4/25/19 MILPITAS, Calif.–(BUSINESS WIRE)– 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. ASC co-founder Ruby Tsai Ph.D., will present both as an invited speaker and 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 provide supporting in vitro study results. Below are the titles of the presentation and posters.
- 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.
- Site-Specific Genome Editing by CRISPR/cas9 for Hemophilia A in Human and Non-Human Cells (scientific poster) – in vitro studies in human and NHP cells, validating the feasibility of our approach in multiple species and preliminary off-target analysis.
- Adeno-associated Virus Serotypes Screening in Non-human Primates for Hemophilia A Genome Treatment (scientific poster) – AAV serotypes screening in NHPs for Hemophilia A GE treatment
The American Society of Gene and Cell Therapy’ s (ASGCT) is the world’s largest gathering of gene and cell therapy professionals and provides an international forum where the latest scientific discoveries are presented and discussed. 3,500+ potential customers, including basic and clinical investigators from academic, regulatory, and biotechnology sectors.
Ruhong Jiang, 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 of study.”.
About Applied StemCell
Applied StemCell, Inc. is a fast-growing biotechnology company and ASC Therapeutics division is focused on the development of a portfolio of curative therapeutic products that are enabled by its proprietary gene editing platforms, TARGATT™ and other gene editing technologies. The company’s therapeutic pipeline focuses on monogenic blood disorders that exhibit high genetic penetrance.