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

May 16, 2019 Off By BusinessWire

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 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 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
for tissue-directed codon optimization and ancestral sequence
reconstruction-based bioengineering of coagulation factors. To learn
more about Expression Therapeutics, please visit www.expressiontherapeutics.com.