Charles River and ASC Therapeutics expand gene therapy manufacturing agreement
ASC618 is designed to deliver a shortened but optimized version of the F8 gene to liver cells (the gene is mutated in people with Hemophilia A, preventing them from producing a blood clotting protein).
Currently in pre-clinical trials, the gene therapy from California-headquartered ASC has received IND clearance and Fast Track Designation from the US FDA.
In addition, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion for an Orphan Medical Product Designation of ASC618.
New processes for AAV production and purification
ASC Therapeutics and Charles River have been working together since 2019 on GMP-virus manufacturing and have established processes for AAV production and purification.
“Together, Charles River and ASC Therapeutics have established a high-yield upstream process for AAV8 production, optimized the downstream purification methods to result in a more predictable drug product output, and fine-tuned a scalable manufacturing process in both upstream and downstream,” say the companies.
Charles River made a series of acquisitions in 2021, and the company says that through gaining Cognate BioServices, Cobra Biologics, and Vigene Biosciences it has enhanced its end-to-end CDMO capabilities. Its platforms now cover cell therapy, viral vector and plasmid DNA production.