ASC and Expression Therapeutics Collaborate on Hemophilia Gene Therapies

ASC Therapeutics recently announced that they have obtained an exclusive therapeutics license from Atlanta, GA-based Expression Therapeutics. ASC, a division of Applied StemCell, Inc., specializes in the development of proprietary gene editing technologies. Expression Therapeutics develops novel technologies as well, including several gene therapies for hemophilia A and B that utilize adeno-associated viral vectors (AAVs). AAVs carry genetic codes to the liver via a one-time administration in order to prompt the production of therapeutic levels of clotting factors such as factor VIII or IX in individuals with hemophilia A and B.

According to a recent press release, ASC, which is headquartered in Milpitas, CA, has successfully completed a pre-Investigational New Drug (IND) meeting with the U.S. Food and Drug Administration (FDA). The focus of the meeting was a hemophilia A gene therapy platform based on a vector known as ASC-618. ASC is currently conducting toxicology studies prior to filing an IND application with the FDA.

“We have a common mission with Expression Therapeutics – to develop novel therapies that will dramatically improve the lives of persons with hemophilia around the world. Hemophilia A is a very serious clinical condition with an unmet clinical need,” said Ruhong Jiang, PhD, CEO and founder of ASC. “Our partnership with Expression Therapeutics will enable our hemophilia A gene therapy program ASC618 to leverage Expression Therapeutics’ proprietary technologies allowing for maximal therapeutic efficacy for one of our lead therapeutic candidates.”

“We are very excited to partner with ASC to bring a gene therapy treatment to patients with hemophilia A, said Mohan Rao, PhD, CFO of Expression Therapeutics. “ASC is a leader in gene editing and stem cell technologies and we look forward to developing innovative and cost-effective therapies for curing hemophilia.”

Source: The Associated Press, May 16, 2019