Hongkong, China, Decemeber 8th, 2023 – Preliminary clinical study results of FT-002, an AAV gene therapy treatment of Frontera Therapeutics, a potential first in class therapy for the treatment of XLRP, were reported at the 16th Asia-Pacific Vitreo-Retina Society, APVRS, 2023).
Professor Sui Ruifang from Peking Union Medical College Hospital was invited to participate in the Symposium on Retinal Medicine and gave a presentation on the topic “Preliminary Results of an AAV Gene Therapy Trial on X-linked Retinitis Pigmentosa Caused by Mutations in Retinitis Pigmentosa GTPase Regulator”.
Professor Sui Ruifang introduced the AAV therapeutic details, mechanism of action and preclinical study results of FT-002, with an emphasis on sharing the preliminary safety and efficacy results of the IIT study. The clinical study results showed that no dose-limiting toxicity was observed in XLRP patients after receiving low, medium and high doses of FT-002, and the safety and tolerability were good. In the medium and high dose groups the visual function indexes such as full-field photosensitivity threshold and micro-field were improved to some extent.
XLRP is a sex-linked genetic retinopathy caused by mutations in the RPGR gene, with a prevalence of 3.4 to 4.4 per 100,000 males in Europe and the United States with about 20,000 cases, and about 30,000 to 50,000 patients in China. Currently, effective therapeutic drugs and treatments are lacking. FT-002 Injection is the first AAV gene therapy drug approved by the CDE for clinical trials conducted in humans in China.
About FT-002
FT-002 injection is intended to treat patients with X-linked Retinitis Pigmentosa (XLRP) caused by RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutations. FT-002 Injection is a recombinant adeno-associated virus (rAAV) gene therapy drug. After intraocular injection, retinal cells express active RPGR protein, resulting in photoreceptor ciliary transport function, rescue of photoreceptor cell loss due to RPGR gene mutation, and improved visual function of patients or delay of the further progression of visual impairment. It is currently being tested in a multicenter Phase I/II clinical study nationwide.