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Homology Medicines Announces Appointment of Gabriel M. Cohn, M.D., as Chief Medical Officer

- Experienced Clinical Geneticist with Track Record in Rare Disease Drug Development Joins Homology’s Senior Management Team -

BEDFORD, Mass. – December 3, 2019 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that it has appointed Gabriel M. Cohn, M.D., M.B.A., as Chief Medical Officer. Dr. Cohn will join Homology’s senior management team to direct all clinical development and operations related to Homology’s dual gene therapy and gene editing technology platform. Dr. Cohn’s efforts will focus primarily on advancing the Phase 1/2 pheNIX gene therapy, randomized, concurrently controlled, dose-escalation trial for adults with phenylketonuria (PKU), which is expected to report initial clinical data this year, as well as progress a growing clinical development pipeline.

“Gabe’s expertise in the clinical development of rare disease treatments, including gene therapies and enzyme replacement therapies, comes to Homology at a time when we are advancing our gene therapy trial for adults with PKU,” stated Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “With Gabe on board, we look forward to sharing the initial data from our pheNIX Phase 1/2 trial this month, and to leveraging his experience across multiple genetic disease areas to execute on our programs and expand our rare disease portfolio of potential new treatments and cures for patients in need.”

Dr. Cohn brings to Homology a unique blend of more than twenty years of clinical development experience focused primarily in rare genetic diseases, as well as an academic career focused on reproductive and cancer genetics. Prior to Homology, Dr. Cohn was Vice President, Clinical Development Lead at AVROBIO, Inc., where he led the clinical development for the Gaucher disease and Pompe disease gene therapy programs, providing medical leadership for protocol design, regulatory filings and FDA interactions, global clinical trial site identification and initiation, and investigator interactions. His additional genetic disease experience included positions of increasing responsibility at Shire Plc, including Shire Human Genetic Therapies (HGT), where he most recently held the position of Senior Medical Director, Clinical Sciences, providing strategic medical, scientific and technical expertise and leadership for potential business development opportunities. Before that, he was the interim Global Franchise Medical Lead, Gaucher Disease, focused on developing the franchise strategy, and earlier on served as Medical Director, North America, driving the medical affairs strategy and support for the transition of VPRIV, an enzyme replacement therapy for Gaucher disease type 1, from clinical development through to U.S. launch. Prior to joining industry, Dr. Cohn served as the Chief, Division of Clinical and Reproductive Genetics and Medical Director, Genetic Services at Baystate Medical Center and as Assistant Professor at Tufts University School of Medicine where he won numerous teaching awards. He began his academic career as the Director, Reproductive Genetics at SUNY Health Science Center (HSC) at Syracuse (now Upstate). Dr. Cohn is a Fellow of the American College of Medical Genetics (FACMG), a Fellow of the American Congress of Obstetricians and Gynecologists (FACOG), and author on over 40 peer-reviewed publications, book chapters and review articles, and over 70 published abstracts and presentations. He maintains his medical license in Massachusetts. Dr. Cohn has a Bachelor of Science in Biology from Brooklyn College, a Doctorate of Medicine from SUNY HSC at Syracuse School of Medicine and an M.B.A. from University of Massachusetts Amherst. He completed a residency in Obstetrics & Gynecology at SUNY HSC at Syracuse and a fellowship in Medical Genetics at the National Institutes of Health.

“I have spent my career focused on helping patients, and I am excited to work with the Homology team, many of whom I have worked with before to develop treatments that make a difference in the lives of patients and their families,” stated Dr. Cohn. “I am a strong believer in the potential of Homology’s dual gene therapy and gene editing platform to change the course of rare genetic disease by advancing new treatments and ultimately cures, and I look forward to progressing our clinical pipeline to address the unmet needs of patients.”

About Homology Medicines, Inc.
Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of clinical data from the Phase 1/2 pheNIX trial; our ability to deliver potential cures to patients and change the course of rare genetic disease; and beliefs about preclinical data and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

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