Homology Medicines Presents Data Demonstrating Suite of Novel AAV Vectors Targeted Key Cell Types for Inherited Retinal Diseases

- Presentation Highlights Targeted Biodistribution in Multiple Models following Different Routes of Administration -

BEDFORD, Mass., April 30, 2019 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of preclinical data demonstrating that, with a single dose, the Company’s suite of novel adeno-associated virus vectors targeted cell types frequently associated with inherited retinal diseases. The presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2019 Annual Meeting showed that Homology’s vectors, which are derived from human hematopoietic stem cells (AAVHSCs), were able to target and enter (transduce) therapeutically relevant cells in the eye in three models using multiple routes of administration, including intravenous, intravitreal and subretinal injections.

“Our suite of AAVHSCs enables us to select the best vector for addressing a particular disease, and today’s data further demonstrates the potential flexibility of our vectors to target the eye when employing multiple routes of administration and across different models,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our presentation also demonstrates that AAVHSCs were able to deliver genes to cells that are therapeutically relevant for many ophthalmic diseases, thus expanding the potential of our platform to new indications over time.”

In the biodistribution studies, which included a total of eight different AAVHSCs, data demonstrated:

  • In non-human primates, retinal cells were transduced following intravenous injections;
  • In the porcine model, photoreceptors and retinal pigment epithelium (RPE) cells were transduced following subretinal injections;
  • In the murine model, photoreceptors, RPE cells and retinal ganglion cells (RGCs) were transduced following intravitreal injections; and
  • In the murine model, photoreceptors, RPE cells, horizontal cells and RGCs were transduced following subretinal injections.

In November 2017, Homology entered into a collaboration with Novartis to develop new genetic medicines using Homology’s homologous recombination-based gene correction approach for select ophthalmic targets.

For more information about the presentation, visit Homology’s website at www.homologymedicines.com/publications.

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 www.homologymedicines.com.

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 statements regarding the potential for expansion of our platform to new indications; advancing our novel gene therapy and gene editing technology platform and pipeline; our goal of improving the lives of patients with rare genetic diseases; 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: the fact that 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 marketable products; the early stage of our development efforts; our failure or the failure of our collaborators to successfully develop and commercialize drug candidates; 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; the inability to obtain orphan drug exclusivity; 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; the price of our common stock may be volatile; significant costs as a result of operating as a public company; and any securities class action litigation. These and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2018 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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