Charles River Laboratories and ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in vivo gene replacement, gene editing and allogeneic cell therapies, have agreed to manufacture ASC618, a second-generation gene therapy for hemophilia A.

Hemophilia A is caused by the lack of the blood clotting factor VIII (FVIII), a protein whose instructions are provided by F8, a gene that is mutated in people with the disease. ASC618 is designed to deliver a shortened, but optimized version of the gene to liver cells. ASC Therapeutics has received IND clearance, as well as key regulatory designations in the U.S. and Europe for its ASC618 program.

Viral Vector Manufacturing Services

Since 2019, ASC Therapeutics has worked with Charles River, previously Vigene Biosciences, to leverage the company’s expertise in Good Manufacturing Practice (GMP)-virus manufacturing, and 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.

The collaboration will take advantage of Charles River’s end-to-end CDMO capabilities, building on the company’s acquisitions of Cognate BioServices, Cobra Biologics and Vigene Biosciences in 2021 that expanded its comprehensive cell and gene therapy portfolio to span each of the major CDMO platforms—cell therapy, viral vector and plasmid DNA production.

“We are proud to work with ASC Therapeutics as they progress their second-generation gene therapy program for hemophilia A to clinical stage,” said Kerstin Dolph, senior vice president, global biologics, Charles River. “For three years, our collaboration has relied upon a strong and transparent relationship to support a seamless transition between process development and GMP production, and we’re excited to expand that work.”

Ruhong Jiang, CEO, ASC Therapeutics, said, “Our work with Charles River has a strong foundation of trust—strengthening our shared outlook for the future and building confidence in our internal processes and oversight of external capabilities. As we moved toward the next phase of our therapeutic development, we are excited to work with Charles River to further maximize our manufacturing know-how.”