SparingVision to Acquire Therapy for Resurrecting Dormant Cones for Vision Restoration

SparingVision, a genomic medicines company targeting ocular diseases, has entered into an agreement to acquire GAMUT Therapeutics, a company developing a gene therapy to bestow light-sensitivity to cone photoreceptors that are in a non-functional, dormant state but haven’t degenerated in people with late-stage retinitis pigmentosa (RP) and related conditions. The company hopes to move the treatment, known as SPVN20, into a clinical trial in 2024.

Deniz Dalkara, PhD, GAMUT’s scientific founder, was previously funded by the Foundation for lab research to develop the cone-resurrecting approach that has become SPVN20. She will continue working on the project with the SparingVision team.

The Foundation’s RD Fund, a venture philanthropy fund for moving promising therapies into and through early-stage clinical trials, is an investor in SparingVision.

SparingVision says that the best candidates for SPVN20 are people who have lost all their rods and many of their cones but still have dormant cones. The emerging treatment is designed to work regardless of the mutated gene causing the retinal disease. Delivered by a human-engineered adeno-associated virus (AAV), SPVN20 leads to the production of a protein, which activates phototransduction (i.e., light sensitivity) in cones to generate the electrical signals that the brain uses to create images. Cones provide visual acuity, vision in lighted settings, and color and central vision.

SparingVision is also developing a cone-preserving gene therapy known as SPVN06 that leads to the production of rod-derived cone-viability factor (RdCVF), a naturally occurring protein in the retina identified by SparingVision co-founders José Sahel, MD, and Thierry Léveillard, PhD, at the Institut de la Vision. The scientists demonstrated in laboratory studies that RdCVF prevented or slowed the degeneration of cones. SPVN06 is also designed to work regardless of the mutated gene causing the retinal disease. A clinical trial is planned for SPVN06 in late 2021.

The company is considering the possibility of developing a combined SPVN20 and SPVN06 therapy in the future.

“We are very excited about the addition of SPVN20 to SparingVision’s portfolio because it shows promise for restoring vision in people with late-stage disease, regardless of their mutated gene,” says Ben Yerxa, PhD, chief executive officer, Foundation Fighting Blindness. “Furthermore, SPVN20’s potential for cone resurrection complements well the cone-preserving properties of SPVN06. While we first need clinical success for these projects individually, the combination of the two is certainly intriguing.”