SparingVision Receives Authorization to Launch US Clinical Trial for its Cone-Preserving Treatment
Research News
The emerging therapy is designed to work independent of the mutated gene causing retinitis pigmentosa
SparingVision, a French company developing therapies for ocular conditions including inherited retinal diseases, has received authorization from the US Food & Drug Administration (FDA) to launch a Phase 1/2 clinical trial known as PRODYGY for SPVN06, its gene-independent, cone-preserving therapy for people with retinitis pigmentosa (RP). The company is also seeking authorization to conduct the trial in France. The US trial will take place at the University of Pittsburgh Medical Center.
SparingVision plans to enroll a total of 33 RP patients who have disease-causing mutations in PDE6A, PDE6B, or RHO.
SparingVision is funded through the Foundation’s RD Fund, a venture philanthropy fund for emerging therapies that are in or nearing early-stage clinical trials. The Foundation also provided several years of research grant funding for the preclinical development of SPVN06.
“We at the Foundation are very excited for this promising gene-agnostic treatment to move into human studies. Gene-independent approaches are a priority for us because they have the potential to help the most people, ” says Claire Gelfman, PhD, chief scientific officer at the Foundation.“
SPVN06 expresses a protein called 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, the cells in the retina that provide central and color vision and enable people to read, drive, and recognize faces. RdCVF is naturally secreted by rods, the retinal cells that provide night and peripheral vision.
RP initially affects rods. The progressive loss of rods leads to loss of cones. There are currently no therapies for RP.
RP affects more than two million people worldwide. The retinal disease is usually diagnosed in childhood, progressively leading to legal or total blindness in adulthood.