22 February 2018 - - Paris, France-based clinical-stage biopharma company GenSight Biologics (Paris, Euronext: SIGHT) has published detailed results from the Phase I/II clinical trial and long-term follow-up of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients in Ophthalmology, the journal of the American Academy of Ophthalmology, the company said.
The study demonstrated that GS010 (rAAV2/2-ND4) is safe and well tolerated 2 years after a single unilateral intravitreal administration.
The study was an open-label single-center Phase I/II clinical trial that included 4 dose-escalation cohorts and an extension cohort.
At week 96, there were no unexpected TEAEs, no serious adverse events related to the treatment or procedure, and no suspected unexpected serious adverse reactions. No deaths and no TEAEs leading to study discontinuation were reported.
GenSight Biologics is currently conducting two Phase III clinical studies (RESCUE and REVERSE) in Europe and the United States to assess the efficacy of GS010 in subjects affected with LHON due to the ND4 mutation, with vision loss up to one year at the time of treatment.
LHON is a rare maternally inherited mitochondrial genetic disease characterized by the degeneration of retinal ganglion cells that results in vision loss that can lead to legal blindness, and mainly affects adolescents and young adults.
GenSight Biologics is focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Its pipeline leverages two core technology platforms, the mitochondrial targeting sequence and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases.