GenSight reports one-year safety and early efficacy signals from PIONEER trial of GS030

GenSight Biologics/GS030 optogenetic therapy

Nine patients in three dose cohorts showed GS030 was safe and well tolerated for at least a year, with only mild or moderate eye side effects. Some went from barely perceiving light to locating and counting objects, best at the highest dose, which was chosen for an extension cohort.

• Doses: 5e10, 1.5e11 and 5e11 vector genomes, one injection in the worse eye
• Intraocular inflammation in 70%, mostly mild and resolved with steroids
• Extension cohort adds patients with hand-motion or counting-fingers vision