5/16/19 MILPITAS, Calif.–(BUSINESS WIRE) — 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 10 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 www.asctherapeutics.com.
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 to improve microfluidic transduction efficiency and tissue-directed codon optimization. To learn more about Expression Therapeutics, please visit www.expressiontherapeutics.com.
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.