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

- 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 www.homologymedicines.com.