Date: 2017-11-13
Type of information: Research agreement
Compound: gene editing technology
Company: Homology Medicines (USA - MA) Novartis (Switzerland)
Therapeutic area: Technology - Services - Ophtalmological diseases - Hematological diseases
Type agreement: R&D - research - development
Action mechanism:
- gene editing. 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.
Disease:
Details:
- • On November 13, 2017, Homology Medicines announced 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 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.
Financial terms:
- 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.
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Is general: Yes
