GenSight Biologics Moving Forward with Clinical Trials
Mohamed Genead, MD, chief medical officer of GenSight Biologics, updated OIS on the early-stage clinical progress of the company’s lead gene therapy product to treat Leber hereditary optic neuropathy (LHON). GenSight ran a Phase I/II trial in Paris on 15 patients, with each patient receiving a single intravitreal injection in the worse functioning eye. Patients in the study showed a favorable safety and tolerability profile. Over 78 weeks, patients less than two years from onset demonstrated sustained improvement in visual acuity. Dr. Genead said 60% to 80% of patients reported three lines of visual gain. Next, the company has enrolled two 74-patient Phase III trials called RESCUE and REVERSE. Results are expected next year.