Animal Origin Free Strategies for Gene Therapy Manufacturing and Formulation

High-Performance, Regulatory-Friendly Viral Vector Production for Gene Therapy

After decades of effort, the field of gene therapy has finally reached critical mass. The FDA has now approved two adeno-associated viral vector (AAV) therapies, LUXTURNA (voretigene neparvovec-rzyl) for inherited retinal dystrophy and ZOLGENSMA (onasemnogene abeparvovec-xioi) for pediatric, inherited spinal muscular atrophy. An attenuated, genetically modified herpes simplex virus, IMLYGIC (talimogene laherparepvec), has also been approved for local oncolytic viral therapy of unresectable cutaneous, subcutaneous, and nodal melanoma lesions after postsurgical recurrence. There are now hundreds of gene and cell therapies in the FDA approval pipeline, on pace to reach as many as 10 to 20 related approvals per year by 2025.

Gene therapy relies on viral vectors like AAV or lentiviral vectors, which deliver the desirable genetic sequences into the cells of the target tissue. To produce enough material for clinical use, the viral vectors must be propagated in a host cell such as HEK293. Cultured cells and viral vector products destined for cell and gene therapy or other clinical cGMP applications must meet the highest standards for manufacturing quality, safety, performance, consistency, and efficiency. InVitria has carefully optimized its animal-free, serum-free, defined media formulations for clinically and industrially significant cultured cell lines. InVitria’s product line and media formulations services take the guesswork out of transitioning to regulatory-friendly, serum-reduced, or serum-free cell culture for your RUO or cGMP needs.

InVitria’s Products for Gene Therapy Include:

• Cellastim S recombinant human albumin to replace BSA or purified human albumin in cell culture.
• Exbumin excipient-grade recombinant human albumin for improving stability of viral vector particles in injectable therapeutics.
• Optiferrin recombinant human transferrin for managing iron in serum-free or serum-reduced culture applications.
• Insulin-transferrin supplements provide supplemental rh-insulin, rh-transferrin, and selenium, with or without ethanolamine and rh-albumin for serum-free or serum-reduced culture applications.