Homology Medicines Emerges with Next Generation Gene Editing Technology Driven by Industry-leading Team in Rare Diseases

- Completes $43.5 Million Series A Financing and Appoints Leadership and Scientific Advisory Board -

May 02, 2016, 07:30 ET from Homology Medicines, Inc.

LEXINGTON, Mass., May 2, 2016 /PRNewswire/ -- Homology Medicines, Inc., a newly formed genetic medicines company translating proprietary, next generation gene editing and gene therapy technologies into novel treatments for patients with rare diseases, today announced the closing of a $43.5 million Series A preferred stock financing co-led by 5AM Ventures and ARCH Venture Partners. Additional investors included Temasek, Deerfield Management, and ARCH Overage Fund.  In conjunction with the financing, Homology hired biotechnology industry veterans who have a demonstrated track record in identifying and developing novel gene therapy and editing technologies, including Arthur Tzianabos, Ph.D., as Chief Executive Officer, Sam Rasty, Ph.D., as Chief Operating Officer, and Albert Seymour, Ph.D. as Chief Scientific Officer.  The Company also appointed an experienced Board of Directors and members of the 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 Dr. Tzianabos. "We are convinced that this single technology platform, which 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 was founded and incubated with a seed investment within the 4:59 Initiative, the company creation engine of 5AM Ventures.  Homology is based on groundbreaking science that harnesses the naturally occurring process of homologous recombination.  This non-nuclease-based approach offers clear advantages in its precision, efficiency and on-target in vivoediting of genetic mutations.  Homology obtained an exclusive worldwide license to this technology platform, which is based on the pioneering research of Saswati Chatterjee, Ph.D., Department of Surgery, member of the Beckman Research Institute at the City of Hope in California and co-founder and Chair of Homology's Scientific Advisory Board. Dr. Chatterjee is also a member of the Recombinant DNA Advisory Committee (RAC) to the Office of the Director, National Institutes of Health (NIH) and former charter member of the Therapeutic Approaches to Genetic Diseases Study Section of the NIH.  Dr. Chatterjee and her team led the first adeno-associated virus (AAV) vector-mediated gene transfer studies into human hematopoietic stem cells and subsequently identified and isolated a series of naturally-occurring AAVs from human CD34+ cells.

"Homology's ability to attract top management, who over many years have gained scientific and practical knowledge of the complexities of developing treatments for rare genetic diseases, is a testament to the strength of our technology platform," saidKush Parmar, M.D., Ph.D., Managing Partner at 5AM Ventures and Homology Board member.  "The Company has an aggressive development plan and a significant opportunity to leapfrog current strategies in gene therapy and editing as it builds an expansive and high-value clinical pipeline."

Commenting on the launch of Homology Medicines, Steven Gillis, Ph.D., Managing Director with ARCH Venture Partners and Homology Board member, noted, "As a scientist and investor, I have evaluated many breakthrough technologies in this field and I believe Homology's technology platform is unique in its ability to leverage naturally occurring genetic mechanisms to both treat and correct diseases in vivo.  I look forward to helping Homology lead the next generation gene editing technology that could result in new curative medicines for life-threatening diseases."  

Citation: 

          "Homology Medicines Emerges with Next Generation Gene Editing Technology Driven by Industry-leading Team in Rare Diseases."Homology Medicines Emerges with Next Generation Gene Editing Technology Driven by Industry-leading. Ed. Homology Medicine. Homology Medince Inc., 2 May 2016. Web. 03 May 2016. <http://www.prnewswire.com/news-releases/homology-medicines-emerges-with-next-generation-gene-editing-technology-driven-by-industry-leading-team-in-rare-diseases-300260438.html>.

Response:

I think its great that they have a new technology for rare diseases. I think this will finally be the answer to the diseases and illness that are just exist because the genes lines are broken up and need to be "unmutated". This is also a natural process, so its not something that will cause potentially more harm to the person. This is an amazing step in medical history and the science of curing diseases. If this breakthrough is for real, then rare diseases will be soon eradicated.