GenSight Biologics and the 15-20 National Hospital (l’Hôpital national des 15-20) announced the publication of the results of the GS030 PIONEER trial in The New England Journal of Medicine (NEJM). The article reports the trial’s primary safety findings and exploratory visual function assessments evaluating GS030, GenSight Biologics’ candidate optogenetic treatment for late-stage retinitis pigmentosa. GS030 uses an intravitreal injection of an AAV-based gene therapy that encodes light-sensitive proteins in the intact retinal ganglion cells of 1 eye, coupled with light-stimulating goggles to activate the light-sensitive proteins.1
Published a few days after the awarding of the 2026 Nobel Prize for Physiology or Medicine to Karl Deisseroth, MD, PhD, Peter Hegemann, PhD, and Georg Nagel, PhD, for their discoveries concerning light-gated ion channels and optogenetics, the NEJM article illustrates the therapeutic potential of the scientific approach recognized by the prize, GenSight Biologics and the 15-20 National Hospital said in the joint press release.
According to the press release, most ocular adverse events were mild or moderate, with the single event classified as severe resolving within minutes after instillation of apraclonidine. There was no systemic adverse event determined to be related to the study drug or to the intravitreal injection.
The study was not designed to establish the efficacy of GS030, but the authors highlight several results. In 6 of the 10 participants, exploratory assessments showed a clinically meaningful improvement in light sensitivity, as assessed with full-field stimulus threshold testing. Four of the 8 participants who completed visual behavioral testing showed positive changes in the ability to perform certain tasks while using the light-stimulating goggles: detecting, localizing or touching objects, or determining the orientation of bars.
In addition, exploratory high-density EEG recordings in a subset of participants showed changes consistent with the transmission of optogenetically evoked visual signals to the visual cortex, supporting the interpretation that the observed behavioral responses reflected changes in visual processing rather than being an artifact, GenSight Biologics and the 15-20 National Hospital said. RP
1. GS030 has not received marketing authorization in any jurisdiction.