GenSight Biologics (EPA:SIGHT) said today that the first person was treated in a first-in-man trial evaluating GenSight’s gene therapy candidate, GS030-DP, in combination with a wearable optronic visual stimulation device, GS030-MD.
Scientists are evaluating the drug-device combination in 18 people with retinitis pigmentosa.
The Pioneer Phase I/II trial is an open-label, dose-escalation study designed to assess the safety and tolerability of GS030. The company’s drug-device therapy is based on GenSight’s optogenetics platform, which introduces a gene encoding for a light-sensitive protein into the retinal ganglion cells via an intravitreal injection. GenSight’s wearable device then stimulates the cells, ideally restoring vision to the user.
“We are thrilled to see our second lead program, GS030, now entering the clinic. This is a fantastic achievement for the team, and a promising step forward for patients. Optogenetics has been studied for years in research laboratories, and it bears a great clinical promise in fighting vision impairment and other degenerative sensorial and neurological disorders,” co-founder & CEO Bernard Gilly said in prepared remarks. “GenSight continues to deliver on its strategy to develop novel approaches for treating and curing blinding diseases.”
The first three cohorts of the trial will include people affected by end-stage non-syndromic retinitis pigmentosa with no light perception or light perception level of visual acuity, according to GenSight. The extension cohort will also include participants with hand motion and counting fingers levels of visual acuity.
The trial’s primary outcome will the safety and tolerability of the therapy. Researchers plan to follow the study participants for one year after the injection.
GenSight is slated to finish enrolling participants by the fourth quarter of 2019 and expects topline data in the fourth quarter of 2020.