GenSight Biologics S.A. announced this week that the first patient has enrolled in the company’s two parallel Phase 3 trials for GS010, a medicine designed to treat Leber’s Hereditary Optic Neuropathy (LHON).
The names of the trials are RESCUE and REVERSE. GenSight is a biotechnological company that discovers and develops novel gene therapies for the treatment of neurodegenerative retinal diseases.
The two Phase 3 trials are pivotal in the further development of GS010 and are intended to discover if the drug can stop or even reverse vision loss that is related to LHON. Vision loss is caused by the NADH dehydrogenase 4 (ND4) mutation. The trials will also determine whether or not GS010 can act as a prophylaxis for the loss of vision in eyes that have not yet been affected. Also determined by the trials will be the window of opportunity for therapeutic treatment of vision loss after the onset of LHON.
Beginning treatment for vision loss in LHON at an early stage is a critical part of how successful the therapy will be. The two trials will treat patients whose eyesight has begun to deteriorate for up to a year. GS010 will be injected into the patient’s eye.