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Homology Medicines and Novartis Enter Collaboration to Develop New Treatments Using Homology’s Proprietary Gene Editing Technology

BEDFORD, Mass., November 13, 2017 – Homology Medicines, Inc., a genetic medicines company, announced today that it entered into a research and development collaboration with Novartis to use Homology’s proprietary gene editing technology to develop new treatments for select ophthalmic targets and a hemoglobinopathy disease.

Under the terms of the agreement, Novartis made an upfront payment to and an equity investment in Homology. In addition, Novartis is providing funding to advance the programs and to explore new opportunities for Homology’s technology platform. Homology is also eligible to receive milestone payments from Novartis, plus royalties from the sale of products commercialized under the collaboration.

“This collaboration leverages Homology’s differentiated gene editing technology and Novartis’ ability to bring innovative therapies to patients,” said Arthur Tzianabos, Ph.D., Chief Executive Officer of Homology Medicines. “We look forward to working with Novartis to expand our pipeline and rapidly translate our platform into new and potentially curative treatments for patients.”

For Novartis’ feature story on the collaboration with Homology, visit

Under the collaboration, Novartis will gain worldwide exclusive rights to Homology’s proprietary technology platform for select ophthalmic targets and a hemoglobinopathy disease. Homology will retain U.S. commercial rights and share U.S. profits with Novartis for in vivo applications related to the hemoglobinopathy program, a strategic area of focus for Homology. Homology’s broadly applicable technology enables highly efficient homologous recombination-based in vivo gene editing. Using Homology’s proprietary adeno-associated virus vectors derived from human hematopoietic stem cells (AAVHSCs), the technology deploys a single component system to mediate gene editing. This approach has demonstrated highly efficient and precise on-target gene editing capabilities in multiple disease models and does not require exogenous nucleases or promoters. Outside of this collaboration, Homology is currently in IND-enabling studies for an inborn error of metabolism disease. Homology also has ongoing discovery efforts targeting several rare diseases using its AAVHSC gene correction platform, which is capable of both gene editing and gene therapy.

About Homology Medicines, Inc.
Homology Medicines is a genetic medicines company translating proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases. The combination of a new multidimensional gene correction platform and a management team that has successfully developed and commercialized rare disease therapies positions the Company to move beyond the current limitations of gene therapy and gene editing approaches to improve patient care. Homology Medicines has built foundational intellectual property on gene editing and gene therapy using vectors derived from naturally occurring human adeno-associated viruses (AAVs). The Company’s technology is precise, on-target and highly efficient for in vivo editing of genetic mutations. The team and technology create a significant opportunity for Homology Medicines to rapidly advance a diverse pipeline of new medicines that address and potentially cure the underlying cause of genetic diseases. For more information, please visit

Theresa McNeely
SVP, Corporate Communications & Patient Advocacy

Cara Mayfield Director
Corporate Communications
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