GenSight Biologics is a biopharmaceutical company that focuses on creating gene therapies for retinal neurodegenerative and central nervous system disorders. They have announced that the FDA has awarded Fast Track Designation of GS030, which uses AAV2 gene therapy combined with optogenetics to treat retinitis pigmentosa. Fast Track makes it easier and quicker to develop and review drugs that treat serious conditions with unmet need. GS030 is a gene therapy where a gene that makes a light-sensitive protein is introduced directly into the patient’s retina via an injection. This makes the retina light responsive without the use of photoreceptors that may have been damaged by disease, such as RP. After injection, patients wear glasses that encode the visual scene and send this information to the treated retina. GS030 is not gene mutation-specific and can thus be used for different retinal disorders. A multicenter clinical trial is being conducted to look at the safety and tolerability of GS030 in late-stage RP patients in the United Kingdom, France, and the United States. Results should be available in 2023.
What this means for Usher syndrome: If these clinical trials are proven successful, the Fast Track designation speeds the time required for the therapy to be reviewed and approved for patient use. Usher patients may be able to receive these beneficial gene therapies sooner.