CBM and Penn Strike $100M Cell & Gene Therapy Deal

The Center for Breakthrough Medicines (CBM) and the University of Pennsylvania’s Gene Therapy Program (GTP) have signed a landmark collaboration agreement, to combine Penn’s knowledge and expertise in the development of gene therapies with CBM’s manufacturing capacity and singular focus on advanced therapies.

This partnership is focused on moving therapeutics safely and rapidly from concept to clinic by connecting certain of Penn’s established gene therapy platforms with CBM’s manufacturing and analytical capacity. This combination will allow small biotech firms, universities, and large pharmaceutical companies to advance gene therapies from discovery to First-In-Human (FIH) studies with the potential for less risk and expense. By utilizing Penn’s gene therapy expertise, which has already been applied to numerous commercially sponsored clinical programs and avoiding the typical capital-intensive infrastructure builds associated with gene therapy manufacturing, gene therapy developers will be able to enter the field sooner and with less risk with confidence that this platform is scalable for later clinical stages and commercialization.

Under the renewable five-year deal, CBM will be the only for-profit contract development and manufacturing organization (CDMO) with commercial rights to certain of GTP’s gene therapy manufacturing platform, advanced analytics, and any future process or analytical improvements achieved through its collaboration with GTP.

“This agreement represents a giant leap forward for the Center for Breakthrough Medicines and its mission to accelerate the development and manufacturing of life-saving gene therapies,” said Audrey Greenberg, co-founder, Center for Breakthrough Medicines. “This partnership with the GTP at Penn enables our clients the potential to advance to Investigational New Drugs with a high-quality process, materials and analytical methods.”

Gene therapies hold promise to treat and cure both rare and non-rare hematological, ophthalmic, musculoskeletal and neurologic diseases such as hemophilia, choroideremia, Duchenne muscular dystrophy, and Huntington’s disease, as well as oncology solid and non-solid tumor diseases. CBM is committed to successfully scaling up viral vectors to commercial scale and producing consistent personalized products for any patient population.

“Our mission at the Penn Gene Therapy Program is to discover, translate and greatly accelerate the development of next-generation gene transfer vectors and their application in the treatment of a variety of acquired and inherited diseases,” said James M. Wilson, MD, PhD, Director, Gene Therapy Program; Rose H. Weiss Professor and Director, Orphan Disease Center; and Professor of Medicine and Pediatrics, Perelman School of Medicine at the University of Pennsylvania. “This partnership is ideal in furthering that mission.”

As part of the partnership Penn will receive access to a best-in-class, state-of-the art GMP manufacturing partner with 700,000 planned square footage capacity plus a Sponsored Research Agreement to continue advancing the field of gene therapies. A primary goal of the partnership is to decrease the development costs, timelines and manufacturing Costs of Goods Sold (COGS), thus increasing the accessibility of life-changing treatments to patients. 

“Our collaboration with the GTP at Penn will allow CBM the ability to offer accelerated gene therapy manufacturing services under one roof regardless of where a program is in its development timeline,” said Joerg Ahlgrimm, president and CEO, Center for Breakthrough Medicines. “With this access to GTP’s gene therapy production expertise, CBM becomes an appealing manufacturing partner with a differentiated offering. We will offer the best of an academic vector core (speed to clinic) and commercial CDMO (commercially viable process) combined.”

This deal comes on the heels of Penn GTP’s expansion to the Discovery Labs’ campus, located in the same facility as CBM, which creates a further synergized collaboration between the two organizations.