Ramin Baghirzade, senior director and global head of commercial gene therapy services, Charles River Laboratories

The cell and gene therapy (CGT) sector has reached a significant turning point. Between 2017 and 2022, regulatory agencies approved only five gene therapies. But in 2023 alone, five more were approved. The Alliance for Regenerative Medicine (ARM) predicts another record year for 2024, estimating approval for up to 14 gene-therapy products by the European Medicines Agency (EMA) and/or the US Food and Drug Administration (FDA). “It’s a great time to be in the industry,” Baghirzade said. Gene therapy is “no longer science fiction, it’s becoming a reality.”

Despite the industry enthusiasm, drug developers and contract development and manufacturing organizations (CDMOs) still face conflicts with one another about how to approach CGT production. Baghirzade described two common interactions that cause friction between the two entities: Sometimes drug developers approach CDMOs to create products with only gene-of-interest (GoI) sequences prepared. And in another scenario, drug developers already running clinical trials require more or different materials and reach out to a CDMO for help.

To solve those issues, Charles River Laboratories has developed some fit-for-purpose solutions. For clients still in early development stages, the company offers two plug-and-play navigation platforms. And to address the problem of incorporating new or additional materials into clinical trials, Charles River created a viral-vector technology transfer program. Baghirzade described it as a “Goldilocks” solution: Charles River teams work with clients side by side to find a balanced, “just right” approach to process development without over- or underengineering the solution. The transfer program offers a fast-track approach and a modular framework to address different customer needs during drug development.

The fast-track program is intended for clients that need a drop-in process compliant with good manufacturing practices (GMPs). The Charles River team takes the client’s product as is and simply generates more of it. Development then can continue with quality-control (QC) testing and release, stability studies, and regulatory support. On the other hand, the modular program works best for processes that are not sufficiently robust or that require additional development. Adjustments are made to the client’s process as needed while it is being approved for GMP production.

With over 20 years in the viral-vector CDMO business and over 200 combined years of experience within the manufacturing science and technology (MSAT) team, Charles River Laboratories has completed over 50 successful technology transfers for clients. The company also has supported the development of 86% of novel, FDA-approved drug products and will assist in the development of 20 more drug products for CGTs. About 1000 CGT studies are conducted at Charles River every year. “We know that CDMOs come and go, but we’re here to stay for many more decades to come,” Baghirzade concluded.

Watch Online

See full presentations online at https://bioprocessintl.com/ategory/bpi-theater/bpi-theater-bio-2024.