May 6, 2024–Frontera Therapeutics is pleased to announce significant advancements in our clinical study of FT-002, presented at Retinal Cell and Gene Therapy Innovation Summitin the United States on May 3, 2024.
Professor Sui Ruifang, from Peking Union Medical College Hospital, was invited to present the clinical results, titled “Preliminary safety and efficacy of an open-label, dose-escalating genetic drug FT-002 in patients with x-linked retinal pigmentary degeneration (XLRP) associated with RPGR gene variants”.
Professor Sui Ruifang shared the preliminary safety and efficacy data of 18 subjects on behalf of the investigators. The patients demonstrated favorable safety profiles throughout the one-year follow-up period, with significant improvements observed in retinal sensitivity and visual function among specific dosage cohorts.
FT-002 is the first rAAV gene therapy cleared for clinical trial by the CDE and tested in XLRP patients, a disease lack of effective treatment options. The encouraging clinical safety and efficacy data from 18 patients demonstrate the therapeutic potential of FT-002. At present, FT-002 is undergoing a multicenter phase I/II clinical study nationwide in China, offering hope to a broader population of XLRP patients.
About FT-002
FT-002 Injection is a recombinant adeno-associated virus (rAAV) gene therapy drug designed to address unmet medical needs in patients with vision impairment. Administered via intraocular injection, FT-002 aims to facilitate the expression of active RPGR protein within retinal cells. This mechanism holds promise in mitigating photoreceptor cell loss attributed to RPGR gene mutations, thereby potentially enhancing visual function and delaying the progression of visual impairment in individuals with X-linked Retinitis Pigmentosa (XLRP) linked to RPGR gene mutations.