“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,” Ruhong Jiang, PhD, CEO & founder of ASC, said in a press release.
Hemophilia A is caused by mutations in the gene holding the instructions to produce blood clotting factor VIII (FVIII), and is mainly treated with therapy that replaces that missing or faulty factor via regular injections of a lab-made version of FVIII into the bloodstream.
FVIII is mainly produced by cells in the liver; gene therapies for hemophilia A aim to deliver a healthy copy of the FVIII gene specifically into the liver. This will induce the production of a working clotting factor.
ASC Therapeutics is developing gene therapies for hemophilia A and will take advantage of Expression Therapeutics’ expertise in packaging the large FVIII gene into adeno-associated virus (AAV). The common, naturally occurring virus has been shown to work as an effective gene therapy delivery vehicle, efficiently targeting the gene into liver cells. This has led to positive results in preclinical models of hemophilia A.
“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,” Jiang said.
An ASC Therapeutics gene therapy delivering a functional FVIII gene into a group of liver cells in a living mouse model of hemophilia restored the levels of the clotting factor. Further tests in the rhesus macaque (a monkey) and human liver cells also led to a good response without evidence of problems, supporting the therapy’s potential for humans.
ASC Therapeutics is in discussions with the U.S. Food and Drug Administration (FDA) to file for an investigational new drug application (IND) to test ASC-618 for hemophilia A. The company is conducting toxicology studies to support its IND.
“We are very excited to partner with ASC to bring a gene therapy treatment to patients with hemophilia A. 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,” said Mohan Rao, PhD, CEO of Expression Therapeutics.
ASC Therapeutics is a division of Applied StemCell, both based in California.