Homology Medicines is focused on advancing our genetic medicines platform as one-time treatments and potential cures for patients with rare diseases

Homology Medicines is focused on advancing our genetic medicines platform as one-time treatments and potential cures for patients with rare diseases

Release Details

Homology Medicines Regains Worldwide Rights to Ophthalmology Program Based on its In Vivo Nuclease-Free Gene Editing Platform

March 1, 2021

- Company plans to continue advancing ophthalmic program toward a development candidate for lead indication -

BEDFORD, Mass., March 01, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today it has regained worldwide exclusive rights from Novartis to research, develop, manufacture and commercialize Homology’s proprietary nuclease-free gene editing technology platform for an ophthalmic target. Following a portfolio review, Novartis decided to conclude the collaboration and licensing agreement with Homology to pursue other opportunities in their pipeline.

“Our nuclease-free gene editing platform is applicable across a broad range of genetic disorders, including ophthalmic diseases where we have generated positive in vivo data in two different eye targets,” said Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “Our work with Novartis has been productive in demonstrating that our AAVHSC vectors can edit the back of the eye, and we plan to share additional data from the ophthalmology program at a scientific meeting in May. The data are promising and support advancing this program, which we now intend to do on our own as we drive toward naming a development candidate.”

“Our collaboration with Homology has generated data that support gene editing in retinal cells in a rare ophthalmic disease, providing early proof-of-principle for further research using this approach.” said Jay Bradner, M.D., President of the Novartis Institutes for BioMedical Research (NIBR). “While we have made the decision to reallocate our resources to support other programs, we look forward to tracking Homology’s continued progress on this technology.”

The companies are co-authors on an upcoming scientific abstract that highlights the results of studies which showed that human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) transduced relevant cell types after sub-retinal injection, supporting a nuclease-independent approach to editing across two targets. In addition, the data showed AAVHSCs crossed the blood-brain and blood-retinal barriers in non-human primates following I.V. administration.

In 2017, Homology granted Novartis worldwide exclusive rights to the Company’s proprietary AAVHSCs using its nuclease-free gene editing platform for certain ophthalmic targets, which are now fully owned by Homology.

About Homology Medicines, Inc. 
Homology Medicines, Inc. is a clinical-stage 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 family 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 additional data from, and our intention to advance, the ophthalmology program; our beliefs regarding our manufacturing capabilities; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. 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 impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; 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 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 quarterly period ended September 30, 2020 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.

Company Contacts
Theresa McNeely
Chief Communications Officer
and Patient Advocate

Media Contact:
Marisa Citrano 
Corporate Communications Associate

Primary Logo

Source: Homology Medicines, Inc.