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Homology Medicines Presents Data on Platform’s Ability to Target the Central and Peripheral Nervous System and Therapeutic Potential in the Rare Disease Metachromatic Leukodystrophy

- Homology to Nominate a Clinical Development Candidate for MLD This Quarter and to Begin IND-Enabling Studies in 2019 -

BEDFORD, Mass., November 7, 2018 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today data demonstrating that Homology’s proprietary vectors are able to cross the blood-brain-barrier and have a positive impact on the rare neurological disorder metachromatic leukodystrophy (MLD).

The presentation at the Society for Neuroscience (SfN) 2018 Annual Meeting reported biodistribution data on three of Homology’s 15 human-derived adeno-associated virus vectors (AAVHSCs). Following a single intravenous (IV) injection in a large species, the AAVHSCs demonstrated transduction of a variety of peripheral and central nervous system tissues and cell types. Additionally, in the preclinical MLD model, a single IV injection of a Homology vector led to 30-115% of normal human ARSA enzyme activity levels, exceeding the established therapeutic target of 10-15%. Homology plans to nominate an MLD development candidate this quarter and to begin IND-enabling studies in 2019.

“The key requirement for developing single IV gene therapies for neurological disorders is the ability for the therapeutic to cross the blood-brain-barrier, and we are pleased to report that our vectors effectively reach the central and peripheral nervous system following systemic administration,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “We are striving to provide a one-time treatment option that may lead to a cure for patients, and these preclinical data are informing our plans to nominate a development candidate by the end of the year.”

For more information about this presentation, visit Homology’s website at

About Metachromatic Leukodystrophy (MLD)
MLD is a rare lysosomal storage disorder caused by a mutation in the ASA gene. ASA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. In MLD, these cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within 5-10 years after onset. 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 anticipated timing for nominating an MLD development candidate and initiating IND-enabling studies; advancing our novel gene therapy and gene editing technology platform and pipeline; our beliefs regarding our manufacturing capabilities; our goal of improving the lives of patients with rare genetic diseases; the anticipated timing of the release of clinical data; 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 build out 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 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 Quarterly Report on Form 10-Q for the quarter ended June 30, 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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