Applied StemCell Therapeutics Cures Hemophilia A in Mice

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’s 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 world’s
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 company’s 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 www.asctherapeutics.com.

Contacts

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