The Center for Breakthrough Medicines (CBM) announced that it had completed the technology transfer of the University of Pennsylvania Gene Therapy Program’s (GTP) adherent cell manufacturing platform for producing adeno-associated virus (AAV) gene therapies.
Through the agreement, CBM becomes the exclusive contract development and manufacturing organization (CDMO) partner for the platform, allowing the company to provide its clients with a platform for AAV vector manufacturing.
According to GTP, the platform focuses on the development of next-generation gene transfer vectors and their application in the treatment of acquired and inherited diseases.
The platform has been used to manufacture 100 production scale batches and production runs for four clinical phase programs, the partners stated. Further, they noted that due to limited process development, and materials ready to be used, the platform can enable clinical candidate selection to toxicity studies in as little as four months and clinical candidate selection to good manufacturing practice (GMP) manufacture in as little as six months.
Jennifer Manning, SVP of Global Strategic Partnerships at CBM, said, “The platform, combined with CBM's manufacturing expertise and state-of-the-art, high throughput cGMP suites, as well as on-site experts, provides innovator companies the critical elements required to efficiently translate lifesaving therapies from bench to bedside.”
Additional services provided by the platform include aiding the acceleration of development through assays and analytics, onsite testing, reduced sample requirements, and reduced testing turn-around times, CBM stated.
In terms of the downstream process, the platform is able to ensure an average recovery of 30% and greater than 80% full capsids by analytical ultracentrifuge analysis, the partners stated. In addition, up to three upstream runs can be performed into a single downstream harvest, with a typical yield of 4.8E16 total GC at harvest.
The partnership deal to gain access to the GTP platform is one in a series of such moves that CBM has carried out. In March 2023, CBM announced two deals, one with Nucleus Biologics, a provider of customer cell culture media solutions, and one with Stoic Bio, a provider of sustained technology for cell media manufacturing.
The month prior, CBM partnered with Virion Therapeutics to develop the latter’s checkpoint modifier programs, including a first-in-human immunotherapy for chronic hepatitis B virus infection. According to CBM, its aim is to become “the number one advanced therapies CDMO.”