Homology Medicines Inc. Launches as a Newly-Formed Genetic Medicines Company Founded Upon a Unique City of Hope Gene Editing and Gene Transfer Platform Technology

DUARTE, Calif.–(BUSINESS WIRE)–City of Hope today announced that it has licensed its pioneering
AAV-based gene editing technology exclusively to Homology Medicines
Inc., a newly-formed genetic medicines company. A City of Hope lab led
by Saswati Chatterjee, Ph.D., developed the single platform technology
that enables both gene editing and gene therapy with a built-in in
vivo
delivery system. The foundational technology is a novel
non-nuclease based gene editing platform that is currently in
preclinical phase and is initially being developed to help find cures
for rare genetic diseases.

Chatterjee, a City of Hope professor of surgery and a member of the
institution’s Beckman Research Institute and the Gene Editing and Viral
Vector Core unit, together with her team, which includes Laura Smith,
Ph.D., a postdoctoral fellow in the AAV Lab and the Department of
Surgery, were the first to isolate and study a novel family of AAVs
derived from human hematopoietic stem cells. These nonpathogenic AAVs
are naturally present in the majority of healthy individuals and possess
unique gene editing and gene transfer properties, including unmatched
precision, efficiency and on-target in vivo editing of genetic
mutations.

“We are confident that the strong partnership between City of Hope and
Homology will pave the way in bringing novel genetic therapies to
patients with incurable diseases worldwide,” said Chatterjee, the
scientific founder of Homology Medicines and chair of the company’s
Scientific Advisory Board.

“It is rare to find cutting-edge science that takes a fundamentally new
approach placed in the hands of experts who have worked together as a
team before to translate new technologies into treatments targeting the
underlying cause of serious genetic diseases,” said Arthur Tzianabos,
Ph.D., CEO of Homology. “We are convinced that this single technology
platform that enables in vivo editing by efficient gene transfer
has broad, unmatched capabilities. With the strong support of our
investors and advisors, we will rapidly advance novel treatments that
have the potential to cure patients.”

Homology Medicines secured $43.5 million in a financing co-led by 5AM
Ventures and ARCH Venture Partners, and supported by Deerfield Capital
and Temasek Holdings. The company has recruited biotechnology industry
leaders with an extensive track record in rare genetic diseases and the
rapid translation of new technologies into treatments that improve the
lives of patients and their families. For more information about
Homology Medicines, please visit www.homologymedicines.com.

About City of Hope

City of Hope is an independent research and treatment center for cancer,
diabetes and other life-threatening diseases. Designated as one of only
45 comprehensive cancer centers, the highest recognition bestowed by the
National Cancer Institute, City of Hope is also a founding member of the
National Comprehensive Cancer Network, with research and treatment
protocols that advance care throughout the world. City of Hope is
located in Duarte, California, just northeast of Los Angeles, with community clinics
throughout Southern California. It is ranked as one of “America’s Best
Hospitals” in cancer by U.S. News & World Report.
Founded in 1913, City of Hope is a pioneer in the fields of bone marrow transplantation,
diabetes
and numerous
breakthrough cancer drugs
based on technology developed at the
institution.

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