Applied StemCell Therapeutics Cures Hemophilia A in Mice

Applied StemCell, Inc. (ASC), a leading gene-editing company, announced that its therapeutic division (ASC Therapeutics) has cured Hemophilia A in mice and will be presenting their in vivo and in vitro findings at the 22nd Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) held April 29 “ May 2 in Washington D.C.

ASCs co-founder Ruby Chen-Tsai, Ph.D., will present both as an invited speaker and as a scientific poster on the in vivo proof-of-concept study results for their Hemophilia A mice model based on the integration of FVIII transgene in the Alb locus using CRISPR/Cas9 and non-homologous end joining DNA repair technologies.

In addition to the oral presentation, two additional scientific posters will be presented alongside to support the in vitro study results:

1. Hemophilia A Cured in Mice by CRISPR-based In Vivo Genome Editing of Human Factor FVIII (scientific poster & invited oral presentation): proof-of-concept results in Hemophilia A mice.

2. Site-Specific Genome Editing by CRISPR/Cas9 for Hemophilia A in Human and Non-Human Cells (scientific poster): in vitro studies in human and non-human primate (NHP) cells, validating the feasibility of our approach in multiple species and the preliminary off-target analysis.

3. Adeno-associated Virus Serotypes Screening in Non-human Primates for Hemophilia A Genome Treatment (scientific poster): AAV serotypes screening in NHPs for Hemophilia A gene therapy.

The American Society of Gene and Cell Therapy’s (ASGCT) is the worlds largest gathering of gene and cell therapy professionals, with over 3500 attendees from various biotechnology sectors. It provides an international forum where the latest scientific discoveries are presented and discussed, while offering an inspiring platform for networking and advancing gene and cell therapy pipelines.

Ruhong Jiang Ph.D., CEO & founder of ASC, stated: We are very proud of the achievements and rapid progress our therapeutics team has made thus far. We have learned a great deal from these initial studies, which we will leverage as we move forward into the next phase.

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 companys therapeutic development pipeline includes several preclinical stage projects focusing on monogenic blood disorders that exhibit a high genetic penetrance.

Applied StemCell, Inc. is a fast-growing biotechnology company with more than 10 years 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, visit us at

Applied StemCell, Inc.
Gianluca Roma, 650-741-8053
Business Development
[email protected]