City of Hope licenses its unique gene editing technology to Homology Medicines Inc.

May 2, 2016

City of Hope
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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
The adeno associated virus (AAV)-based gene editing technology is being developed to help find cures for rare genetic diseases

DUARTE, Calif. — 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  

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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