Company is using adeno-associated virus (AAV) technology to develop treatments for eye and blood disorders.

Novartis made headlines in August, 2017 when it received approval from the US Food and Drug Administration for the first chimeric antigen receptor (CAR) T-cell therapy, Kymriah™ (tisagenlecleucel), produced using gene editing technology. Now the company is making further investments in gene editing techniques for the production of other types of diseases. In mid-November, 2017, Novartis announced that it is using a proprietary adeno-associated virus (AAV) gene-editing platform technology from Homology Medicines to develop novel treatments for ophthalmic and hemoglobinopathy diseases.

The two companies signed an agreement with an undisclosed value to collaborate on research and development of treatments for a blood disorder and various eye diseases. The agreement is, according to Novartis, intended to accelerate ongoing research at its Novartis Institutes for BioMedical Research (NIBR), which works with researchers around the world on technologies for the genetic engineering of cells.

As part of the deal, Novartis gains global, exclusive rights to Homology's proprietary technology platform for select ophthalmic targets and a hemoglobinopathy disease. In return, Homology received an upfront payment and an equity investment from Novartis, and has the potential to receive additional milestone and royalty payments for any products that are commercialized through the collaboration. Novartis will also fund the program.

The platform technology from Homology is designed to enable highly efficient homologous recombination-based in vivo gene editing, according to the company. Proprietary AAV vectors derived from human hematopoietic stem cells (AAVHSCs) are used to deploy a single-component system to mediate gene editing, leading to highly efficient and precise on-target gene-editing without the need for exogenous nucleases or promoters.

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

There are three parts to the collaboration. The first will focus on the development of a single AAV reagent that can be injected directly into the bloodstream of any patient with a defective gene to cure an unspecified blood disorder. We want to figure out if these AAVs are safe enough to inject directly into the bloodstream—and if we can use them to fix a defective gene once and for all,” commented Susan Stevenson, Ph.D., Executive Director at Novartis Institutes for BioMedical Research, leading the initiative.

The other two involve investigation of the use of Homogoy’s gene-editing technology for the development of drugs to treat eye diseases and testing of AAV’s prepared with the technology on as-yet-unnamed cell types and model systems.