NEJM publishes Phase 1/2 GS030 results: 6 of 10 patients gain meaningful light sensitivity

GenSight Biologics/GS030 optogenetic therapy

The PIONEER trial of GS030 in late-stage retinitis pigmentosa was published in NEJM as "Optogenetic Therapy for Restoring Aspects of Visual Function". The trial was built to test safety; exploratory results showed measurable visual responses in some patients, and no systemic side effect was linked to the drug or injection.

• 6 of 10 participants had a clinically meaningful gain in light sensitivity
• 4 of 8 who did behavioural tests improved at detecting, locating or touching objects
• High-density EEG in a subset showed signals consistent with input reaching the visual cortex
• One severe ocular event resolved within minutes after eye drops