The French biotech GenSight Biologics has received regulatory authorization in the UK to launch the PIONEER Phase 1/2 clinical trial for its GS030 system — a light-sensing gene therapy (optogenetics) coupled with eyewear, which enhances visual stimulation. The system is designed to restore vision for people who are blind from retinitis pigmentosa (RP) and potentially other retinal conditions such as: Usher syndrome, Stargardt disease, and dry age-related macular degeneration.
The GS030 will be evaluated in 18 people with RP who can see no better than counting fingers. Additional trial details are available at the clinical trials Web site hosted by the National Institutes of Health.
The Foundation Fighting Blindness funded lab research for the optogenetic component of the system.
“Moving GS030 into phase 1/2 is a very important step for us,” says Bernard Gilly, Ph.D., GenSight co-founder and chief executive officer. “The trial will recruit patients with very low vision. We therefore believe we will be able to rapidly understand the potential of the technology to restore useful vision in these patients.”
People with retinal diseases such as RP lose vision due to degeneration of light-sensing photoreceptors, the retinal cells that make vision possible. People in the GS030 clinical trial will receive an optogenetic therapy to bestow light sensitivity to ganglion cells — retinal cells which are not normally light sensitive, but often survive after photoreceptors are lost. The GS030 eyewear will transmit an amplified light image signal to further stimulate ganglion cells and enhance the user’s visual experience. The GS030 system works independent of the gene mutation causing RP.
“GenSight’s technology is a very exciting development for people who have lost most or all of their vision,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “While Allergan is evaluating optogenetics alone for RP in a clinical trial, GenSight’s optogenetic therapy, combined with an eyewear component, provides the potential for a better visual experience.”
Both Allergan’s and GenSight’s optogenetic therapies involve injection into the vitreous (gel in middle of the eye) of gene copies that express light-sensitive proteins. GenSight’s eyewear is designed to help users see a broader light spectrum.
Optogenetics is a quickly advancing therapeutic approach being developed in research labs around the world, primarily for brain and neurological conditions.