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TRANSCRIPT
STEVEN DUBINETTMICHAEL PALAZZOLOSTEVEN GEORGE
JUNE LEEVISH KRISHNAN
University of California Center for Accelerated Innovation
National CAI Kick-Off MeetingOctober 29, 2013
Contents
• Consortium Membership & Expertise• Leadership and Governance Structure • Center Resources • Technology Solicitation to Exit • Skills Development Overview • Defining Success: Metrics & Deliverables
University of California,
Los Angeles
UC-BRAID
UC Biomedical Research Acceleration Integration and Development (UC-BRAID)
UC Biomedical Research Acceleration Integration and Development (UC-BRAID)
Investigator
• IRB harmonization• Master contracting
• Integrated Research Data Repository
• Harmonizing biobanking
• 12 million patients
UCSF
UC Davis
UCLA
UC San Diego
UC Irvine
• UC CAI
• UC ReX– Enables search of 12 million de-identified patient
records from the 5 UC medical centers– Counts of eligible patients by gender, race,
ethnicity
• EngageUC– Global consent and biobanking– $2-million in supplemental funding from NCATS
UC Biomedical Research Acceleration Integration and Development (UC-BRAID)
Expertise
• Rich research base: 7% of NHLBI’s FY2012 grant funding
• Proximity to large biomedical industry clusters in San Diego, Irvine/Orange County and San Francisco
• More than 70 industry experts in:– heart, lung and blood diseases– technology platforms– commercialization
Expertise
• History of innovation:
– breath biomarkers for asthma (D. Cooper) – a hemodynamic system for transfusion of blood
products and administration of blood pressure medications (J. Rinehart)
– hydrogel for cardiac tissue repair (K. Christman)
– pulmonary vein ablation catheter for atrial fibrillation (M. Lesh)
– biologic for idiopathic pulmonary fibrosis (D. Sheppard)
– biopolymer implant for ventricular reconstruction in congestive heart failure patients (R. Lee)
Michael Palazzolo, MD, PhD - Center Director
• Director of Human Genome Center, Berkeley Lab
• Associate Director, Drosophila Genome Project, Berkeley Lab
• Senior Director of Biosystems, Amgen– led 270 scientists conducting high-throughput genomics
research
• Partner at Coastview Capital, a Los Angeles-based venture capital firm
• Project manager– international Stand Up to Cancer collaboration– international, multiyear collaboration between academic
laboratories at U Toronto (Mak) and UCLA (Slamon)
Introduction to Leadership
Contents
• Consortium Membership & Expertise• Leadership and Governance Structure• Center Resources• Processes: Technology Solicitation to Exit• Skills Development Overview• Defining Success: Metrics & Deliverables
Local and National Announcements
Posted on Sept 30, 2013 Aired October 1, 2013
CTSA Central NBC Channel 4 News
Governance
Center Director
Associate Director
Domain/Site Leaders
Cardiovascular
Therapeutics Devices
Lung & Sleep Disorders
BloodDiseases
Diagnostics
Domain Areas
Projects
Program Resources
Administrative& Budgetary
Support
Website & Data Management
Industry Relations & IP
CTSA Infrastructure
Evaluation & Tracking
Project Management
External Selection
Committee
Business Review Panel
Skills Development Program
External Advisory Board
Executive Committee
Site Leaders
Campuses
June Lee, MDUCSF
Shaun Coughlin, MD,
PhDUCSF
Sotirios Tsimikas,
MDUCSD
Joseph Witztum,
MDUCSD
Laura Marcu, PhDUC Davis
Steven George, MD,
PhDUC Irvine
Tomas Ganz, MD, PhDUCLA
Domain Leaders & Skills Development Program
Shaun Coughlin, MD,
PhDCardiovascular Disease
Sotirios Tsimikas,
MDCardiovascular Disease
June Lee, MDLung and Sleep
Diseases
Tomas Ganz, MD, PhDBlood Disorders and Resources
Vish KrishnanSkills Development
ProgramUCSD
Dis
eases
Domain Leaders
Platforms
June Lee, MDCo-Leader,
Therapeutics
Shaun Coughlin, MD,
PhDCo-Leader,
Therapeutics
Sotirios Tsimikas, MDCo-Leader, Diagnostics
Joseph Witztum, MDCo-Leader, Diagnostics
Laura Marcu, PhDCo-Leader,
Devices and Tools
Steven George, MD,
PhDCo-Leader,
Devices and Tools
External Advisory Board
• Consists of no fewer than 5 members• Experienced business leaders• Includes NHLBI Program Officer• Advice about operations, project
development
Catherine Mackey, PhDFormer Senior VP, Pfizer
Founder, MindPiece Partners
Francis Duhay, MDVP Medical Affairs and CMD,
Edwards Lifesciences
Lawrence Souza, PhDFormer Senior VP, Amgen
Founder, Coastview Capital,
Business Review Panel
• Five members
• VCR on each campus appoints one member
• Evaluate Center’s progress toward sustainability
Bill Ouchi, PhDUCLA Initial ChairAnderson School
Progress to Date
• Campus meetings• Face-to-face meetings• Site and domain leadership meetings• Website development• Administrative meetings• 100-day Implementation Plan
Progress to Date
Progress to Date
Contents
• Consortium Membership & Expertise• Leadership and Governance Structure• Center Resources• Processes: Technology Solicitation to Exit• Skills Development Overview• Defining Success: Metrics & Deliverables
Center Resources by Development Stage
• Discovery– Cardiovascular Research Institute (UCSF)– Lung Biology Center (UCSF)– Biomarker Laboratory (UCSD)– Drug Discovery Institute (UCSD)– Institute for Engineering in Medicine (UCSD)– Small Molecule Discovery Center (UCSF)
• Pre-clinical and Clinical– Cardiovascular Physiology Core (UCSD)– National Primate research Center (UCD)– Large Animal Survival Science Service Facility (UCD)– Airway Clinical Research Center (UCSF)– Animal Care Program Diagnostic Laboratory Services
(UCSD)– Applied Physiology-Human Performance Lab (UCI)– UC Medical System
Center Resources by Platform• Diagnostics
– Translational Pathology Core Laboratory (UCLA)– Tissue Array Core Facility (UCLA)– West Coast Central Comprehensive Metabolomics
Resource Core (UCD)– Crump Institute for Molecular Imaging (UCLA)’
• Therapeutics– GMP Facility (UCD)– Medicinal Peptide Synthesis Core (UCLA)– Center for Molecular and Genomic Imaging (UCD)– Metabolomics Central Service Core (UCD)– Molecular Screening Shared Resource (UCLA)
• Devices– Edwards Lifesciences Center for Advanced
Cardiovascular Technology (UCI)
Center Resources for Commercialization
• Entrepreneurial– von Liebig Center for Entrepreneurism and Technology Advancement
(UCSD)– Business of Science Center (UCLA)– Harold and Pauline Price Center for Entrepreneurial Studies (UCLA)– West Health– CONNECT– BIOCOM
• Incubators– QB3 (UCSF)– Institute for Technology Advancement (UCLA)– Center for Innovative Therapeutics (UCSD)
• Industry Partners– Bristol-Myers Squibb– Care Fusion– Edwards Lifesciences– Life Technologies – MedImmune Ventures– Pfizer Centers for Therapeutic Innovation– Quest Diagnostics
Edwards Lifesciences CenterResearch Vision
Heart valve fluid dynamics Perfused human microtissues
(Professor Kheradvar) (Professors George, Lee, and Hughes)
• 6 core (22 affliated) faculty in 13,000 asf• 40 doctoral students and postdoctoral researchers• Research focus areas:
– Valve replacement technology– Regenerative cardiovascular medicine– Non-invasive (wireless) cardiovascular monitoring– Novel stent or catheter-based therapies
Edwards Lifesciences CenterTraining Vision
Train future translational cardiovascular researchers at all levels (undergraduate, graduate, post-doctoral)
• Training fellowships from endowment• Cardiology Fellow (3-yr dedicated research time)• NIH T32 training grant (CARE Program)
• Business plan competition• 6 funded slots (3 doctoral positions per year)
• E-SURP (paid summer internships – UCI ugrads)• Summer Scientists (high school students)
Contents
• Consortium Membership & Expertise• Leadership and Governance Structure• Center Resources• Processes: Technology Solicitation to
Exit• Skills Development Overview• Defining Success: Metrics & Deliverables
Technology Selection Overview
• Solicit 2-page pre-applications
• Each campus reviews and selects the best proposals for full application
• 1st Review: Highest ranked proposals from each campus submitted for review by panels assembled by Domain Leaders
• 2rd Review (External Review): External Selection Committee scores proposals and sends to NHLBI
• 3rd Review: NHLBI makes final selection
Technology Selection Overview
Pre-applicationReview
RFP Pre-application
FullApplication
External Selection Committee Review
NHLBI Review
UC BRAID Review Committee
Technologies Selected for
Entrance to Center
Technology Selection Timeline• Annual solicitation for 3 tracks
– Therapeutics
– Devices/ digital health
– Diagnostics
• Timeline from initial solicitation to External Selection Committee recommendation: 6 months
• Awards of up to $200K/2yrs
Solicit Pre-
Application
Review Pre-
Application
1 month
Develop and Submit Full Application
First Review of
Full Application
ESC Review of
Application
1 month 1 month 1 month2 months
Pre-application and Review
Pre-applicationReview
RFP Pre-application
FullApplication
External Selection Committee Review
NHLBI Review
UC BRAID Review Committee
Technologies Selected for
Entrance to Center
Eligibility
• Faculty in all series and ranks at UC Davis, UC Irvine, UCLA, UC San Diego, and UC San Francisco
• Postdoctoral scholars are eligible to submit applications as Co-PI with a faculty PI
• Projects with existing or imminent target validation and a clear clinical indication
• Patents or patent applications are filed or potential for obtaining defensible intellectual property is strong
Solicitation Process
• Broad solicitation– Focus on NHLBI priority areas (heart, lung,
blood)
• Centralized RFP for all 5 campuses
• Webinar on submission process
• Each campus is accountable for supporting the highest potential projects with product development related issues
Two-page Pre-application• Centralized online submission
• Two-page pre-application contains:– Executive summary
• Unmet Need/ Clinical impact• Research/development/ regulatory plan • Intellectual Property• Business strategy/Commercialization plan
• Local review of two page pre-applications assess:– Scientific merit– Product development potential
• Highest potential projects will be requested to submit full proposals
Technology Selection: Full Application
Pre-applicationReview
RFP Pre-application
FullApplication
External Selection Committee Review
NHLBI Review
UC BRAID Review Committee
Technologies Selected for
Entrance to Center
UC BRAID Review Committee
• Selected by Domain Experts/Platform Leads
• UC BRAID Executive Committee
• Domain Leads, Site Leads, Platform Leads
• External experts from industry
Full Application
• Review Criteria– Unmet medical need– Development feasibility– Commercial attractiveness– Intellectual property status– Relevance to NHLBI mission– Metrics for success
oEvidence of target validation (therapeutic)oTime and cost of prototyping (device)oCombination of the above (diagnostic)
Full Application: External Selection Committee
• Prioritize applications received from Leadership Review
• Same review criteria as Leadership Review
• 1 month to review
• Reviewers– Targeting total pool of 100– Selected to review proposals based on
domain/functional expertise– Must be external to institution(s)– Chair/Co-Chair to finalize recommendations to
be submitted to NHLBI
Technology Development Pipeline
Pre-applicationReview
RFP Pre-application
FullApplication
External Selection Committee Review
NHLBI Review
UC BRAID Review Committee
Technologies Selected for
Entrance to Center
Referral
Referral
Consultation Award
Consultation Awards
• Eligibility– Proposal not selected for Center but
identified as high potential– Leadership Review or External Review
recommends Consultation Award consideration
• Amount and duration of awards vary– Most awards for 3-6 months
• Recipients must agree to resubmit and target a specific RFP for resubmission
Consultation Awards
• Awards will address the following gaps:– In vivo proof of principle – Hypothesis testing – IP assessment – Target product profile discussion – Regulatory assessment – Further development planning
• Based on results of work done during award, awardee may be invited to submit full proposal for subsequent cycle
Contents
• Consortium Membership & Expertise• Leadership and Governance Structure• Center Resources• Processes: Technology Solicitation to Exit• Skills Development Overview• Defining Success: Metrics & Deliverables
Key Goals and Objectives
Offer actionable cross-campus skills development opportunities that:
• Encourage PI’s to consider commercialization considerations when writing grant proposals
• Impart core project management and business planning skills for life sciences projects.
• Improve awareness of market, financial, and IP issues in the early stages of a project.
• Guide researchers on the assessment of technologies on their readiness and risks
• Mitigate various types of risks with appropriate project design and partnership business models.
Skills Development CanvasEngage “Proto-innovators”
Activ
ities
Funded Prospective Post-Docs Fellows Grad Faculty Faculty Students
Commercialization Content Portal (Readings, Videos, Cases)
Advisory Services/Clinics
Courses/Workshops on Commercialization
Networking Events and Contests
Segments
Education and Training• Technology Commercialization Primer
– Gateway course for Center innovators– Teach innovators how to frame proposals for Center– Covers technology readiness, market research, risk mitigation,
competitive analysis, company start up– Builds on existing courses
• Lab to Market (UCSF), Idea to IPO (UCSD)
• Catalog commercialization resources across Center– Business plan development– SBIR grant writing– IP, licensing, contract negotiation– Existing courses on campuses
• Match innovators with educational and networking events
• Annual Heart, Lung, Blood Technology Forum– Innovators present progress and lessons learned– Networking with industry participants
Mentoring and Advising• Highly experienced industry professionals,
venture investors, entrepreneurs
• “Mentor the Mentor” training– Modeled on UCD Mentoring Academy– Recognition for superior mentoring
• Three levels of mentoring:– Generalist -- Help innovators prepare competitive proposals– Lead -- Guide innovators selected for the Center– Specialist -- Available to address specialized problems during
technology development
• Online Commercialization Clinics– Led by mentors– Held quarterly– Archived on Center website
Feedback • Compile data on technology pipeline and
licensing activity– Provide feedback to Center on investment
needed to enhance attractiveness of pipeline
Timing• Develop and launch Technology
Commercialization Primer in year 1
• Mentoring programs and technology commercialization clinics in years 1-2
Contents
• Consortium Membership & Expertise• Leadership and Governance Structure• Center Resources• Processes: Technology Solicitation to Exit• Skills Development Overview• Defining Success: Metrics &
Deliverables
Goals
• Build an entrepreneurial ecosystem in the NHLBI domain – skills development
• Identify the best technologies across all 5 UC campuses
• Incubate 30-40 of the most promising technologies within the center towards a commercially rewarding exit
• Sustainability
Why Did We Institute a Business Review Panel?
• Each campus has the responsibility to manage its own exits
• We agreed there isn’t a dataset that exists that would allow the selection of an optimal exit strategy for all five campuses
• Best approach would be to run an experiment and use the data to guide us in the future
• The data would best be evaluated by experts outside the Center reporting to the EC and not the Center
Why Is Sustainability Hard?
University Research
Tech Transfer
Venture Capital Biotech Pharma
The technological bottleneck is the mechanistic understanding of a disease that can be used to validate therapeutic intervention points. This happens largely in the university.
However, the value transition point occurs at proof-of-concept in clinical trials in phase I or phase II. This currently takes place in Biotech and Pharma.
Sustainability May become Even Harder
• “However, although investment in pharmaceutical research and development has increased substantially...the lack of a corresponding increase in terms of new drugs begin approved indicates that therapeutic innovation has become more challenging.”– Nature Reviews. Drug Discovery 10, No. 6,
pp.428-438.
• “venture financing for biotech has been in decline...Some venture capitalists have stopped funding new biotech altogether.”– Wall Street Journal, March 16, 2012
Bill Ouchi’s Views on Problems That Need to Be Solved to Achieve Sustainability
• Tech transfer offices are undercapitalized
• Tech transfer offices tend to be highly politicized
• Tech transfer offices frequently have insufficient business expertise and management
• Faculty need mentoring
• Universities may need to step up to incubate technology to the point of proof of concept on its most promising programs