The results of the PIONEER clinical trial, evaluating an optogenetic gene therapy approach in patients with advanced retinitis pigmentosa, are published today in The New England Journal of Medicine (NEJM). Teams from the Hôpital national des 15-20 contributed to this international study.
This publication marks a new step in the development of optogenetics, an approach that aims to restore a form of visual perception by bypassing photoreceptor loss. The PIONEER trial evaluates GS030, developed by GenSight Biologics. This approach combines gene therapy administered by intravitreal injection with light stimulation glasses to activate the treated retinal cells.
The study, primarily focused on evaluating safety and tolerability, also reports several exploratory results concerning visual function. Improvements in light sensitivity and performance on visual tasks were observed in some participants. These results remain exploratory and will require confirmation through further research.
The teams from the Hôpital national des 15-20, particularly within the CRMR RefeRet coordinated by Professor Isabelle Audo, took part in the clinical evaluation of this approach, alongside several international expert centers. This participation demonstrates the hospital's commitment to developing and evaluating new therapeutic approaches for rare retinal diseases.
This publication is part of over twenty years of work dedicated to vision restoration through optogenetics, to which Professors José-Alain Sahel and Botond Roska have made major contributions, from fundamental research to clinical translation.
