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Homology Medicines to Participate in the World Orphan Drug Congress

- Presentations to Highlight Advanced Gene Editing Technologies and Innovative Approaches to Enhance Orphan Drug Development for Patients -

BEDFORD, Mass., April 23, 2018 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that Company management is scheduled to participate in the World Orphan Drug Congress USA 2018 at the Gaylord National Harbor Hotel in Oxon Hill, Maryland. The World Orphan Drug Congress is a global gathering of leaders in orphan drugs and includes presentations covering all aspects of orphan drug development and rare disease research.

  • Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology, will present, “Next generation in vivo and ex vivo gene editing using homologous recombination to treat rare diseases,” on April 25, 2018 at 11:20 a.m. ET.
  • Dr. Tzianabos will also participate in the keynote panel titled, “How rare disease companies are ramping up innovation to boost orphan drug development and patient centricity,” on April 26, 2018 at 5:20 p.m. ET.
  • Michael Blum, Vice President of Commercial Strategy at Homology, will chair the keynote address on gene editing on April 27, 2018 at 8:30 a.m. ET.

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 statements regarding upcoming presentations 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; 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 international marketing approval; failure to obtain U.S. 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 prospectus filed in connection with our initial public offering 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.

Investor Contact:
Theresa McNeely
SVP, Corporate Communications and Patient Advocacy

Media Contact:
Cara Mayfield
Senior Director, Patient Advocacy and Corporate Communications