ASC Therapeutics Licenses Critical Technologies for Hemophilia A Gene Therapy from Expression Therapeutics

MILPITAS, CA, USA I May 16, 2019 I ASC Therapeutics, A Division of Applied StemCell, Inc. (ASC), announced today that it has obtained an exclusive therapeutics license from Expression Therapeutics, LLC (ET) for coagulation factor VIII expression technologies including a highly compact, yet potent, liver-directed promoter and matching liver-codon optimized factor VIII transgene for adeno-associated viral vector gene therapy of hemophilia A. The combination of these two technologies demonstrated exceptional potency in preclinical models including head-to-head comparisons with alternative promoter and transgene vector technologies designed to mimic those under clinical investigation.

ASC Therapeutics has successfully completed a pre-IND meeting with the U.S. FDA on the gene therapy based on this high-expression vector, ASC-618, for hemophilia A and is currently conducting toxicology studies prior to filing an IND.

Ruhong Jiang, Ph.D., CEO & Founder of ASC, stated: “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. 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.”

Mohan Rao, Ph.D., CEO of Expression Therapeutics, commented: “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.”

About ASC Therapeutics and Applied StemCell, Inc.

ASC Therapeutics is a division of Applied StemCell, which focuses on the development of curative therapeutic products that are enabled by its proprietary gene editing platform, TARGATT™ and other gene editing technologies. The company’s therapeutic development pipeline includes several preclinical stage projects. Applied StemCell, Inc. is a fast-growing biotechnology company with more than 11 years of experience in genome editing and stem cell technologies. Our mission is to advance genome editing technology innovation to develop more efficacious and safer gene and cell therapies. Applied StemCell has raised $63 million US dollars in support of their gene and cell therapy programs. To learn more about ASC Therapeutics, please visit

About Expression Therapeutics, LLC

Expression Therapeutics is a privately-owned biotechnology company founded in 2005 and based in Atlanta, Georgia. Our mission is to develop novel therapies that will dramatically improve the lives of people with hemophilia. Our pipeline includes lentiviral and adeno-associated viral vector gene therapy strategies for hemophilia A and B along with a high expression factor VIII protein for use as prophylactic therapy and in Immune Tolerance Induction. ET has also developed platform technologies for tissue-directed codon optimization and ancestral sequence reconstruction-based bioengineering of coagulation factors. To learn more about Expression Therapeutics, please visit

SOURCE: ASC Therapeutics

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