Frontera Therapeutics Doses First Patient in Phase 1 Clinical Trial for Gene Therapy FT-001 for the Treatment of Leber Congenital Amaurosis-2
BEDFORD, Mass., and SHANGHAI, China, January 6, 2023 — Frontera Therapeutics, a global clinical-stage biotechnology company that seeks to develop novel and best-in-class gene therapy medicines to improve the lives of patients across multiple disease areas, today announced that it has dosed the first patient in a Phase 1 clinical trial of its lead gene therapy program, FT-001, for the treatment of Leber Congenital Amaurosis-2, a severe inherited retinal disease related to a mutation in the RPE65 gene. FT-001 is administered by a one-time injection into the subretinal space of the eye that delivers a functional copy of the human RPE65 gene to the nuclei of the patient’s retinal cells.
2023-01-06