual Early Stage Life Sciences Conference

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March 24, 2008Harvard Medical School Conference Center ∙ Boston, MA

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Table of ContentConference Agenda .................................................................................................................vSpeaker Biographies .................................................................................................................7

Glenn E. Mangurian ........................................................................................................................................ 8James Heywood ............................................................................................................................................... 8Peter D. Kiernan, III .......................................................................................................................................... 9Brock Reeve ........................................................................................................................................................ 9Larry Steranka ..................................................................................................................................................10

Sponsors, Affiliate & Host ..................................................................................................... 11Sponsors ...........................................................................................................................................................13Nonprofit Affiliate ...........................................................................................................................................16Host .....................................................................................................................................................................17

Presenters Profiles ................................................................................................................ 19About the Investor Pitches ..........................................................................................................................21Advanced Kinase Diagnostic ......................................................................................................................22Avaxia Biologics .............................................................................................................................................24Boston University * ........................................................................................................................................26Children’s Hospital Boston * .......................................................................................................................28Fission Pharmaceuticals, LLC ......................................................................................................................30Nemucore Medical Innovations Inc. * .....................................................................................................32NKT Therapeutics Inc. ...................................................................................................................................34Northeastern University and Draper Laboratory * ..............................................................................36P.A. Technologies, LLC ..................................................................................................................................38Progenestem ....................................................................................................................................................40SkelScan, Inc. ....................................................................................................................................................42Wesfolk Corp. ...................................................................................................................................................44

Additional Exhibitors Profiles ............................................................................................ 47Beth Israel Deaconess Medical Center * .................................................................................................48Children’s Hospital Boston * .......................................................................................................................50Children’s Hospital Boston * .......................................................................................................................52Eutropics Pharmaceuticals ...........................................................................................................................54Massachusetts General Hospital *.............................................................................................................56Massachusetts General Hospital * ...........................................................................................................58Tufts University ................................................................................................................................................60University of Massachusetts Medical School ..........................................................................................62University of Massachusetts Medical School* .......................................................................................64VasoTech, Inc. ..................................................................................................................................................66

* Recipient of MTTC Technology Investigation Award


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CONFERENCE AGENDA

1:00 – 1:30

Registration, Dessert Bar, Networking, and Exhibits (Lower Lobby)

1:30 – 2:30 Opening Program (Amphitheater)

Welcome: Abi Barrow,

Director, MTTC

Panel: “A New Translational Medicine Player - The New Role of Patients

and Philanthropists in Pushing Technologies into the Marketplace”.

Moderator: Glenn E. Mangurian,

Founding Partner, Frontier Works and Patient Advocate

Panelists: James Heywood, Founder , ALS TDI Peter D. Kiernan III,

Chairman of the Board of Directors, Christopher and Dana Reeve Foundation Brock Reeve,

Executive Director, Harvard Stem Cell InstituteLarry Steranka,

Managing Director, Cancer Research Technology, Inc.

2:30 – 3:20 Company Pitch Presentations - Session One (Amphitheater)

3:20 – 3:35Coffee Break (Lower Lobby)

3:20 – 3:35 Company Pitch Presentations - Session Two (Amphitheater)

5:15 – 7:00 Networking Reception and Poster Session (Lower Lobby)

March 24, 2008The Conference Center at Harvard Medical School

Biographies �

Speaker BiographiesGlenn E. Mangurian ..................................................................................................................8

Founding Partner, Frontier Works and Patient Advocate

James Heywood ........................................................................................................................8Founder, ALS TDI

Peter D. Kiernan, III ...................................................................................................................9Chairman of the Board of Directors, Christopher and Dana Reeve Foundation

Brock Reeve .................................................................................................................................9Executive Director, Harvard Stem Cell Institute

Larry Steranka .......................................................................................................................... 10Managing Director, Cancer Research Technology, Inc.

The Early-Stage Life Sciences Technology Conference IV

8 Biographies

Panelist Biographies

Glenn E. MangurianFounding Partner, Frontier Works and Patient Advocate Panel Moderator

Glenn Mangurian has over three decades of experience driving innovation and results with his clients. Through personal experience he has discovered that we are capable of overcoming challenges that seem insurmountable. As a skilled partner, Glenn teams with both individuals and organizations to aim high, take action and realize aspirations.

As a former, SVP with CSC Index he was responsible for commercializing the concept of Business Reengi-neering. Glenn has experienced many of growth challenges including geographic expansion, market rein-vention and acquisition. In 1989, the Index Group as acquired by Computer Sciences Corporation (CSC). He was a member of the leadership team that reinvented the firm after acquisition and grew it from $25 million to $200 million in annual revenues. In addition, he orchestrated the acquisition of two professional services firms into CSC Index and had operational responsibility for one of them.

In May 2001 Glenn suffered an injury to his spinal cord resulting in the paralysis of his lower body. Undeterred by his injury, Glenn remains active in his consulting, speaking and writing. He combines his decades of busi-ness experience with his personal experience in overcoming adversity to inspire individuals and organiza-tions to achieve extraordinary results. He has published several articles on change management, business reengineering and resilience. Drawing on his personal experience and others, he is the author of an article published March 2007 in the Harvard Business Review: “Realizing What You’re Made Of.” He is a frequent speaker to executive audiences on the subjects of leadership and resilience.

Glenn has an undergraduate degree in mathematics and an MBA from the University of Massachusetts where he also serves as an executive-in-residence. In partnership with University President Wilson he is host to several leadership programs. An accomplished networker and coach, he is a trusted confidant to many successful business leaders.

James HeywoodFounder, ALS TDI

An MIT engineer with a background in product development and neuroscience Jamie entered the field of translational research when his 29 year old Brother Stephen was diagnosed with ALS.

Jamie is the Chairman and co-founder of PatientsLikeMe, a web 2.0 company where patients can share in depth information on treatments, symptoms, and real world information on managing life changing diseases. PatientsLikeMe is working with the pharmaceutical industry, providers, non-profits and patients to align and illuminate value and improve medical care.

Jamie founded ALS TDI the world’s first non-profit biotechnology company and was its CEO from 1999 to 2007. Jamie implemented an industrialized therapeutic validation process and built the world’s largest ALS drug discovery program. Jamie pioneered an open research model, posting in real time the results of its stud-ies for patients, doctors, and the research community.

Jamie is an active advisor to companies and non-profits working to improve the way in which biomedical research is conducted. His work has been profiled in the New Yorker, 60 Minutes, Pulitzer Prize winner Jonathan Wiener’s book, His Brothers Keeper, and in the Sundance award winning documentary So Much So Fast.

March 24, 2008The Conference Center at Harvard Medical School

Biographies �

Peter D. Kiernan, III Chairman of the Board of Directors, Christopher and Dana Reeve Foundation

Mr. Kiernan is CEO of Kiernan Ventures, a venture capital firm committed to growing companies of conse-quence. He spent nearly 18 years at Goldman Sachs, most of them as a Partner and was instrumental in advising companies and wealthy families around the globe in ways to expand their business. His specialty was forging unique relationships and finding creative and unconventional ways to help growing companies both large and small achieve their promise. After leaving Goldman, Peter founded and led numerous compa-nies including Tech Health, a high growth medical services company where he serves as Chair of the Board. Kiernan was also President and Partner at Cyrus Capital Partners, a hedge fund based in New York and London where he continued to serve as Senior Advisor. He continues as Senior Adviser there. Kiernan was asked by Dana Reeve in the days before her untimely death to lead the organization she and her husband Christopher founded. In the past two years, as Chairman of the Board of Directors of the Christopher and Dana Reeve Foundation, Kiernan has led a dramatic turnaround in the organization’s fight to cure paralysis. He also served as past Chairman of the prestigious Robin Hood Foundation for nearly five years. He serves on the Board of the Darden School at the University of Virginia and served on the Williams College Board and Finance Committee for many years.

Brock ReeveExecutive Director, Harvard Stem Cell Institute

Brock Reeve, a graduate of Yale and the Harvard Business School, is Executive Director of the Harvard Stem Cell Institute. In partnership with the Faculty Directors, he has overall responsibility for the operations and strategy of the institute whose mission is to use stem cells, both as tools and as therapies, to understand and treat the root causes of leading degenerative diseases.

HSCI is comprised of the schools of Harvard University and all its affiliated hospitals and research institu-tions. The institute currently has 60 principal faculty and 70 affiliated faculty. Under the leadership of the Ex-ecutive Committee, HSCI invests in scientific research in three main areas – seed grants, core facilities and large-scale disease programs. Beyond the science, the institute also has programs to address ethics and public policy issues, to provide lab experiences for undergraduates, to educate high school science teachers, science journalists and the public at large.

Brock comes to this role from the commercial sector with extensive experience in both management consult-ing and operations for technology-based companies, with a focus on life sciences.

Brock’s business career started with the Boston Consulting Group. Prior to Harvard, Brock was COO and Managing Director of Life Science Insights, an IDC company, a consulting and market research firm special-izing in information technology in life sciences. Previously, Brock was an Associate Partner in the Pharma-ceutical and Life Sciences practice in IBM’s Business Consulting Services group, working with biotech and pharmaceutical clients on issues ranging from R&D portfolios to operations strategies. Brock also has had hands-on operational responsibility in product management and marketing roles in software start-ups as well as additional experience in IT and the healthcare/life science market as the Healthcare Practice Director at Viant Corp. and a Principal at SRI Consulting, where his clients included some of the leading pharmaceutical, biotechnology and medical device companies.

The Early-Stage Life Sciences Technology Conference IV

10 Biographies

Larry SterankaManaging Director, Cancer Research Technology, Inc.

Larry Steranka is Managing Director of Cancer Research Technology, Inc. Previously, he worked in the field of academic technology transfer, most recently as Executive Director of the Office of Technology Licensing at Brandeis University, before that as Associate Director for Licensing in Harvard University’s Office for Technol-ogy and Trademark Licensing and, before that, as Director of the Office of Technology Transfer at Vanderbilt University. Prior to entering academic technology transfer, Larry held research management positions in the pharmaceutical industry, first with a startup and then with a multinational company. Larry has a Ph.D. in pharmacology, completed one of Harvard Business School’s Executive Education Programs (PMD), and is the author of over 40 peer-reviewed scientific publications.

Sponsors & Host 11


Sponsors, Affiliate & HostSponsors ................................................................................................................................... 13

Deloitte Merck Research Laboratories BostonNutter McClennen & Fish LLP

Nonprofit Affiliate ................................................................................................................... 16Massachusetts Biotechnology Council

Host ............................................................................................................................................. 17The Massachusetts Technology Transfer Center

12 Sponsors & Host


Sponsors & Host 13


We don’t just know your issues. We have the solutions

Our Technology & Life Sciences practice serves leading biotechnology, medical device/product, and technology companies throughout New England and the world. Our practitioners provide solutions to the wide range of issues that life sciences companies face throughout their life cycle, from early-stage research companies to global marketing and manufacturing leaders. At our Tech-Venture Center we leverage our intellectual capital, our knowledge, and our experience in the industry to help our early-stage technology and life sciences clients meet their strategic objectives with an approach tailored to the unique needs of a growing company.

For additional information about Deloitte and its Tech-Venture Center, contact:

Steve ZamierowskiDirector, Tech-Venture CenterDeloitte & Touche LLP Bay Colony Corporate Center 1000 Winter StreetWaltham, MA 02451 USAPhone 617 437 [email protected]

www.deloitte.com/us/lifesciences

Deloitte refers to one or more of Deloitte Touche Tohmatsu, a Swiss Verein, its member firms and their respective subsidiaries and affiliates. As a Swiss Verein(association), neither Deloitte Touche Tohmatsu nor any of its member firms has any liability for each other’s acts or omissions. Each of the member firms is aseparate and independent legal entity operating under the names “Deloitte,” “Deloitte & Touche,” “Deloitte Touche Tohmatsu,” or other related names. Servicesare provided by the member firms or their subsidiaries or affiliates and not by the Deloitte Touche Tohmatsu Verein.

Deloitte & Touche USA LLP is the U.S. member firm of Deloitte Touche Tohmatsu. In the U.S., services are provided by the subsidiaries of Deloitte & Touche USALLP (Deloitte & Touche LLP, Deloitte Consulting LLP, Deloitte Tax LLP, and their subsidiaries), and not by Deloitte & Touche USA LLP.

Member of Deloitte Touche TohmatsuCopyright © 2005 Deloitte Development LLC. All rights reserved

14 Sponsors & Host


You’ve discovered something significant. Now discover us! Please contact:

Barbara YanniVice President and Chief Licensing OfficerMerck & Co., Inc.One Merck DrivePO Box 100Whitehouse Station, NJ 08889-0100 USATelephone: 908–423–4350Fax: 908–735–1201

Copyright © 2008 Merck & Co., Inc., Whitehouse Station, NJ, USA. All rights reserved. LIC 2008-W-84529-AH

www.merck.com/licensing

“We are serious about our commitment to strategic alliances. Our goal is for our partners and us to remain in the forefront of turning scientific breakthroughs into medicines that make a difference. We have established a clear path to partnering, and invite you to explore the combination of your strengths with ours, and the successes we may share.”

– Mervyn Turner, PhD, Senior Vice President, Worldwide Licensing and External Research

LIC 08-W-84529-AH_BioAsia.indd 1 1.2.08 2:08:47 PM

Sponsors & Host 15


Nutter in the Life Sciences

Nutter McClennen & Fish has a long track record of working with clients in the life sciences throughout the enterprise lifecycle to meet their legal and strategic needs. With in-depth legal and scientific credentials as well as experience in executive, in-house counsel, and other industry capacities, our attorneys take responsibility for knowing the client’s needs and objectives, and for partnering to achieve successful outcomes and competitive advantage.

Our roster of life sciences clients is international and diverse, including:

U.S. and multinational medical device, biotechnology and pharmaceutical companies Renowned medical institutions and research universities Major hospitals and other health care providers Emerging growth companies Venture capital firms and entrepreneurs

Nutter’s experience and knowledge cover a wide range of technologies and products, from medical devices such as implants, surgical instruments, and diagnostic and therapeutic products, to research instrumentation and tools, to biopharmaceuticals ranging from functional genomics and immunology to diagnostics and gene therapy. Our services include:

Corporate Matters, Financing and Securities Intellectual Property and IP LitigationGovernment Regulation, including clinical trials, SEC and marketing/pricing compliance, and privacy.Health CareLitigation

Nutter TechnOvation Awards®

To assist promising entrepreneurs in managing the legal issues and risks in growing ideas into successful companies, Nutter founded the Nutter TechnOvation Awards to provide credits for legal services to selected early-stage companies. Award recipients have access to the full platform of services offered by Nutter, which are tailored to each company’s unique needs and stage of development. See nutter.com for more information.

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One Cambridge CenterCambridge, MA 02142TEL: 617-674-5100FAX: 617-674-5101

www.massbio.org

Become a part of the Massachusetts biotechnology community…Join the MBC

Member Benefits include:

• Group Purchasing Consortium

• Investor, Partnering Forums

• Events and Committees

• Public Affairs and Policy

• Economic Development

• Education Initiatives and Training

• Professional Development

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Sponsors & Host 1�


www.MaTTCenter.org

[email protected]

www.MassTechPortal.org

The Massachusetts Technology Transfer Center is funded by the Commonwealth of Massachusetts. Its goal is to support technology transfer activities from public and private research institutions to companies in Massachusetts. To achieve this goal, the Center works with technology transfer offices at Massachu-setts research institutions; faculty, researchers, and students who have commercially promising ideas; and companies across the Commonwealth.

__________________________________________________________________________________

The Center: facilitates and accelerates technology transfer between research institutions and Massa-chusetts companies;promotes collaboration between research institutions and the Commonwealth’s technol-ogy industry; assists in the growth of Massachusetts companies, including startups, by enhancing tech-nological leadership; andsupports regional and statewide economic development priorities.

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The Center supports the commercialization of research technologies through a variety of programs:

The Center provides mentoring to researchers who believe they have a technology that could serve as the basis of a new company. The process includes the development of a business presentation for an expert board of external reviewers.

Commercialization and Entrepreneurial Education seminars and workshops enable researchers to un-derstand the process of commercializing technologies.

Expert technology reviews provide opportunities for Massachusetts research institutes to have external industry experts evaluate technologies and give advice regarding their commercial potential.

Technology Forums allow investors and potential corporate partners to meet with companies formed around technologies developed in Massachusetts research institutes.

Mini-grants support technology development. The Center awards up to $40,000 to inventors from Mas-sachusetts research institutions to fund proof-of-concept research or the development of more refined marketing and commercialization plans.

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Startup Companies 1�

Presenters ProfilesAbout the Investor Pitches .................................................................................................. 21Advanced Kinase Diagnostic .............................................................................................. 22

Janusz M. Sowadski, Founder and CEO

Avaxia Biologics ..................................................................................................................... 24Barbara Fox, CEO

Boston University * ................................................................................................................ 26Theodore Morse, Professor

Children’s Hospital Boston * ............................................................................................... 28Carmen Barnes, Instructor in Surgery

Fission Pharmaceuticals, LLC............................................................................................... 30Charles Hoffman, Professor

Nemucore Medical Innovations Inc. * ............................................................................. 32Tim Coleman, President & CEO

NKT Therapeutics Inc. ........................................................................................................... 34Mark Tepper, President & CEO

Northeastern University and Draper Laboratory * ...................................................... 36Dale Larson, Director

P.A. Technologies, LLC .......................................................................................................... 38Robert M. Weis, Co-founder

Progenestem ............................................................................................................................ 40Edward Kislauskis, Co-founder

SkelScan, Inc. ............................................................................................................................ 42Jerome L Ackerman, Chief Scientific Advisor

Wesfolk Corp. ........................................................................................................................... 44Lewis Geffen, President



20 Startup Companies


Startup Companies 21

About the Investor Pitches

IntroductionThe Massachusetts Technology Transfer Center hosts several conferences a year, each to showcase early stage entrepreneurs in a different technology or industry sector. The heart of each showcase is the ten-minute investor pitches given by selected entrepreneurs followed by a reception in an exhibit hall at which interested attendees can meet the entrepreneurs and network with other participants.

The pool of applicants to present has grown since our inaugural conference in 2005, so we have selected as broad a range as possible, from technologies still in the university laboratory to companies starting to sell product; from the most polished and experienced presenters to those who have never presented to the business community before; from the most cutting edge technology to established technologies that still support an innovative business model.

Business and Technology MaturityThe mandate of the MTTC is to help entrepreneurs based in Massachusetts’ nonprofit research institu-tions to commercialize their technologies. Thus, we always give priority to applicants from our hospitals and universities. However, we are also working toward economic development in the state, so we also welcome entrepreneurs already working in startup companies, giving preference to those who are collabo-rating. Those who have already received significant venture capital are beyond the scope of this section of the conference.

Some presenters are alumni of prior Conferences. They are invited to participate only if they have signifi-cant business updates to incorporate into their investor presentation.

Life Sciences TechnologiesAll aspects of Life Sciences technologies may be represented at this Conference. This year, there are pitches for advanced imaging devices, innovative drug discovery and development technologies, new stem cell utilization devices and state-of-the-art services to the pharmaceutical industry.

MTTC CoachingEach presenter participates in a coaching session prior to the Conference to ensure that no pitch is longer than ten minutes and that each contains critical business information along with high-level data on the technology. Our purpose is to provide investors in the audience with a time-efficient opportunity to learn of the latest technologies being made available for commercialization and to be able to evaluate their busi-ness potential at a preliminary level.



Advanced Kinase DiagnosticPresented by:Janusz M. Sowadski, Founder and CEO

Company Overview Our company aims to improve the clinical effectiveness of kinase inhib-itors in stratified cancer patient populations. We speed up the process and lower the risk of all anti-kinase drug developers in clinical trials through genetic stratification of cancer patients.

We target anti-kinase agents by translating aberrant kinase signals into signature designs for high patient response using our unique AKD scans, i.e. genetic assays across diverse cancer subtypes.As discov-erers of Gleevec®, we translate what we have learned into highly effi-cient drug discovery process. Our expertise in oncology drug discovery leads to a dynamic IP portfolio, which is the core of our AKD scan technology.

The founder and CEO of our company is an entrepreneur with exper-tise in anti-kinase structure-based drug design, who has received the US Supercomputing Award. He de-termined the first crystal structure of protein kinase and is a co-discoverer of Gleevec®. He is a co-founder and executive officer of Kinetix Pharmaceuticals & Pintex Pharmaceuticals. Other team members include the clinical discoverer of Gleevec®, who has in-depth practical knowledge of genetic stratification of patients for specific kinase inhibitors; a national expert in cancer tissue microarray technology and Her2 cancer di-agnostics with in-depth knowledge of biomarker development; and an expert in protein kinases signaling pathways, who has expertise in cancer pathway signature designs. We offer to highly qualified investors an investment of $10 million and we aim to sell the Company within 3 years to one of the major anti-kinase drug developers.

Product/Technology ProfileOur key products are clinical biomarkers that target anti-kinase agents in specific cancer patients. Patients’ responses to anti-cancer drugs differ markedly depending on their specific kinase “phenotypes”, suggesting that genetic characterization of patients’ subtypes is necessary for maximum treatment benefit. We facili-tate targeted therapy by developing clinical biomarkers in early stages of clinical discovery for pre-selected patients and by guiding the therapy selection of approved drugs. Clinical biomarkers enhance the value of anti-kinase molecules by lowering the risk of failing in clinical trials and lower the cost of clinical trials by lowering the number of patients required for clinical studies. In addition, biomarkers speed up the process of FDA approval for new drug development and therapy.

Market and ApplicationAll large pharmaceutical companies as well as many small and medium-sized ones have anti-kinase pro-grams. Over 100,000 compounds in patent data bases are protein kinase inhibitors. These include pre-clinical and lead candidates. In addition, 130 protein kinase inhibitors are in Phase I and II trials and eleven kinase inhibitors are in Phase III trials. Currently, ten approved protein kinase inhibitors are approved with a total sale of over $4 billion in 2007.

We will contract with pharmaceutical and biotech companies to facilitate preclinical and clinical programs for anti-kinase therapy in oncology by providing genetic assays for the identification of highly responsive cancer

Officers and DirectorsJanusz M. Sowadski, Founder and CEO

Contact Information31 Rutland SquareBoston MA.,02118Phone: 617 267 [email protected]



patients. All pharma companies focused on anti-kinase therapeutic development are our potential clients, including those who purchased Kinetix and Pintex.

Commercial Opportunity Targeted customers are small and medium-sized biotech companies that do not have the resources or tech-nical expertise to develop clinical assays for their molecules. Their strength is the structure-based design of molecules. Failure to develop and proceed with clinical assays, however, often means the end of a company. We believe that offering our services to these biotech companies at affordable rates using fee-for-service contracts will enable them to overcome their limitations with respect to clinical assays and ultimately allow them to enter into a lucrative collaborative contract with larger pharmaceutical companies.

Competitive AdvantagesCurrently, pharmaceutical and biotech companies carry out clinical trials without assays for genetic signa-tures, resulting in an overall low treatment response rate in the anti-kinase drug program. Using biomarkers would lead to a substantially higher response rate for specific genetic patient subtypes, similar to results seen for Gleevec® for CML and GIST.

Our company is uniquely qualified to improve the clinical effectiveness of kinase inhibitors through the use of biomarkers. Our team consists of a group of highly qualified experts in the fields of protein kinases and cancer who have proven track records in collaboration with industry. We employ state-of-the-art technology to allow for rapid identification of short signatures in specific cancer sub-types through our unique and flexible AKD genetic assay scan. Matching the in vitro specificity profile of one molecule or combination of molecules with a patient’s signature profile in particular cancer sub-types will provide a crucial advantage over other techniques currently employed by our competitors, resulting in the highest treatment response rates. We will increase the speed of critical oncology drug approval for more effective treatment.



Avaxia Biologics Presented by:Barbara Fox, CEO

Company OverviewAvaxia Biologics is developing an oral antibody product for mucositis, the painful lesions of the mouth and gastrointestinal (GI) tract that form during cancer chemotherapy and radiation therapy. Mucositis is a seri-ous unmet medical need and the pharmaceutical industry is actively seeking products in this area. Avaxia is also developing antibody prod-ucts for inflammatory bowel disease, celiac disease and pandemic in-fluenza. Avaxia’s antibodies, isolated from the early milk of immunized cows, are safe, active within the GI tract, and have a well-accepted and rapid development path. Avaxia’s products and platform are protected by both patents and trade secrets. Avaxia has produced two lots of bovine antibody, filed 4 patent applications and generated compelling pre-clinical data in an animal model of influenza.

Avaxia has an accomplished management team. Barbara S. Fox, PhD, CEO is an experienced entrepreneur and scientist with a strong track record of successfully advancing drugs through early clinical develop-ment. Anne Baia, CPA, MBA, CFO has served as CFO of many early and mid-stage biotech companies. Eileen Bostwick, Ph.D., VP Prod-uct Development has previously developed bovine antibody products through Phase II clinical trials. This core team is supported by a world-class group of business, scientific and clinical advisors.

Avaxia plans to raise $5 mm in equity investment and grants to advance its mucositis product through early clinical efficacy testing. With a pipeline of promising products and a novel antibody platform, the company anticipates being acquired in 3 years for $50 - $200 mm.

Product/Technology ProfileAvaxia is developing polyclonal antibodies for local delivery to the gastrointestinal (GI) tract. Antibodies are a highly effective class of therapeutics, yet have never before been systematically targeted to the GI tract. Avaxia is utilizing Guardian Antibodies™, isolated from the early milk of immunized cows. Guardian Antibod-ies™ are designed by nature for oral delivery to the GI tract, are stable to gastric digestion, and have an exceptional safety profile.

Avaxia’s lead product is an antibody to treat the oral mucositis resulting from cancer radiation therapy and chemotherapy. The antibody blocks an inflammatory pathway that is central to the development of mucositis. Additional products include an oral anti-cytokine antibody for IBD and an oral anti-gluten antibody for celiac disease.

Markets and ApplicationsMucositis is the major dose-limiting side effect of many cancer treatment regimens and both patients and physicians are seeking new products. Approximately 400,000 patients develop mucositis each year and more than 2 million are at risk of developing the disease. The market for an effective product for mucositis is pro-jected to be greater than $1B. The current market for inflammatory bowel disease is $1.6 B and is projected to grow to $4B by 2016. The celiac disease market is estimated to be > $1B. Recent acquisitions of antibody

Officers and Directors:Barbara Fox, CEOAnne Baia, SFODavid Poorvin

Contact Information:Avaxia Biologics

24 Bedford St.Burlington, MA 01803

Phone: 508-259-5929Fax:[email protected]



products and antibody-based companies have ranged from $50mm for pre-clinical product to $400mm for Phase II products, reflecting the value of antibody-based products to the pharmaceutical industry.

Commercial OpportunityAvaxia’s products will drastically improve the lives of cancer and patients and those suffering from gastroin-testinal diseases. Avaxia’s products will be marketed to physician specialists, either oncologists or gastroen-terologists, who can be reached with a limited sales force.

Avaxia’s near term customers are the pharmaceutical companies who need to fill their pipelines. Poten-tial partners / acquirers include Amgen, Abbott, BiogenIdec, Eisai, Genzyme, J&J (Centocor) and Novartis. Avaxia will seek early feedback from potential pharmaceutical partners on the data package they would want to see prior to an acquisition.

Competitive advantage There are no medications available to treat most forms of mucositis. Only one product has been approved for mucositis, and it is only used for a limited class of cancers. Products in development reduce the severity or duration of mucositis by less than 30%. There are several marketed products for inflammatory bowel disease but each of these products has significant safety and / or efficacy concerns. There are no pharmaceutical products available to treat celiac disease. We are unaware of other companies developing oral antibodies for GI disease. Avaxia has 4 pending patent applications protecting both its products and platform as well as an aggressive strategy to develop and in-license additional antibody-based intellectual property.

Future Financial PlansAvaxia is currently seeking $400K from individual investors. The funds will be used to advance the compa-ny’s product development programs and intellectual property, with less than 20% of the investment used for overhead. At the completion of this funding period, Avaxia will have in place a data package needed to support both early partnering discussions and grant applications in multiple indications. Avaxia anticipates raising a total of $5 mm prior to an acquisition. The funding will come from additional angel investment as well as non-dilutive sources, including government and foundation grants and strategic partnerships.



Boston University *Presented by:Theodore Morse, Professor

Product/Technology ProfileThe product would be a detector element that could be retrofitted to existing X-ray technology. The technology may be described as fol-lows. To create a digital image, the X-rays pass through the target and then impinge on a thin film of scintillating material which produces vis-ible photons that are detected by a CCD or TFT photo-detector array. Degradation in image quality and resolution result from the scattering of the X-rays in the target and dispersion of light in the finite thickness of the scintillator. Scattered radiation sensed by the detector array contributes to image noise and decreases signal-to-noise ratio. The dispersion of light photons in the finite thickness of the scintillator al-lows these photons to interact with pixels distributed over the CCD array resulting in loss of spatial resolution. These problems could be solved if it were possible to collimate the X-rays prior to interaction with the scintillating material, curtail detection of off-axis scattered X-rays, and at the same time, confine the visible photons to a single spatial location. We propose to achieve this in the following manner.

Nanoscale scintillating crystals will be embedded in a polymer coherent array, with the guiding cores of the array less than 1 m-6 in diameter. The visible photons generated by the interaction of X-rays and the scintilla-tor will be confined to these cores. In addition, we will place nanoparticles in a cladding around the cores that will stop X-rays but do not scintillate. Such a coherent bundle will intrinsically reject scatter as it will produce visible photons only from those X-rays that are parallel to the guiding cores. This will result in increased sig-nal-to-noise ratio, light capture efficiency, and DQE (Detective Quantum Efficiency). Digital X-ray imageswith a resolution of 1 m-6 would do for medical imaging what the transistor has done for electronics.

Market and ApplicationThere is an enormous market for improved medical imaging, and we would first concentrate on applications for X-ray screening for breast cancer. The key objective of this development project is to improve the spatial resolution and the detective quantum efficiency (DQE) of X-ray detectors. Currently, the highest clinical reso-lution of digital X-ray imaging systems(dose limited) is of the order of 35 m-6. At this spatial resolution, many pathologies are too small to be resolved. Increasing the spatial resolution to 1 m-6, while keeping the X-ray dose acceptable, would initiate a revolution in medical imaging; the clinical applications are innumerable. For example, in mammography, the resolution of present digital machines is of the order of 50 to 100 m-6. At this resolution, many of the lesions are indeterminate necessitating a biopsy. Currently, 80% of the biop-sies reveal a benign condition. With 1 m-6 technology, it would be possible to better define the morphology of micro-calcifications. More accurate characterization of heretofore indeterminate lesions can significantly reduce the number of biopsies.

Officers and Directors:Theodore Morse, Professor

Contact Information:Boston University

8 St. Mary’s St.Boston, MA 02215

Phone: 617-375-9543Fax: [email protected]




Commercial Opportunity Target customers would be companies that develop applied technologies for the screening of breast cancer. Future efforts will focus on detectors compatible with CT technology.

Competitive AdvantageAt the present time, breast cancer X-ray is limited such that cancerous lesions, which are of the order of 5 m-6, can not be detected. A 1 m-6 X-ray technology has the possibility of eliminating one million unnecessary biopsies/year.

Future Financial PlansWe have applied for government funding through the Congressionally Directed Medical Research Program, CIMIT, and an NIH program. The funding would be used for a feasibility experiment.



Children’s Hospital Boston *Presented by: Carmen Barnes, Instructor in Surgery

Product/Technology Profile 2-Methoxyestradiol (2ME2) is a natural estrogen metabolite and a potent angiogenesis inhibitor. Because of its antiangiogenic activity, 2ME2 has emerged as a novel therapy for the treatment of cancer, and most recently has been investigated as a potential treatment for several complications of prematurity, preeclampsia, inflammatory dis-orders, diabetes, and pulmonary hypertension. Despite this interest in 2ME2 and its long history in clinical trials, there is currently no assay to precisely and rapidly determine the level of 2ME2 in bodily fluids.

With our recently funded MTTC award, we will raise a specific antibody against 2ME2 and develop a sensitive immuno-based assay for this key modulator or angiogenesis. Our technology will allow physicians to rapidly determine the 2ME2 level in blood, and thereby pave the way for the development of therapeutic strategies to prevent complications of prematurity and pre-eclampsia.

Market and ApplicationThe number of extremely premature infants born in the U.S. exceeds 50,000 per year, and the number of pregnancies complicated by preeclampsia is over 160,000 per year.We expect that the market size will be at least twice as large given that 2ME2 replacement therapy for either complication will involve multiple tests per patient. In addition, we anticipate that our assay may be used in the future for prenatal testing of all pregnant women; in this case, the market size is greater than 4 million per year in the U.S., with the potential of more than one test throughout gestation. Our assay may also be used as a companion assay for guiding clinical development of other 2ME2 therapeutics, with a variable market size depending on whether it is used exclusively for research or also as a companion diagnostic.

Commercial OpportunityWe will first use our product for research use and to continue our clinical studies investigating the correlation between 2ME2 levels and complications of prematurity. We then foresee licensing our product as compan-ion diagnostic for complications of prematurity. Because the mechanism of 2ME2 is applicable to other disor-ders, several companies are developing 2ME2 as either replacement therapy, in the case of pre-eclampsia, or as a therapeutic. Therefore, our third and fourth products would involve licensing our blood test as a prenatal test for detection of preeclampsia and its companion diagnostic. Finally, our immunoassay has the potential for multiple licenses to those companies developing 2ME2 as a therapeutic.


Officers and Directors:Carmen Barnes, Ph.D.Instructor in Surgery

Contact Information:Children’s Hospital Boston300 Longwood AveBoston MA 02115Phone: 617-919-2403Fax: [email protected]



Competitive Advantage2ME2 levels in clinical samples are currently measured by gas chromatography-tandem mass spectrometry (GC-MS), a method that is costly, slow, and which requires specialized equipment and trained personnel. Instead, we will develop an immunoassay. Immunoassays have become the alternative of choice for measur-ing steroids in biological fluids because they are simple to use, show low cross reactivity to related steroids, and yield results in a few hours. We have filed a provisional patent for the 2ME2 immunoassay, and have in-ternational patents for the diagnosis, prevention, and treatment of prematurity. Furthermore, we have an on-going clinical study at Children’s Hospital Boston to correlate 2ME2 levels to complications of prematurity.



Fission Pharmaceuticals, LLCPresented by: Charles Hoffman, Professor

Company Overview Fission Pharmaceuticals, LLC is a drug discovery company work-ing in the area of PDE4 and PDE4/7 inhibitors, which have tremen-dous potential to treat inflammatory diseases, neurological diseases, and HIV infection. The pharmaceutical industry approaches this field with a structural model that fails to produce compounds that distin-guish among members of a PDE family (such as PDE4A, PDE4B, and PDE4D). Our cell-based high throughput compound screen uses ge-netically-engineered fission yeast strains that express full-length tar-get enzymes and a positive growth assay that requires the identified compound to not only inhibit the target protein, but to display other characteristics of successful drugs, including low toxicity, high stability, and high cell permeability. We have identified a small group of com-pounds that inhibit PDE4A, PDE4B, and/or PDE7A, but not PDE4D, which is considered the emetic and arrhythmia target, whose inhibition has prevented PDE4 inhibitors from receiving FDA approval. These compounds are leads from each of six structural classes that include other PDE4 or PDE7 inhibitors identified in our screens. They act by elevating cAMP levels in our strains (as expected for PDE inhibitors) and the first four to be tested using an in vitro assay display IC50 val-ues in a range similar to FDA-approved drugs. We will develop these as therapeutic compounds by completing our characterization their biochemical properties, rigorously testing for safety, and demonstrating their anti-inflammatory properties, before establishing partnerships with phar-maceutical companies to pursue clinical studies.

Product/Technology Profile PDE4 inhibitors have the potential to treat inflammatory and CNS diseases, yet side effects due to inhibi-tion of the PDE4D subtype have prevented them from receiving FDA approval. Fission Pharmaceuticals is developing compounds that inhibit PDE4A and PDE4B subtypes (as well as the PDE7A feedback response enzyme), but not the emetic/arrhythmia target PDE4D. We are looking to eventually partner with industry to become the premier pipeline in this market. Expansion in to other PDE areas (including PDE5 for erectile dysfunction and hypertension) will be possible in the future, once we are established in the PDE4 space. Therefore, our goal is to enter the market with the PDE4 inhibitors described above, and to expand through the ability to use our screening technology to identify inhibitors of other PDE families, possibly via industry collaborations.

Market and ApplicationCompounds that inhibit PDE4 family proteins are in development by pharmaceutical companies for the following indications: depression, anxiety, Alzheimer’s disease, schizophrenia, psychosis, asthma, chronic obstructive pulmonary disease, rhinitis, psoriasis, memory loss, chronic lymphocytic leukemia, multiple scle-rosis, rheumatoid arthritis, osteoporosis, cystic fibrosis, pain, and ulcerative colitis. Other studies show that such compounds block HIV replication. These are obviously substantial markets with significant unmet needs

Officers and Directors:Charles Hoffman, Professor

Contact Information:Boston College

140 Commonwealth Ave.Higgins 401BChestnut Hill, MA 02467

Phone:617-552-2779Fax: 617-552-2011

[email protected]



Commercial Opportunity We will establish strategic alliances with pharmaceutical companies interested in testing our compounds for specific indications of interest. Approximately 50 companies have PDE4 programs focused on different indications described above.

Competitive AdvantageThe pharmaceutical industry has taken a structural approach to developing PDE4 inhibitors by targeting the active site, which is the most highly conserved region within the PDE4 subtypes. Their approach has failed to produce any FDA-approved drugs in the past 25 years. Our cell-based approach uses a high throughput screen for compounds that stimulate growth of genetically-engineered fission yeast strains that express full-length mammalian PDEs. We have identified a small collection of compounds, falling into six structural groups, which are effective inhibitors of PDE4A, PDE4B, and/or PDE7A, but not PDE4D. By patenting the use of these structural families of subtype-selective PDE4 inhibitors, we seek to establish ourselves as the leaders in the PDE4 space.

Future Financial PlansFission Pharmaceuticals, LLC is pursuing a seed round of funding on the order of $2,000,000 to $3,000,000. Our milestones include determining the PDE inhibitory profile for lead compounds from six distinct structural groups of compounds that display promising PDE4 subtype-selectivity as effective inhibitors of PDE4A and PDE4B, or PDE4A, PDE4B, and PDE7A, but not PDE4D. We will hire a medicinal chemist to further opti-mize lead compounds, while we enhance our contacts with industry and pursue strategic alliances. We will contract out for ADME Tox testing of lead compounds to examine their druggability, followed by more rigorous safety tests to address issues of emesis and arrhythmias that have prevented other PDE4 inhibitors from receiving FDA approval. We will also establish efficacy of the compounds in anti-inflammatory cell culture assays and whole animal models. Finally, we will establish strategic alliances with pharmaceutical compa-nies interested in particular indications, so as to best diversify a portfolio of efforts to bring these compounds to the clinic.



Nemucore Medical Innovations Inc. *Presented by:Tim Coleman, President & CEO

Company Overview Nemucore Medical Innovations, Inc. (“NMI”) is a preclinical stage phar-maceutical company dedicated to developing, manufacturing, and marketing proprietary compounds with improved therapeutic index. NMI’s patent pending process is intended to overcome the long-term macro trend in the pharmaceutical industry to discover compounds with improved pharmaceutical activity but poor solubility. This trend has become more apparent as productivity of pharmaceutical pipelines plummet despite increased money being spent to develop compounds. NMI has a clear vision and ability to develop novel formulations of FDA approved drugs for the market. Our Layer-by-Layer technology repre-sents an innovative way to reduce development time and allow NMI to create proprietary oncology products. NMI expects to create a devel-opment program suitable for filing a 505(b)(2) application to achieve licensure of our compounds. Such a program can reduce product de-velopment times to 3-5 years while still producing products that fulfill unmet medical needs. With our internal efforts focused on developing chemotherapeutics, we intend to create a partnering strategy for other indications that will provide tangible value to our pharmaceutical part-ners and a sustainable business for NMI.

NMI has established an experienced management team. Leading NMI is Dr. Tim Coleman, an experienced business leader with experience with both large and small entrepreneurial efforts. As a consultant for PwC he has advised government, hospital, medical device, and biopharmaceutical clients on a variety of man-agement issues including the creation and implementation of business strategy. Prior to PwC, Tim founded and built a cancer vaccine company where he developed an innovative way to deliver cancer antigens to the immune system via toll-like-receptors. Tim is complemented by an entrepreneurial team: Mike Masterson - Executive Chairman - 22 years experience as a serial life science entrepreneur and venture partner; Dr. Deb Norman JD - Sr. VP. Regulatory - 22 years experience in life science regulatory, clinical development, and patent law ; Dr. Rod Cole - VP pre-clinical Development - 17 years experience working at Pfizer, Arqule, Millennium, and AstraZeneca managing preclinical development programs; and Dr. Susan Zawaski - Director of Operations - Currently at Sloan School of Management, Susan received her Ph.D. in Biomedical Engi-neering from the Illinois Institute of Technology were she received the National Science Foundation Gradu-ate Research Fellowship, the IIT Dean’s Research Scholarship, and the Pritzker Fellowship for Biomedical Engineering.

Product/Technology Profile Our Layer-by-Layer technology (LbL-Rx) uses FDA approved biopolymers to render insoluble compounds soluble without the use of harsh solvents that can be linked to toxicity in the clinic. LbL-Rx is a nano-encap-sulating platform suitable for many different classes of poorly soluble drugs. Our near-term market opportu-nity is to become the provider of LbL-Rx chemotherapeutics with enhanced therapeutic index amenable to most major oncology markets.

* Northeastern University recipient of MTTC Technology Investigation Award

Officers and Directors:Tim Coleman, President & CEOMike Masterson, Executive ChairmanDr. Deb Norman JD, Sr. VP. Dr. Rod Cole, VP pre-clinical Development Dr. Susan Zawaski, Director of Operations

Contact Information:33 Kirkland CircleWellesley, MA 02481Phone: 617-8172305 [email protected] www.nemucore.com



The combination of generic chemotherapeutics with LbL-Rx should produce patent protected molecules with developmental properties making the molecules amenable for 505(b)(2) FDA regulatory process and thus limiting NMI’s development risk, time, IP and expenditures. The first product we expect to develop is LbL-paciltaxol. In addition to our internal efforts, the LbL-Rx platform is highly amenable to partnering with the pharmaceutical industry. Today, 40-60% of compounds discovered at pharmaceutical companies are poorly soluble. We expect to establish value added partnerships with pharmaceutical companies in areas outside of oncology using the LbL-Rx platform to convert their biologically active, insoluble compounds into molecules with improved bioavailability.

Market and ApplicationNMI understands that the multi-billion dollar oncology therapeutic market is highly stratified by tumor type, disease stage, and existing treatment protocols. Our development program will target patient populations with discreet needs for chemotherapeutics where traditional therapies have failed initially and work towards gaining first line status as we gain clinical efficacy data.

Commercial Opportunities Currently, NMI believes it can establish and maintain a sales force to large enough to cover the approxi-mately 2000 oncologists in the US market. We plan to use an account sales team approach to detail the value points (oncologist, pharmacy, and formulary committee) to hospitals and outpatient treatment centers to ensure they are educated on the use and labeling of our products. Market research indicates that sales in the first couple of years after approval could approach $125M. Using head-to-head comparison clinical trial data, we will work with the payer community to develop a price structure that rewards the economic value we expect our products to create.

Competitive Advantage The desired features of pharmaceutical carriers for intravenous administration include:

their small size and biodegradability;good loading capacity for a given drug;high content of a drug in a final preparation;prolonged circulation in the blood; andability to gradually accumulate in required areas (targeting).

NMI has a proprietary system for the development of nanoparticulate drug carriers that display all of these properties for the delivery of poorly soluble pharmaceuticals. Until now, it has been nearly impossible deliver hydrophobic compounds in a consistent, reproducible manner. NMI’s technology changes this paradigm and establishes a new way to transform compounds formally considered “undrugable,” into “drugable” com-pounds.

Future Financial Plans Nemucore Medical Innovations is seeking seed funding of $1.5 million investment to build out the manage-ment team, perform pre-IND studies on our first product, and conduct operations through the fourth quarter of 2008. Upon the successful completion of pre-IND studies, NMI expects to seek an investment to fund scale up operations, clinical trials, and launch partnering efforts.

In today’s financial market environment management feels strongly that it can create long-term shareholder value, however, the most likely exit strategy for NMI will be through acquisition. We expect our competitive advantages in technological innovation, regulatory strategy, and product development will result in capturing significant market share. Our ability to deliver pharmacologically active but poorly soluble drugs should lead to a substantial increase in NMI’s valuation. NMI management is focused on creating shareholder value by

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NKT Therapeutics Inc.

Presented by:Mark Tepper, President & CEO

Product/Technology ProfileNKT Therapeutics, Inc. is a biotechnology company focused on devel-oping therapeutics based on a unique subset of immune cells called natural killer T (“NKT”) cells. NKT cells which represent less than 0.01% of the total lymphocyte population, are a central regulator of the human immune system. By regulating the innate arm of the immune system they help eliminate pathogens and tumors. By regulating the adaptive arm of the immune system they modulate antibody and cell mediated immunity which protects our health over time. Until recently, NKT cells where generally considered bystander cells in the immune system. However, recent evidence has clearly established their impor-tance in human health and disease. The mission of NKT Therapeutics is to use its expertise in the biology of NKT cells and leverage its in-tellectual property to develop a pipeline of first in class medicines to treat Cancer, Infectious Diseases, Autoimmune Diseases, Asthma and Dermatitis.

The NKT Therapeutic’s team consists of our 3 scientific founders, Dr. Mark Exley, Dr. Steven Balk, and Dr. Brian Wilson, all members of Harvard Medical School affiliated institutions. Drs Exley, Balk and Wilson have worked as colleagues in the field of NKT cell biology for many years and have extensive expertise in the ap-plication of immunotherapeutics to human disease. Our management team is made up of Dr. Mark Tepper as CEO and Dr. Alem Truneh as Sr. Vice President of R&D and CSO. Drs. Tepper and Truneh are experienced entrepreneurs with extensive experience in the translation of early stage discoveries to commercial products for treating human diseases Combined they have over 35 years of industry experience in drug discovery & development and a proven track record in bringing human therapeutics to market.

NKT Therapeutics is currently raising $1 million in seed capital to bridge it to an ~$8M series A round. The company strategy is to advance its lead programs to clinical proof of concept over the next 3-4 years and then seek an acquisition or alternate liquidity event.

Product(s)/TechnologyNKT Therapeutics has 3 patent protected programs with a total of 6 different product candidates. The lead program is based on proprietary antibodies which bind with high affinity to a receptor on NKT cells (TCR) which is invariant among all humans. The products in this program can activate or deplete iNKT cells and are being developed to treat cancers, infections diseases (activating antibody), asthma and autoimmune dis-eases (depleting antibody). The second program is based on small molecule NKT antagonists that displace glycolipids bound to the CD1d receptor on antigen presenting cells. This program includes 2 product candi-dates which have been shown to be effective in treating asthma and dermatitis in animal models, antagonize the cognate interaction between the iNKT TCR and CD1d leading to NKT inhibition. The third program is based on monoclonal antibodies against human CD1d. This program includes multiple product candidates which activate or antagonize the CD1d-NKT cell interaction leading to NKT cell inhibition or dendritic cell maturation. Combined these 3 programs offer the opportunity to develop first in class products which have the potential to target a number of unmet large market indications.

Officers and Directors:Mark Tepper, President & CEOAlem Truneh, CSO

Contact Information:233 Needham St, Suite 300Newton, MA 02464Phone: 617-413-3020Fax:[email protected]



Market and ApplicationTherapeutics which target NKT cells offer the opportunity to develop a new class of immune modulator with the potential to treat cancer, infectious disease, autoimmune diseases (e.g, SLE, T1D, MS, IBD), asthma and dermatitis, by a totally new mechanism. Each of our products targets a least one large market indication, with most targeting multiple indications. The advantage of our approach is that by targeting NKT cells we can develop more selective and safer drugs than exist today. The table below shows the patient numbers and market size for each of the diseases we are targeting.

Commercial Opportunity NKT Therapeutics has the opportunity to be the first company to develop therapeutics which specifically targeting NKT cells which are a master regulator of the innate and acquired immune system. Our approach is very different from existing immune based therapeutics. Firstly, we are targeting a very specific cellular component of the immune system- the NKT cell, and not all T cells. Since NKT cells typically represent less than 0.01% of the T cells in the body, yet play a very specific role in regulating the immune response, they offer the advantage of being much more selective. Second, we are targeting immune receptors which are identical in each of us. Thus, our approach is applicable to all patients regardless of differences in their MHC and TCR molecules. Thirdly, our approach targets a specific cellular component of the immune system which is directly involved in mediating certain diseases. For instance, NKT cells have been shown to be necessary in both asthma and atopic dermatitis. Elimination of NKT cells in these models inhibits the development of the disease while repopulation of NKT cells once removed returns the disease. Therefore, targeting NKT cells provides a direct way of treating certain immune diseases without eliminating other desirable components of the immune system.

Competitive AdvantagesNKT Therapeutics Inc. is the first company dedicated solely to the development of therapeutics based on NKT cells. It has a dominant IP position with respect to the composition and use of monoclonal antibodies and small molecule antagonists against NKT cells and the counter receptor CD1d on antigen presenting cells. It has acquired through its founder’s laboratories advanced tools developed over the last decade, which give it a clear competitive advantage to facilitate development of NKT therapeutics. These include transgenic and knockout mice (with human NKT receptors), proprietary antibodies and NKT ligands, cellular and biochemical assays, NKT cell lines and many other tools and reagents to accelerate the advancement of its programs. NKT Therapeutics Inc. has an exceptional team of scientists and business leaders who are e highly committed to seeing the development of therapeutics based on NKT technology. Once clinical proof of concept is achieved, any company wanting to be in this area will need to come to us. This puts us in a very strong position once clinical milestones are achieved.

Future Financial PlansNKT Therapeutics Inc. was founded in January 2008 and is just completing the licensing of its core technolo-gies. It has a commitment from a syndicate of top tier VCs which upon closing will provide $0.5 million in seed capital followed by $8 million in equity based funding for the series A round. These funds will be used to advance our 2 lead programs through IND. Currently there is still room for one additional investor in the series A syndicate. Interested parties should contact Mark A, Tepper, Ph.D. at [email protected] or 617-413-3020.



Northeastern University and Draper Laboratory *

Presented by:Dale Larson, Director

Company OverviewThe “Nanohole Array Calorimetry” project aims to construct a bread-board device to confirm predicted improvements to calorimetry that will extend its use in the pharmaceutical and biochemistry fields. The successful completion of this project will position us to raise additional funds to develop a fully functional nanohole calorimeter product within four years.

Product/Technology ProfileThe photonic chip-scale biocalorimeter removes two critical limitations of current calorimeters; it reduces the amount of compound required by a factor of 1000 and increases the analytical throughput by a factor of 10. The photonic biocalorimeter will reduce pharmaceutical drug development cost and risk and expand calorimetry use into new areas. This award will enable further development of the working tempera-ture sensing technology into a breadboard calorimeter that will be used to confirm the expected performance predictions and provide further development information for a deployable prototype and will enhance commercialization opportunities.

Market and ApplicationInitial market estimates are that in five years the photonic calorimeter will have a 20% market penetration worth 161 million dollars with an-nual sales of consumable products for the photonic calorimeter of 362 million.

Commercial Opportunity Major pharmaceutical companies will use this product in drug development, and potential drug screening. Other fields that could use this device include biochemistry and combustion research.

Competitive AdvantagesThe advantages, decrease compound usage, faster run time and high sensitivity, of the nanohole calorimeter over current calorimeters are summarized in the table below. The increased level of information provided by the calorimeter results also will decrease the number of “false starts” that occur with the current initial testing techniques.


Officers and Directors:Dale Larson, ProfessorGreg Kowalski, Director of Biomedical Engineering

Contact Information:Gregory Kowalski Northeastern UniversityBoston, MA 02115Phone: 617 373-2971Fax 617 [email protected]

Dale LarsonCharles Stark Draper Laboratory555 Technology Square MS32Cambridge, MA 02139Phone: [email protected]



EXISTING TECHNOLOGY* NANOHOLE ARRAY CALORIMETER

Sample Size Required (mg) 0.5 – 5 < 0.005Run Time (minutes) 60 – 90 5 – 10Multiple Sample Capability No YesHigh Sensitivity No Yes

Future Financial Plans

The following outlines our financial development plan.

Proof of Concept6 months and MTTC Proposal Funds: $40KLabor for testing and analysis;Test with known reactions: confirm performance advantage Results used to guide next generation device

Prototype1 year and $0.75MTechnology partners (At PI levels): Biogen Idec, Merck, Pfizer and Schering-Plough

Production2 years and $4MProduct release

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P.A. Technologies, LLCPresented by:Robert M. Weis, Co-founder

Company OverviewP.A. Technologies is focused on developing novel methods for the study of signal transduction pathways. Proper cell signaling is critical for proper regulation of cell growth and division. Cancer and other hu-man diseases are associated with cells that are deregulated. Our main technology, Template-Directed Assembly (TDA), will allow researchers to explore key pathway interactions, in vitro, in a manner that is simple, affordable, and biologically relevant. Our products are compatible with the high-throughput assays used in the process of drug discovery.

P.A. Technologies was formed under the leadership of Dr. Robert M. Weis and Tim J. Shrout; together they bring a unique blend of exper-tise. Dr. Weis was the PI in charge of development of the technology at the University of Massachusetts Amherst, and Tim Shrout has created over 50 new businesses from conception and is currently President and Owner of a diversified conglomerate with over $50MM in annual revenue.

Product/Technology ProfileTDA technology restores activity and regulation to the many proteins that are involved in cellular signaling pathways important to human disease in a biologically relevant manner that currently does not exist using standard test-tube assays. In the cell, a host of proteins residing in or near the cell membrane are respon-sible for information processing and intra and intercellular communications. These communication networks consist of thousands of proteins all interacting in concert to carry out their specific functions. Diseases, including cancer, diabetes, and many others arise when these networks fail to communicate properly. Drug discovery efforts are carried out to produce agents that in effect correct these communication issues, but this process is plagued by many problems. The cell membrane plays critical roles in these communication net-works, 1) as a barrier between the interior and exterior of the cell, and 2) as a ‘protein organizer’ that orders and allows communication to occur within the context of protein teams and arrays that would not be possible in the absence of the membrane. It is precisely the latter role that TDA technology addresses, providing as-says yielding biologically relevant results while allowing the researcher to use reagents that are purified and defined.

Market and ApplicationFor drug discovery the potential is immense. For example, targeting protein-protein interactions, either as the sole target or in combination with ATP binding pocket inhibitors, is now possible with template-assembled protein complexes and represents assays that have not been afforded previously. Thus, TDA provides a new avenue for drug discovery as a simple test-tube assay that restores the essential interactions among signal-ing proteins. Importantly, TDA as a reagent is simple to incorporate into typical assay formats and is consid-erably less expensive, expense and time, than cell based assays while bridging the gap between traditional test-tube assays, which are plagued with the issues outlined above, and cell based assays.

Officers and Directors:Robert M. Weis, PartnerAnthony L. Shrout, PartnerEdward A. Esposito, PartnerTim Shrout, Business Manager

Contact Information:

P.A. Technologies, LLC101 University Dr., Suite A5Amherst, MA 01002Phone: 413-545-1651Fax: [email protected]



Commercial Opportunity Since the introduction of TDA by Shrout, Montefusco and Weis, it has proven to be an effective means to reconstitute the membrane-associated biological activities of purified protein components from a variety of systems. Important results demonstrating the effectiveness of TDA technology were presented at a Millipore research conference in Dundee, UK in June of 2006. Ultimately, these results led to a set of agreements with Millipore. We have a nonexclusive distribution agreement for the TDA reagent, now sold under the Bio-FormTM name, and a research and development agreement that will commence activities in the very near future to expand and exploit the TDA reagent in many aspects of drug discovery, basic research, assay de-velopment, and production of novel reagents. Target customers range from basic researchers in academia, to Big Pharma.

Competitive AdvantagesDuring the drug discovery process purified proteins are used to screen against large libraries of compounds. Sizes of these libraries range from small, ~5000 compounds, to large, ~2 million compounds. While these proteins are easy to use, one major drawback is that they have been removed from their natural environ-ment, the membrane environment. Ultimately, the end result is a set of less-than-optimal protein reagents and buffer conditions that often lead to false positives, missed leads, and misleading information regarding inhibitor specificity and potency during early stage screening. As a result, these shortcomings impede the already lengthy and costly drug discovery process (average drug takes 12 years at a cost of over $1 Billion to bring to market). TDA technology places these membrane associated proteins back into their natural en-vironment easily and effectively thus restoring proper function, activity and the ability to form higher ordered signaling complexes with other proteins. TDA technology represents a new platform for assembling and as-saying the activity of the critical signaling components, which has created a new ability to assess functions that only emerge as a result of biologically relevant assembly. In contrast to the current art, which is focused on screening pair-wise interactions among signaling components, TDA technology represents a qualitatively new testing platform and will permit the many signaling pathways relevant to human disease to be mined for greater understanding and screened for potential therapeutics; novel therapeutics. TDA technology is truly a ‘game-changing’ technology and will introduce a new market for production of many novel reagents and assay services as the process matures.

Future Financial Plans Our products and technology will be amenable to the high-throughput methodologies utilized in the drug dis-covery field as well as to researchers in academia and industry. We have attracted Millipore as a distributor of our technology and plan to continue to make progress with Big Pharma and other businesses concerned with the application of high throughput screening and drug discovery, while progressing TDA technology through in-house research and collaborative efforts with academic labs.

We estimate a funding requirement of ~5 million dollars. This will further TDA and establish the new market this technology represents. We plan to develop and enhance our product line and assemble a larger, more diverse, team of scientists and advisors. We expect that in 4 years our product/technology will be sufficiently developed for significant market penetration in both the academic, and the more lucrative pharmaceutical screening and drug discovery markets.



Progenestem

Presented by:Edward Kislauskis, Co-founder

Company OverviewOur vision is to develop enabling technology for the advancement of cell therapy in regenerative medicine. Patients and physicians are en-couraged by the number of authoritative studies showing that concen-trated autologous (adult) stem cells harvested from either bone marrow aspirate, peripheral blood, and collagenase-digested adipose tissue are effective in repairing tissue and treating chronic diseases. Simi-larly, clinical studies report that concentrated neonatal stem cells from umbilical cord blood and Wharton’s jelly are also effective in repairing tissue and treating chronic disease. It is possible that many conven-tional medical treatment and surgical interventions will be replaced by non-invasive, cell-based therapies. Our patent pending core technol-ogy includes products to address a potential $500B market (US).

Our core technology is an automated device and disposables can safe-ly and efficiently concentrate adult stem cells harvested from bone mar-row (or fat) aspirate, peripheral blood, or neonatal (umbilical) stem cells for administration to the site of injury, at the point of care, without the complicates or expensive or additional processing steps, like expan-sion in vitro.

Our founders have extensive experience in cell biology & bio-physics, engineering & manufacturing, opera-tions & regulatory affairs, finance & strategic development, M&A, and understand this emerging technology market.

Product/Technology ProfileOur patent-pending product/technology covers the flexible bladder supported in the exoskeleton during cen-trifugation; the automated extraction device to retrieve the autologous stem cells; and a method to make the entire system closed. Our proof-of-concept data has achieved >95% TNC recovery in <10% of the original sample volume within 20 minutes of processing. For autologous (adult) stem cell-based therapy, our auto-mated device and disposables are easily adapted to standard O.R. SOPs for processing peripheral blood, bone marrow aspirate, and umbilical cord blood (UCB). For applications of cryopreservation of UCB cells, Progenestem will offer a closed system should significantly reduce personnel, GMP compliance, and facility costs.

Autologous (adult) or neonatal stem cell-based therapies will replace many conventional and invasive proce-dures. Our approach offers a non-invasive alternative, speedy recovery, and reduced pain. For orthopedics, combining concentrated bone-marrow aspirate with a synthetic bone matrix offers an effective alternative to autograft and eliminated a second site surgery to harvest a section of bone from the patient’s hip.

Officers and Directors:Edward Kislauskis, Co-founderNeil F. Duffy, Co-founderJeffrey R. Chabot, Co-founderSteve Cannon, Co-founder

Contact Information:Edward Kislauskis2 Nobscott RoadMedway, MA 02053Phone: [email protected]



Market OpportunityOver 300 clinical trials are underway or have been completed using concentrated autologous (adult) or neo-natal stem cell therapies: Orthopedics: bone grafting, osteonecrosis, ACL repair, spinal cord injury, osteo-arthritis; Neurology: trauma, stroke, Parkinson’s disease, autism; Cardiology: acute myocardial infarction, pediatric congenital heart defects, ischemic heart disease, CHF; Urology: sexual dysfunction, stress urinary incontinence; Endocrinology: liver failure, diabetes; Vascular Disease: CLI, diabetic neuropathy, PAD, CAD. Positive clinical results from these early trials, typically correlating cell dose with efficacy, formed the basis for the U.S. DHHS prediction that the U.S. market for regenerative medicine (including stem cell therapy) will eventually exceed $500 billion, annually.

Competitive AdvantagesThere are 3 alternative technologies used to concentrate stem cells: apheresis (Cobe and Metronics); shelf-based products (Harvest, Biomet, and Genesis), and density gradient technology (ficoll and CPT method). Their individual faults include: low yield (<70%); poor concentration (>20% original volume); are cumber-some; variable performance (hematocrit); or depend upon laboratory processing. The Progenestem Solution captures more cells (>95%) in a smaller volume (<10%) from a wider variety of tissue types, using a common platform, in the O.R., in less time (<20min). Improvement in collection and concentration efficiency greatly reduces bone marrow aspirate volumes required to isolate the same number of cells. Furthermore, the au-tomated Progenestem protocol require minimal expensive and manipulation, in comparison to the competing technology which is dependent on laboratory equipment, sedimentation agents and/or harsh washing steps that reduce cell survival and yield.


Private financing of $3.0M (US) is required to automate manufacturing, production and achieve commercial sales in the orthopedic market. Under CFR 1271, bone marrow-sourced cells are “minimally manipulated human tissue” and therefore would require a 5(10k) submission to CDRH, based on known predicate devices and will not require human clinical data. Based upon this regulatory path, POC work accomplished to date using the proto-type, our management team, and interest shown to date by major orthopedic companies, we anticipate commercial sales within 12 months of financing in the multi-billiondollar autograft (orthopedic) market.



SkelScan, Inc.

Presented by:Jerome L Ackerman, Chief Scientific Advisor

Company OverviewSkelScan’s core technology is a novel method and apparatus for mag-netic resonance imaging (MRI) to diagnose osteoporosis and other bone diseases, and to monitor their treatment. This breakthrough MRI technology marks a significant advance over existing methods. Im-mediate medical applications of SkelScan technology include safe and accurate large-volume screening for osteoporosis, a growing public health problem for aging populations worldwide. Another application distinguishes osteoporosis from certain other bone diseases, which cannot be readily done with existing bone imaging methods. Because SkelScan technology is based on MRI rather than x-rays, patient safety is improved, and patients’ concerns are reduced. The company was founded by three Harvard Medical School scientists who invented the technology.

Product/Technology ProfileSkelScan’s bone scanning technology is based on a specialized adaptation of magnetic resonance imaging (MRI), known as solid-state MRI, which permits the mineral and matrix (protein) content of bones to be mea-sured. By contrast, conventional bone scanning methods can only measure the bone mineral content, and therefore do not capture all the information on bone composition that is potentially available, and which could be used to make a more accurate and detailed diagnosis. The scanning can be carried out on a specially equipped standard MRI scanner or on a compact, comparatively low cost dedicated MRI scanner designed specifically for this purpose.

Market and ApplicationOsteoporosis is a major public health threat for 44 million older Americans, and many more people world-wide. Due to advances in medicine and public health, the population of seniors is increasing briskly, creating a growing need for diagnosing and treating osteoporosis and other conditions of aging. Essentially everyone over age 50 is at risk for metabolic bone disease, and should receive screening. In the US alone, 15-20 mil-lion people already are diagnosed with osteoporosis, 2/3 of them women. 34 million more with osteopenia (low bone mass), are at risk. There are nonmedical applications of the technology, and it is expected to find use in research and production in other industries.

Commercial OpportunityAlthough SkelScan bone scans may be carried out using specially equipped standard MRI scanners, the greatest value is likely to be found in compact MRI scanners. These devices can be far less expensive to purchase and operate. In addition to these advantages for medical centers and clinics which now offer MRI scanning, the lower cost expands the market to smaller clinics and medical practices and to screening procedures which carry much lower insurance reimbursements. Compact MRI scanners, being smaller and quieter, are designed to scan limbs, hands and feet, and are more acceptable to claustrophobic patients.

Officers and Directors:Karin Wetmore, PresidentJerome L Ackerman, Chief Scientific Advisor

Contact Information:Karin Wetmore 165 Pleasant St. #102Cambridge, MA 02139Phone:[email protected]



Competitive AdvantagesToday, screening for osteoporosis is most commonly performed with a dual energy x-ray absorptiometry (DXA) scanner. The technology is widely accepted as a diagnostic tool, but also has important limitations. It uses ionizing radiation, lacks 3-D capability, and is susceptible to certain measurement errors. Most im-portantly, DXA measures only the bone mineral content, and does not measure the protein content, both of which have an important influence on the ability of a bone to resist fracturing when impacted. A significant number of patients who appear to have osteoporosis based on DXA scans may actually have another condi-tion (osteomalacia, inadequate mineral for the amount of protein) which could have been readily recognized and treated if a means to measure the protein content had been available. SkelScan’s technology permits both the mineral and protein content to be measured.

Future Financial Plans SkelScan is seeking investment for a five year program to commercialize its technology. Laboratory studies have been published and are ongoing, but the technology must be productized and implemented on a com-pact MRI scanner platform developed either by the company internally or through partnering and licensing arrangements.



Wesfolk Corp.Advanced Emulsion Technology for the Food and Beverage Industries

Presented by:Lewis Geffen, President

Company OverviewWesfolk was incorporated in 2008 to commercialize a portfolio of scien-tific work conducted by University of Massachusetts, Amherst Professors Eric A. Decker, D. Julian McClements and Jochen Weiss.

The Company’s technology platform includes (i) utilization of emulsions that are engineered to produce oxidatively stable lipid delivery systems, both aqueous and particle, (ii) molecular or colloidal based approaches to coating compositions using food grade components and (iii) charged or dispersed compositions capable of antibiotic and/or antimicrobial end-use applications. Each approach uses GRAS food ingredients, ensuring low cost and regulatory compliance.

Product/Technology ProfileThe technology platform enables several significant commercial applications in the food and beverage in-dustries, including:

Encapsulation of hydrophobic molecules: stabilization of oxidative unstable compounds such as Omega-3 (ω-3) fatty acids, carotenoids, phytosterols, natural pigments

Fat Extender: a way to mimic the texture and taste of a full fat product. Stable emulsions: Formation of very stable emulsions, including freeze-thaw stable emulsions.Stabilization of beverage emulsions: Potential replacement of gum arabic and modified starches. Preservation of beverage emulsions: Potential replacement of sodium benzoate. Food Coating: delivery and stabilization of various components to the surface of foods.

The Company intends to focus initial efforts on an oxidatively stable delivery system to bring Omega-3 (ω-3) fatty acids to functional foods and beverages, where the scientific support for Omega-3 (ω-3) fatty acid’s role in heart health has made Omega-3 one of the hottest functional food ingredients in the market.

According to market researcher Packaged Facts, omega-3 enriched foods make up the strongest sector of the functional foods market in the US; the market for these goods has grown from approximately $100m to more than $2bn in four years. Packaged Facts predicts this category will reach $7bn in sales by 2011. Frost & Sullivan predict that the use of the fatty acid in functional foods will increase, stating that: “With much more potential to be explored in the functional food sector, omega-3 fish oils are poised to demonstrate strong growth.” Our own estimates are that Omega-3 as a functional food ingredient represents between $300-$400 million US/Europe sales in 2006 with an estimated 30% annual growth.

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Officers:Lewis Geffen, President

Contact Information:Wesfolk Corp.c/o Mintz LevinOne Financial CenterBoston, MA 02111Attn: Lewis GeffenPhone: [email protected]



Despite the significant market for Omega-3 food ingredients, fish oil is notoriously difficult to incorporate into formulations since it is highly susceptible to oxidation. The result is a fishy taste and smell which can be off-putting for consumers. Use of existing Omega-3 food ingredients has been hampered due to the adverse effects on taste, flavors, appearance, texture and/or stability. Moreover, industry adoption of Omega-3 food ingredients has been slowed by other limitations of current product offerings, especially products that are too expensive and/or do not deliver sufficient amounts of Omega-3 fatty acids.

The Company’s proprietary oxidatively stable lipid delivery systems enables a competitively advantageous Omega-3 food ingredient, offering superior stability, absence of adverse effects and greater efficacy, all at a competitive cost.

Future Financial Plans The Company believes that private financing can take the Company to revenue generation and profitability.

46 Exhibitors


Exhibitors 4�


Additional Exhibitors ProfilesBeth Israel Deaconess Medical Center * ......................................................................... 48

John Frangioni, Professor

Children’s Hospital Boston * ............................................................................................... 50Farhad Imam, Clinical Fellow

Children’s Hospital Boston * ............................................................................................... 52Pedro J. del Nido, Department Chair

Eutropics Pharmaceuticals ................................................................................................... 54Michael Cardone, President

Massachusetts General Hospital * ..................................................................................... 56Yandong Jiang, Professor

Massachusetts General Hospital * ................................................................................... 58Paul Yu, Instructor in Medicine

Tufts University ........................................................................................................................ 60Peter Wong, Professor

University of Massachusetts Medical School .................................................................. 62Alexander B. Sigalov, Professor

University of Massachusetts Medical School* ............................................................... 64Jie Song, Professor

VasoTech, Inc. .......................................................................................................................... 66Tim Wu, President and CEO


48 Exhibitors


Beth Israel Deaconess Medical Center *Presented by:John Frangioni, Professor

Product/Technology ProfileOur goal is to engineer, construct, and test a prototype hand-held, por-table, and lightweight version of a previously validated intraoperative near-infrared (NIR) fluorescence imaging system for image-guided sur-gery. The key to this system’s success is a unique, LED-based light source module (LSM) that eliminates the need for external cooling and secondary optics. Each LSM measures only 30 mm in diameter and 34 mm in length, and six such modules arranged circumferentially around a main lens illuminates a 10-cm surgical field. Since each LSM weighs only 65 g, the entire imaging system will have the footprint of an early camcorder. Using a patent-pending optics design, color video and NIR fluorescent light will be collected simultaneously, without any moving parts. For the surgeon viewing the image, the color video will show the “anatomy”; the NIR light will highlight the “function”; and a merged im-age of the two will be created simultaneously.

Markets and ApplicationsThe expected low cost of this portable imaging system will render it an indispensable tool in the twenty-first century operating room and beyond. With the prototype in hand, extensive testing in large animals, followed by testing in humans, will pave the wave for the preparation of a 510(k) FDA application based on the predecessor system. Using the many business contacts of the Beth Israel Deaconess Medical Center’s Technology Ventures Office, licensing and commercialization will ensue. Especially exciting for commercialization is the use of the imaging system in non-medical applications includ-ing computer-aided inspection during manufacturing and security watermark detection on mission-critical items.

We believe that this project has outstanding commercialization potential, while solving major problems in hu-man surgery. We foresee a market for our technology in three highly competitive areas of the Massachusetts’ economy including medical applications, manufacturing, and security.

Commercial OpportunityThere are also a multitude of non-medical applications where low-cost and small size will be particularly important. For example, in manufacturing inspection applications, a near-infrared fluorescent dye could be used to test for air or liquid leaks after robotic welding with high sensitivity, and could be seamlessly linked to computer-aided decision-making. In security applications, a small spot or barcode of invisible near-infrared fluorescent dye could be used to “tag” mission-critical equipment and supplies with invisible NIR light used to evaluate such equipment before leaving a secure area.


Officers and Directors:John Frangioni, Professor


Beth Israel Deaconess Medical Center330 Brookline Avenue, SL-B05Boston, MA 02215Phone: 617-667-0692Fax: [email protected]

Exhibitors 4�


U.S. Target Markets:

12,569 - Hospitals and day surgery centers

27,123 - Veterinary hospitals and clinics

8,000 - Military combat support hospitals

~ 50,000 Potential customers

Assuming one device per customer at a $30,000 price, the potential market size is more than a billion dollars. In production, we expect the unit cost to be approximately $5,000 to $8,000. In addition, the consumables market (i.e., NIR fluorescent contrast agents) is expected to be even larger, ranging from $4.0 billion to $13.3 billion annually.

Competitive advantage To our knowledge, there is no comparable, handheld system under development for commercial application. That’s what makes this opportunity so exciting. We have already developed the prototype model and are currently refining ergonomics and functionality.

Currently there are three non-portable, full-size commercial intraoperative NIR fluorescence imaging sys-tems at various stages of FDA approval. Unfortunately, all three of the existing imaging systems are large, expensive (over $100,000 in parts alone), and non-portable. For most community hospitals and outpatient surgery clinics, cost of these systems will be prohibitive. Even in building only a single system and using retail cameras, our handheld unit will cost less than $20K, and with OEM parts and elimination of non-recurring engineering charges, will likely cost under $10K. This makes commercialization to a large market segment, at a competitive cost, possible. Based on the validated performance of its larger predecessor, we believe that a low-cost portable imaging system will become an indispensable tool in the twenty-first century operating room. Moreover, low-cost and portability make available markets with unserved need such as combat sup-port hospitals and veterinary surgery.

Future Financial PlansThe technology has been licensed non-exclusively to GE Healthcare, and additional non-exclusive licenses are available.

50 Exhibitors


Children’s Hospital Boston *Presented by:Farhad Imam, Clinical Fellow

Company OverviewThe Peripherally Inserted Central Catheters (PICC) is an essential tool in providing lifesaving medical care in the Intensive Care Unit or to chronically ill patients. PICC insertion, however, is a blind procedure performed at the bedside and exposes the patient to risk, as catheters can be misdirected during placement. Several current approaches de-veloped to address this significant problem exist, but are expensive, cumbersome for users, and disruptive to physician workflow. We will introduce a series of simple, cost-effective, real-time visualization prod-ucts. The first one is a PICC catheter pre-loaded with a disposable sterile fiber optic stylet, laser light source, and laser-to-fiber coupler. This setup provides the practitioner with the ability to view the location of the catheter in real time during placement with the naked eye. We currently have several prototypes, and proof-of concepts experiments clearly demonstrate success of the technique.

The technology was developed by Farhad Imam, MD PhD, a Clinical Fellow in Newborn Intensive Care (NICU) at Children’s Hospital Bos-ton.

The Intellectual Property Office of Children’s Hospital of Boston is ac-tively seeking national and foreign patent protection for this technol-ogy.

Product/Technology Profile

PICCs are inserted into small veins at distant peripheral insertion origins, often near the elbow or ankle, and are then threaded blindly along their entire length with a desired endpoint near the heart. Blind PICC line placement harbors a substantial risk of misrouting and can cause serious damage by direct contact with adjacent organs or by delivery of strong medications to improper locations. PICC placement has tradition-ally been confirmed by post-procedural X-ray. Real-time visualization can significantly improve safety and accuracy of catheter placement by identifying misrouted catheters instantaneously.

The transilluminating catheter is a simple, innovative method for placement of long, or “central”, catheters in acutely ill patients using visible light. Laser light is conducted from the outside of the patient via fiber optic wire through the lumen of the catheter and inside the body. Light emanates from several points along the catheter course and shines through the skin to the eye of the practitioner, who can plainly see the course of the catheter inside the body in real time during catheter placement without the burden of any additional detection equipment. To date, our technology has been demonstrated successfully in a rabbit model.


Officers and Directors:

Farhad Imam, Clinical Fellow


Children’s Hospital Boston300 Longwood Ave, HUN 430Boston, MA 02115Phone: 617-919-2341 Fax: [email protected]://www.childrenshospital.org/chnews/08-03-07/latest_re-search.html

Exhibitors 51


Markets and ApplicationsThis optically guided technique will be especially effective in children, neonates, and adult patients with thin skin or little subcutaneous fat (i.e., cancer, chronic illness, geriatric). Other applications would include um-bilical arterial and venous catheters in neonates and chest tubes in patients of all ages and could be imple-mented in an outpatient, rural, wilderness, or battlefield setting where X-ray equipment is difficult to obtain.

Commercial OpportunityUp to 85% of blindly-placed PICCs require subsequent manipulation, even if placed by a specialized nursing team.

Payer, payee, and patient should all benefit from a safer and more accurate placement method which im-proves workflow for physicians and nurses. We have spoken with experts in the neonatology field who have expressed interest in using our device and confirmed that a technology providing added safety would gain wide adoption in the medical community

From a financial perspective, the proposed device should decrease the burden on interventional radiology suites and operating rooms to perform central catheter placement as it should improve the success of bed-side placement techniques by non-surgical physicians and specialized nursing teams at significantly reduced cost.

Competitive advantage Catheter placement is currently monitored with post-procedural X-ray which provides only a flat, static image of catheter location. Current attempts to provide real-time visualization at the bedside use ultrasound and/or fluoroscopy. These are resource-intensive due to both capital equipment and personnel training, and have not become widely adopted.

Our technology will:

Increase patient safety: by reducing catheter misrouting, patient exposure to radiation, and increase the ra-pidity at which life-saving therapies can be administered.

Maintain physician workflow: by reducing time to complete and confirm line placement without requiring ad-ditional personnel.

The average cost of an X-ray is $250. It is anticipated that the transilluminating catheter will sell at a premium to the ~$80 catheter typically used in the NICU as it would virtually eliminate the need for multiple X-rays.

Future Financial PlansFormal prototypes have been constructed by Optimum Technologies, Inc., funded through an MTTC Tech-nology Investigation Award. Final tissue and live animal experiments are currently in progress and are scheduled for completion by May 2008. Additional funding will allow us to file an IDE and begin human clini-cal trials within the next year.

52 Exhibitors


Children’s Hospital Boston *Presented by:Pedro J. del Nido, Department Chair

Company OverviewWe provide a less invasive alternative to conventional open heart sur-gery with cardiopulmonary bypass. Traditionally, cardiac surgery has been extremely invasive and traumatic for patients. To date, the applica-tion of minimally invasive techniques in the cardiac surgery market has been limited. Beating Heart Cardiac Surgery Instruments (Instruments) is a portfolio of instruments that form the basis of a platform technology for less invasive repairs of structural intracardiac diseases. The value proposition behind Instruments is improved outcomes, reduced patient trauma, and expanded available patient population.

Our development team is comprised of world leading experts in mini-mally invasive cardiac surgery and engineering. We have developed and tested in animals prototypes of our Instruments. We are currently preparing for a pilot patient study for our first market application, car-diac septal defect closure, and have applied for U.S. and foreign patent protection of the Instruments. We plan to build a company around the Instruments that will penetrate across the cardiac surgery and interventional cardiology markets. Our solution will enable surgeons and interven-tional cardiologists to perform more complex repairs inside a beating heart than are possible with the current minimally invasive techniques.

Product/Technology ProfileThe product line currently is 1) a cardioscope, 2) a patch deployment device, 3) a tissue anchor and delivery device, and 4) a mitral valve clip and delivery device. The specialized cardioscope performs two functions. It permits the safe introduction of the other instruments through the chest wall into the cardiac chambers with-out the risk of introducing air into the heart and significant blood loss. It also has built-in optical imaging. The other Instruments are deployed into the beating heart through the cardioscope. The patch deployment device permits the introduction of a patch for closure of atrial and ventricular septal defects. The patch is secured in place by tissue anchors, which are deployed by a customized delivery device. The mitral valve clip, also deployed by a customized delivery device, is used to repair mitral valve prolapse.

Markets and ApplicationsThe market currently includes atrial and ventricular septal defect closure and mitral valve repair, but it could be expanded to include other cardiac indications such as patent foramen ovale (PFO). About 35,000 babies (1 out of 125) are born with a heart defect every year in the U.S. Atrial septal defects account for about 10% of all congenital heart defects. While some defects will close on their own, they often require surgical intervention. About 5.9 million people in the U.S., 2% of the population, suffer from mitral valve prolapse and 5.3 million people have repairable valves. Of these, about 500,000 people meet the standard for a surgical intervention, but only about 50,000 surgical procedures are done every year.


Officers and Directors:Pedro J. del Nido, Dept. Chair


Children’s Hospital Boston300 Longwood Ave.Boston, MA, 02115Phone: 617-355-8290Fax: [email protected]

Exhibitors 53


Commercial OpportunityOver the past few years, there has been a significant shift to less invasive surgical procedures. In some fields, laparoscopy and endoscopy are becoming the standard of care. In the field of cardiac surgery, the use of less invasive surgical approaches has been limited. In many cases, the clinical incentives of improved out-comes and reduced trauma and hospital stays have encouraged surgeons to learn and adopt less invasive techniques. The adoption of less invasive surgical techniques has also been encouraged by patients.

Competitive advantage The primary advantage over catheter-based cardiac procedures will be improved patient outcomes. We will have the ability to repair a structural defect rather than simply deploying a device. Today, minimally invasive devices are designed to deploy a device that is inserted through an artery or vein and navigated to the appro-priate location in the heart. These devices are limited in how they can be manipulated and there is a tendency for the implants to migrate or embolize. The size of the devices for cardiac septal defects is also limited due to the small size of the peripheral vessels in children. By comparison, the Instruments are inserted through a small incision in the chest wall. As a result, the line of sight visibility and short distance enable the surgeon to repair the defect rather than simply deploying a device.

The primary advantage over conventional open heart surgery, currently the only option for complex cases, will be reduced patient trauma and risk. The Instruments can be used on a beating heart and require a much smaller incision in the chest. This reduces pain, recovery time, and risk of infection.

In addition, the Instruments can expand the eligible patient population. Critically ill patients are refused surgery every year. These patients are often too ill to tolerate the invasiveness of a conventional surgery. While minimally invasive surgical procedures do no eliminate all risks, they can reduce them such that more patients are able to withstand the trauma and benefit from the treatment.

Future Financial PlansFunding will be used to complete pre-clinical testing for Procedure I, beating-heart cardiac septal defect clo-sure (anticipated completion date is Q4 2008) and to prepare for a pilot patient study (anticipated to begin Q4 2009). The required funding to complete pre-clinical testing for Procedure I is projected to be $150,000.

54 Exhibitors


Eutropics PharmaceuticalsPresented by:Michael Cardone, President

Company OverviewEutropics Pharmaceuticals was founded by scientists and business leaders from the biotech and pharmaceutical industries and thought leaders from academic research institutions. With seasoned leader-ship Eutropics is building a strong franchise in oncology and assem-bling a team that is well positioned to develop highly targeted cancer therapeutics. The company’s objective is to discover and develop new cancer drugs that are targeted to non-responsive tumor types in can-cer patients. The Company’s founders have track records in develop-ing early stage biopharmaceutical platforms and have success at the senior management level in discovering and developing exploratory and approved cancer therapeutics at OSI pharmaceuticals, Merrimack Pharmaceuticals and Tanox. Members of Scientific Advisory Board (SAB) have extensive experience in clinical and experimental oncol-ogy and molecular pharmacology and have faculty positions at Dana Farber Cancer Institute, MD Anderson Cancer Center, Harvard Medical School, and the Fred Hutchinson Cancer Research Center.

Our solution relies on proprietary compounds that are highly selective for specific proteins that, when targeted, cause cancer cells to die combined with novel, proprietary diagnos-tic assays that enable us to determine which patients and which cells will be susceptible to our therapeutic agents.

Product/Technology ProfileThe company’s approach is based on the understanding that tumor cells endure ever-present death signaling due to their violation of many rules of normal cellular behavior. Consequently, in order to survive, many can-cer cells become highly dependent on selected blocks to apoptosis or programmed cell death. This survival adaptation is also seen as a cellular Achilles heel, which makes them highly susceptible to therapies that are targeted to the proteins responsible for the block. This phenomenon is known as “oncogene addiction”, an accepted paradigm that provides a backdrop for our technology. Eutropics has developed proprietary assays that can determine if a cancer cell is in such a highly dependent state and identify the specific gene respon-sible for the dependence. The assay therefore, facilitates the development of a drug that can overcome the tumor cell’s inherent resistance to death by targeting this gene. In addition, the assay will also be used to identify patients whose tumors are most likely to be responsive to that therapeutic. The development and progression of Eutropics’ lead compounds into clinical trials will be significantly enhanced by this technology resulting in drugs that will be more specific and effective in treating the patient’s cancer.

Markets and ApplicationsTarget Markets for Therapeutic-Blood Borne Cancers

Non-Hodgkin Lymphoma

Approximately 360,000 cases in US; 550,000 worldwide30-40% of cases are of the largely incurable ‘low-grade’ [indolent] form of the diseaseRituxan is ineffective in these cases

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Officers and Directors:Michael Cardone, PresidentKen RiceAnthony Letai


60 Grampian WayDorchester, MA 02125Phone: 617-314-6431Fax: [email protected]

Exhibitors 55


MyelomaThere are 58,336 people living today with myeloma.In 2007, 16,570 people will be diagnosed with myeloma.

Survival from myeloma five years after diagnosis was only 33 percent in 1996-2002 making it the most dif-ficult blood cancer to treat successfully.

Acute myelogenous leukemia (AML)Approximately 26,000 cases in U.S. with12,000 new cases diagnoses per annum.5-year survival rates: 20.4 percent overall; 53.1 percent for children under 15

Chronic lymphocytic leukemia (CLL)95% of diagnoses for CLL are in individuals 50 years of age and older - “watch and wait” treat-ment prevalentCLL patients have 5-year survival rate of 74%

Approximately 90,000 cases in U.S. with10,000 new cases diagnoses per annum.

Competitive advantage

Highly targeted therapeuticUnique target for hematological cancersKey competitors compounds do not bind this targetProprietary methods for developing compoundsTheranostic approach allows selection of appropriate patients


2008 2009 2010 2011

Seed Round

Series A

Second round

Partner assay

Partner lead

Milestones: Phase 1 safety study,Diagnostic Assay ready

Milestones:Phase llb study

Milestones: Develop Single agentdiagnostic with partner ($ )

.

$15 Million

$1.5 M

$7 Million

$ TBD Millions

$ TBD from partner

Milestones: Phase lll study

Lead optimization, Diagnostic assay development

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56 Exhibitors


Massachusetts General Hospital *Presented by:Yandong Jiang, Professor

Product/Technology ProfileThe device mainly composes of a mask with two one-way valves, which allows patients to inhale through nose only and exhale through mouth only in order to reduce the dead space ventilation and improve the ef-ficiency of breathing.

This device will meet the enormous medical need in treatment of re-spiratory insufficiency, particularly in avoiding invasive treatments such as tracheal intubation and mechanical ventilation.

Markets and ApplicationsThis medical device will be used in patients with respiratory insuffi-ciency. The most common causes of chronic respiratory insufficiency include chronic obstructive pulmonary disease (COPD) and asthma. A recent international investigation showed that the prevalence of spiro-metrically confirmed stage II or higher COPD was 10.1%; while the National Heart, Lung, and Blood Institute estimates that 12 million American adults currently have a diagnosis of COPD, with an additional 12 million unaware that they have the disorder. In addition, approximately 300 million people worldwide currently have asthma, and the prevalence of asthma has increased globally by about 50% every decade

The potential commercial use of this medical device include all but not limited to the following:

1. used independently to improve efficiency of breathing in patients with respiratory failure from various causes, thus reduce the need of more invasive treatment;

2. used in combination with other technology such as non-invasive positive pressure ventilation to im-prove efficiency of treatment;

3. used as a daily tool in patients with chronic lung disease to relieve breathless, and improve exercise ability.

Commercial OpportunityThe potential customers include any patients with breathing disorders, such as patients with COPD, asthma as mentioned above. This device is especially attractive to the potential customers because it is effective, noninvasive and thus avoids many complications related to current other treatments.

Competitive advantage The current treatments of respiratory failure are temporary tracheal intubation and mechanical ventilation or non-invasive positive pressure ventilation through a facemask. These approaches have many drawbacks, in-cluding reduction in cardiac output, worsening ventilation/perfusion match, ventilator associated pneumonia and reduces the efficiency of breathing.


Officers and Directors:Yandong Jiang, Professor


55 Fruit Street, Jackson 422Boston, MA 02114Phone: 617-724-2366Fax: [email protected]

Exhibitors 5�


Our medical device has the following advantages:

has a high efficiency, as proved by computer model.may be used independently or in combination with other current treatments.cost of manufacturing will be significantly less than devices currently used to treat similar condi-tions.non-invasive and only covers the patient’s nose and mouth.increased level of comfort is expected when compared with similar treatments. resulting in high-er compliance rates and patients falling asleep easier. do not need specialized technology or special medical staff to operate, decrease medical ex-pense.has a short investment marketing cycle because of its simple design and easiness to con-struct.minimal side effects are expected.

Future Financial PlansWe have received the Technology Investigation Award from the MTTC to construct the prototype device and to obtain initial clinical data, which will be used to find an industrial partner to commercialize the device. Potential additional funding from industrial partners will be used to fine-tune the device and perform large population study to fully commercialize the device.

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58 Exhibitors


Massachusetts General Hospital * Presented by:Paul Yu, Instructor in Medicine

Company Overview Our group of investigators at Massachusetts General Hospital, com-prised of cardiovascular researchers, physician-scientists, and chemi-cal biologists, have developed small molecules which preferentially modulate the activity of bone morphogenetic protein (BMP) type I re-ceptor kinases, with minimal activity against TGF-β or Activin receptor kinases. These BMP type I receptor kinase antagonists are effective in vitro in blocking BMP-induced SMAD-activation, BMP-induced gene transcription, and have demonstrated efficacy in modulating BMP-me-diated physiology in small animal models. We are actively testing the efficacy of these BMP signaling antagonists in a number of disease models.

Product/Technology ProfileWe have developed the first known small molecule (pharmacologic) inhibitors of the bone morphogenetic protein (BMP) signaling pathway, a signaling pathway that is critical in regulating the growth, differentia-tion, and regeneration of diverse cell lineages. The BMP inhibitors function by inhibiting the kinase activity of BMP type I receptors, including ALK2, ALK3, and ALK6, with high selectivity and high potency. Despite the ubiquity of this signaling pathway in developmental physiology, in preliminary animal studies these mol-ecules appear to be well-tolerated in short-term and chronic dosing. These molecules may have important therapeutic potential in diseases thought to be due to excessive BMP signaling activity, including, but not limited to: Anemia or chronic disease; Calcific atheromatous vascular disease; Malignancies whose clinical behavior appear to be modified by BMP signaling including breast, prostate and renal cell carcinomas; and, Bone metabolic, autoimmune, and inflammatory diseases involving inappropriate or heterotopic ossification. Given the importance of BMP signals in the maintenance, replenishment and differentiation precursor and stem cell populations, BMP inhibitors are likely to be important components of cell-based therapies in which specific lineages or engraftment sites are desirable ex vivo or in vivo.

The potential disease applications are based on cellular and animal based models of disease, clinicopatho-logic studies of human tissues, and clinical/molecular epidemiology. .There is substantial and growing evi-dence that hepatic BMP signaling regulates iron bioavailability, and that BMP signaling modulation can be useful for modifying such signaling to alter iron homeostasis. There is epidemiologic and molecular evidence for the importance of BMP overexpression in the pathogenesis of a number of human tumor types. There is indirect evidence for the role of BMP signaling in both coronary and peripheral artheromatous and calcific vascular disease, both in animal models of atheromatous disease and in vascular lesions from humans. We have begun exploring the possibility that BMP signaling antagonists can be used to limit pathologic bone growth.


Officers and Directors:

Paul Yu, Instructor in Medicine


Thier 50550 Blossom StreetBoston, MA 02114Phone: 617-643-3493Fax: [email protected]

Exhibitors 5�


Markets and Applications

Anemia of inflammation/chronic disease: up to 5 million individuals (U.S.)

Vascular and/or atheromatous vascular disease: up to 500,000 individuals

Malignancies involving BMP signaling: 200,000 - 500,000 individuals

Heterotopic ossification diseases: up to 5,000 individuals

Commercial OpportunityAs would be typical for pharmaceutical development, pending proof-of-principle for each of the potential clinical disorders, potential markets and marketing approaches would be developed during the drug develop-ment and approval process based on each clinical application.

Competitive advantage No current therapies target the involvement of the BMP signaling pathway directly as a means of ameliorat-ing a disease phenotype. While the use of biologics or recombinant proteins have been proposed, our small molecule based strategies are advantageous in terms of cost, have greater potency than recombinant pro-teins, and are advantageous in terms of dosing with potential oral bioavailability.

Future Financial PlansWe are currently pursuing proof-of-concept studies in animal and cell-based models of various human dis-eases in order to demonstrate the viability of these small moecules as potential human therapeutics. We would welcome partners interested in enabling proof-of-concept studies in these disease models.

Our next milestones are:

1. Rigorous demonstration of efficacy and molecular mechanism of benefit in several animal models of human disease;

2. Rigorous demonstration of molecular mechanism of effect in cell-based or biochemical assays of disease physiology;

3. Rigorous demonstration of favorable side effect or toxicity profiles in animals of several potential can-didates in our small molecule family.

These milestones will be tailored for each clinical application towards producing sufficient data towards the long-term goal of first in man studies.

60 Exhibitors


Tufts UniversityPresented by:Peter Wong, Professor

Product/Technology ProfileOur product is a biocompatible scaffold that enables regeneration of cementum and repair of a tooth below the gumline. The scaffold is made using a composite of synthetic and natural materials that are arranged systematically to allow for good fluid flow and tissue growth from a patient’s own dental-related cells. The scaffolds are placed into the patient and the tooth allowed to be repaired via its own biological processes.

Markets and ApplicationsOne market report (http://www.bioportfolio.com/reports/DMD_TISSUE.htm) estimates that the tissue engineering market for regeneration of bone, cartilage, and other connective structural treatments is approxi-mately $15 billion. The analyst sees that success for small companies is inherently complex due to the early stages of this field along with reg-ulatory issues. Thus, partnerships with large companies over longer terms appears to be an emerging business model that looks at therapy ten years down the line. Another report by MedMarkets (http://www.mediligence.com/) published in 2005 reports that tissue engineering products will take at least ten years to go to market due to regulatory issues and that ideal scaffolds will probably be a combination of synthetic and natural materials. Although both reports indicate a large future clinical market for the future, there is an opportunity to introduce products earlier into a significant market for dental repair. We are interested and well-positioned for the dental market which is (1) very large in the US and rest-of-the-world, (2) has practi-tioners who are more receptive to earlier use of new products, and (3) customers willing to pay premium for oral health care. The knowledge and brand gained early in the dental market may lead to adoption of the product in future ones down the line.

Commercial OpportunityThe market plan is to engage oral surgeons first since they are more likely early-adopters and eventually to general dentists as the technology matures and becomes easier to use. The approach will target the dental suppliers who currently have bone-graft products but want to maintain a competitive edge with new products. They have customers (dentists) who are familiar with bone-graft and a patient base who would like to have better products (our scaffolds).

Competitive advantage We are focused on dental repair at the cause of many dental problems, the cementum layer (at the tooth root, below the gumline), which can degrade over time and does not regenerate well on its own. The scaffolds will provide large surface area, access to nutrients, and physical support as the cementum is repaired. The introduction into the dental repair market has the potential for drastically improving oral health care.

Officers and Directors:Peter Wong, Professor


Tufts UniversityMechanical EngineeringDepartmentMedford, MA 02155Phone: 617 627 5162Fax: 617 627 [email protected]/~pwong

Exhibitors 61


Future Financial PlansWith $1.2 million funding in the first two years, the project will have created a systematic range of tissue scaffold patterns to study, conducted cellular growth and testing, redesigned and retested the scaffolds, and then begin approval for clinical testing. Future efforts (with ~$4 million) will be on refining market strategy and product position, developing manufacturing production capabilities, addressing FDA testing, clinical testing, follow-up assessment, partner with distributor, and marketing to dental community.

62 Exhibitors


University of Massachusetts Medical School

Presented by:Alexander B. Sigalov, Professor

Product/Technology ProfilePlatelet disorders play a crucial role in the pathogenesis of thrombotic diseases such as cardiovascular diseases. No other single cell type is responsible for as much morbidity and mortality as the platelet. How-ever, despite intensive research efforts and our current progress in this field, the treatment and prevention of thrombotic and platelet-related diseases still represent a very challenging navigating between Scylla of thrombus formation and Charybdis of uncontrolled hemorrhage, the most-feared complication of current antithrombotic therapies.

The invented technology relates to platelet collagen receptor glycopro-tein VI (GPVI)-targeting inhibitory peptides and compounds that can be used in the treatment/prevention of diseases or other medical condi-tions involving thrombus formation. Such diseases include atheroscle-rosis and coronary artery disease; as well as diabetes mellitus; renal disease, inflammatory diseases and others.

The selective inhibition of GPVI and/or its signaling may inhibit throm-bosis without affecting hemostatic plug formation. Thus, in contrast to other antithrombotic drugs that are currently in use, GPVI-specific inhibitors are potentially ideal antithrombotics suitable for clinical and lacking a side effect of significant bleeding.

Markets and ApplicationsThe antithrombotics market is growing quickly from a relatively low base. Sales of the ATC B1 class of drugs (platelet aggregation inhibitors) that make up the antithrombotics market reached almost $13 billion in the 12 months to March 2004, reflecting a compound annual growth rate of 18% since 1999.

More than 600,000 Americans now receive coronary stents each year. The cardiovascular stent market is approximately $5 billion a year and is dominated by a handful of companies. Drug-eluting stents, introduced in the United States in 2003, cut the reblockage frequency in half and quickly grabbed close to 90 percent of the market, making improvements to stent coatings and technologies have never been more timely.

The proposed technology may be used in:

Treatment and prevention of diseases involving platelet activation and aggregationProduction of drug-eluting medical devices such as cardiovascular stentsRational GPVI-targeting platelet inhibitor design and optimizationGPVI-targeting drug delivery via conjugation of second therapeutic agent with peptides and com-pounds of this invention

••••

Officers and Directors:Alexander B. Sigalov, Professor


Department of Pathology, S2-302,UMass Medical School, 55 Lake Avenue NorthWorcester, MA 01655Phone: 508-856-8803Fax: [email protected]://www.umassmed.edu/pa-thology/faculty/Sigalov.cfm

Exhibitors 63


Commercial OpportunityTarget customers for the proposed technology include, but are not limited to, the following:

1. Pharmaceutical companies (e.g., Pfizer, Trigen, Otsuka Pharmaceuticals)2. Companies producing drug-eluting stents and other drug-coated medical devices (e.g., Boston Sci-

entific)3. Finally, hospitals, clinics, and individual customers

At present, the proposed technology is available to license.

Competitive advantage At present, there are no GPVI-targeting antithrombotics on the market and bleeding complications are com-mon side effects of the antithrombotic drugs used.

The current state of the art suggests using antibodies against GPVI as therapeutic agents. However, there are many disadvantages associated with antibody therapy, including possible contamination and the high cost of production, storage and administration. The technology proposes novel GPVI-targeting therapeutic inhibitors that lack antibody’s disadvantages.

In addition, this invention is advantageous as the peptide variants:

Avoid the dangerous side effects that can occur with other antithrombotic drugsAre potent, specific and have few toxicological side effectsCan be readily used for making novel antithrombotic-coated stents and other medical devicesCan be made protease-resistantDo not accumulate in organsDo not suffer from drug-drug interactions

Future Financial PlansFuture financial plans and milestones can be discussed in detail with Anita L. Ballesteros, Ph.D., a Licensing Officer in the Office of Technology Management at the University of Massachusetts Medical School (tel. 508-856-6611) and Alexander B. Sigalov, Ph.D., a Research Assistant Professor in the Department of Pathology at the University of Massachusetts Medical School (tel. 508-856-8803).

••••••

64 Exhibitors


University of Massachusetts Medical School*

Presented by:Jie Song, Professor

Product/Technology ProfileOur IP centers on first-of-its-kind deployable, biodegradable bone sub-stitutes designed for a wide range of orthopedic applications that con-stitute a growing multi-billion dollar market. The technology is based on novel material design and robust chemistry, with the capability of fine-tuning a number of parameters key to the performance of synthetic bone substitutes, including in vivo biodegradation rates matching with fracture healing or spine fusion rates, tunable bone-like mechanical properties, osteogenic biochemical microenvironment and efficient sur-gical insertion and deployment with minimal potential tissue damage.

Markets and ApplicationsTarget customers include companies developing off-the-shelf ortho-pedic bone substitutes (e.g. synthetic bone grafts, spine fusion bone substitutes); 2) companies specialized in biodegradable, drug-eluting deployable polymer stents or stent coatings; 3) companies specialized in minimally invasive surgical implants and devices.

Bone substitutes and spine fusion products constitute a multi-billion dollar market, with 10% annual growth. Stent industry has a $1.1-1.5 billion current market.

Commercial OpportunityO Potential orthopedic applications include but are not limited to: 1) the stabilization and expedited healing of hard-to-heal fractures (e.g osteoporotic fracture, diabetic fracture, peri-articular fractures); 2) the repair of segmental bony defects (due to trauma, tumor resection, etc); 3) spine fusion (due to degenerative disc disease, DDD); 4) craniofacial reconstructions; 5) dental applications. With a growing and aging population, the number of people who live with cancer, diabetes, osteoporosis and lower back pain (due to DDD) will continue to increase. The orthopedic co-morbidity of these diseases cause tremendous pain to the patients. High-performance synthetic bone substitutes directly contributing to the relief of patient suffering constitute a sustained billion dollar commercial market.

Competitive advantage There is no existing deployable and degradable shape memory polymer for orthopedic bone substitute appli-cations. Our material is the first of its kind. Major advantages include: 1) tunable mechanical property suitable for orthopedic applications; 2) biodegradation rates matching with spine fusion /skeletal repair rate; 3) novel design, robust chemistry; 4) practical and efficient deployment strategy with minimal tissue damage.


Officers and Directors:Jie Song, Professor


UMass Medical School55 Lake Ave. North, S4-827Worcester, MA 01655Phone: 508-334-7168Fax: [email protected]://www.umassmed.edu/igp/faculty/song.cfm?start=0&

Exhibitors 65


We have a provisional patent filing with broad coverage on the compositions, manufacturing methods and methods of use of the shape memory biopolymers. With our in-house expertise from materials design, syn-thesis and fabrication to in vitro and in vivo testing, we have the ability to optimize the materials performance for tailored biomedical applications. Evaluation of the in vivo performance of a prototype for orthopedic ap-plications using small animal models is on-going in the inventor’s laboratory.

It is estimated that 1/7 of the US population suffer some forms of musculoskeletal impairment. With a growing and aging population, there is a sustained and growing need for high-performance synthetic bone substitutes that directly contribute to the quality treatment of the many debilitating musculoskeletal conditions.

Future Financial PlansOn-going in vivo studies in the inventor’s laboratory are funded by an MTTC award and a UMass award, respectively. These studies are expected to generate critical feasibility data to facilitate the marketing of the IP to orthopedic industry. Other funding is sought after to support both the fundamental understanding of the structure-function relationship of this novel material as well as more in depth in vivo studies using small and larger animal models. Commercialization strategies for the technology include licensing in specific Fields of Use to existing companies on either an exclusive or nonexclusive basis. Further, a start-up company may be formed to develop its own products using this technology.

66 Exhibitors


VasoTech, Inc.

Presented by:Tim Wu, President and CEO

Company Overview VasoTech, Inc. is a medical device company dedicated in cardiovas-cular devices—particularly drug eluting stent research, development and manufacturing. The company was formed on Nov, 2000 at Mas-sachusetts with really operation activities since Jan. 2007. The core technology the company currently has is the PowerStent® Drug Elut-ing Stent which was fabricated by coating a patented potent anti-reste-nosis composition(Combo®, US patent application#: 11/144,917 ) with another patented inflammatory-free biodegradable polymer(BioDe®: US patent application#: 1843528) on an unique designed drug eluting plat-form. Based on the technology, VasoTech Inc. is receiving 1.2 millions SBIR fast-track funding from NIH and is scheduled to initiate the clinical trial in China during the summer of 2008. Dr. Tim Wu, M.D, the founder of the company and the inventor of the technology, is an interventional cardiologist with profound knowledge of bioengineering, material science, mechanical design and business management. He has served NIH as a grant reviewer for the study section of cardiovascular device for a consecutive three years since 2004. He was the PI for several SBIR projects in the field of cardiovascular diseases and is currently the PI for the mil-lion dollar SBIR fast-Track grant: Biodegradable Material and Its Made Drug Eluting Stent.

Product/Technology ProfileRestenosis and late-stage thrombosis are the two major problems existed in current drug eluting stents. No biodegradable polymers used in current commercial available stents (CYPHER and TAXUS stents) are the major causes for these two problems. However, current commercial available biodegradable polymers used in making drug eluting stents are unsuccessful due to primarily the sever inflammatory response to surround-ing arterial tissue associated with polymer’s degradation process.

PowerStent is the combination of an inflammatory-free biodegradable polymer, a potent anti-restenosis drug formulation and a featuring designed drug delivery platform. It features and capabilities exceed any cur-rent commercially available and investigational stents. In PowerStent, the specifically formulated BioDe® polymer will not only prevent the polymer-associated long-term issues such as late-stage thrombosis exist-ed in current non-biodegradable stents, but also eliminate the short-term inflammatory responses associated with the polymer’s degradation. The Combo® drug formulation can block both signal transduction ways of Rapamycine and Paclitax-el and, therefore, the anti-Restenosis effects of PowerStent is better than any single Cypher® or Taxus® stent has. Finally, the featuring designed VasoTech® metal stent provides PowerStent a most deliverable and conformable platform with sufficient supporting force for diseased arteries.

Figure in the above is the crossing section of PowerStent®, BioDe®, and PLGA only coated stents in pig coronary arteries for 28 days. As shown in the image, BioDe® polymer (B) coated stent has much improved biocompatibility (less inflammation, less neointima ) than PLGA only polymer(A). While the PowerStent(C)

Officers and Directors:Tim Wu, President and CEO


55 Plainfield Ave, Shrewsbury, MA 01545Phone: [email protected]

Exhibitors 6�


has re-endothelialized inner wall with minimized tissue inflammation response and neointima formation com-pared with either BioDe® or PLGA only coated stents.

Market and Application The market for drug-eluting stents is one of the fastest growing segments within the medical device arena with an annual growth approaching 25%. The global market for coronary stents in the US was $5.5 - $6.0 billion in 2006, and is expected to grow to over $7 billion over the next two years. The growth is due in part to the higher selling price of drug eluting stents versus bare stents, an increase in the average number of drug-eluting stents per patient, and the expanding range of indications for stenting resulting from the treatment of more complex lesions. Physicians sometimes insert 2-3 stents end-to-end to treat twisted arteries.

Commercial OpportunityDespite technical advancements, of which coronary stenting has been the most significant, restenosis and stent thrombosis remain the major problems that hampers the procedure’s efficacy. Stent restenosis rates are reported to be approximately 10% with today’s DES in ideal coronary lesions, but may occur in over 20–40% of patients with complex lesions (e.g., small vessel, diffuse atheromasia, and bifurcation lesion). Late-stage thrombosis occurred in both Cypher® and Taxus® DESs as recently reported, although a small number has a significant impact on current daily management of cardiovascular patients. Therefore, there exists a market need for a more effective, low or non toxicity drug eluting stent to completely eliminate in-stent restenosis and stent thrombosis. PowerStent® DES will fill out this market need.

PCI has become the main method for coronary arterial revascularization. More than 1.5 million procedures take place in the United States each year, in which 80 % are using stents. The current annual worldwide market for stents only is estimated to be greater than $2 billion. With the evolution of stent design, which will produce increasingly safer and easier-to-use devices, the use of PCI procedure is expected to be extended more widely in such diseases as small vessels, multi-vessels disease etc. Therefore the commercial value of PowerStent is tremendous within the foreseeable future.

Competitive advantage Currently Johnson and Johnson’s Rapamycine-coated Cypher® and Boston Scientifics’ Paclitaxel coated Taxus® stents are the only two FDA-approved DESs in the US. Compare to these two stents, PowerStent has two advantages: 1) the polymer in PowerStent is highly biocompatible and biodegradable; therefore, there should be no or less late-stage thrombosis as existed in both Cypher and Taxus stents. 2) The drug in PowerStent is more potent in inhibit restenosis than either Rapamycine or Paclitaxol does.


VasoTech, Inc. currently has two US patents pending and 1.2 million funding from NIH SBIR fast track grant. Those SBIR fund will be used mainly for R&D purpose including preclinical studies in pig coronary arterial implantation. By completing this SBIR project, we will collect most valuable data needed for next clinical studies. The China market of those intellectual properties has been licensed exclusively to Tianyi Scientific, Inc. ---a China based medical device company. The company currently has built a manufacturing facility with over 3000 SF of Class 10,000 and 300SF of Class 100 clean room in China. The company is performing the preclinical studies with PowerStent® and scheduled to initiate the clinical trial around Aug, 2008. Since the China market is relatively easier to penetrate than both US and European markets, Tianyi Scientific Com-pany in China is expected to receive China state FDA Approval around the end of 2009.

The most urgent need for VasoTech, Inc’s expansion currently is to have a GMP manufacturing facility for next clinical trial samples preparation. The facility can be build either in US or from oversea such as China. Purchasing Tianyi Scientific Inc has several advantages: 1) get manufacturing facility immediately. 2) pen-etrate China market immediately. 3) More economic than build a brand new facility in US. Therefore, the

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transfer center (mttc) and the massachusetts association...

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