BioMarin's Gene Therapy Manufacturing Facility Recognized with Industry Award

Congratulations to our gene therapy product management team on their recognition from the ISPE (International Society for Pharmaceutical Engineering) for “Facility of the Year for Project Execution.” ISPE annually recognizes projects that demonstrate global leadership by showcasing cutting-edge engineering, innovative new technology or advanced applications of existing technology.

As you all know, last year BioMarin opened one of the largest gene therapy manufacturing facilities in the world, allowing us to control the process of product and drug development from scheduling, costs and quality. The 18,000 square foot facility in Novato is a contribution to the scientific possibilities in our pipeline with the goal of offering best-in-class and first-in-class therapies to patients with rare diseases.

The project could not have been possible without the dedication of over 300 people and 200,000 construction hours. The product management and contractor teams worked tirelessly to complete the project under an aggressive timeline and almost 7 months faster than a typical schedule for a facility of this size.

The timing of the facility was the highest priority because our hemophilia A treatment valoctocogene roxaparvovec (Valrox) just entered into its global Phase 3 development. The accelerated deadline for the facility in order to produce material for the clinical studies and potentially for commercial demand, led the team project a goal to release the first filled vials of Phase 3 clinical drug product in 18 months from initiation. Now, we are well positioned with the necessary resources to proceed with the development program for Valrox.

“The buildout of our gene therapy manufacturing facility was a logistical challenge that required leveraging our experience gained during licensure of our Galli and Shanbally biological facilities. This

plant provides us with scheduling flexibility allowing for rapid product development and is capable of providing sufficient capacity to meet clinical and projected commercial requirements," said Robert Baffi, Ph.D., Executive Vice President of Technical Operations at BioMarin. "In addition, it creates the opportunity for our engineers and scientists to establish a valuable knowledge base required for manufacturing complex gene therapy products. This project demonstrated how the industry's best people, equipped with the right technology and energized by the right motivation, can make a seemingly impossible project possible."

Carl Albertson, Director of Capital Projects and Logan Kelley, Senior Project Manager explained the development was a rapid success due to the combination of a cohesive team with an organized plan. They started early to gather a team built on trust and communication.

Above: BioMarin colleagues inside the gene therapy facility.

The key strategic differentiator for the project was cohesive implementation of the design and construction instead of piecemeal. While there was overlap with construction and design, there was also overlap with operations, primarily because of the early target date to start manufacturing. Designing in the moment takes risk management and structure, but the team leveraged a continuous culture coupled with the best expertise in the industry. Applying these features all at once allowed for an expedited process, without sacrificing quality.

“It really came down to the commitment of our people,” said Albertson, highlighting the team who gave up holidays, weekends and their personal lives for about a year. “It takes heart and that is the bottom line.”

The ISPE award submission told the inspiring behind-the-scenes story of the construction plans, operation control, time commitment and leadership that lead to the opening of our manufacturing facility for the clinical development of future therapies.

Congratulations again and a special thank you to Carl Albertson, Logan Kelley and Eric Rico for their passion and leadership.

ISPE formally announce the 2018 winners (click here) during the 2018 Europe Annual Conference on March 20 in Rome during the Keynote address.

Click here to see our press release.

Congrats BioMarin!