Homology Medicines, 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.”
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.