Frontera Therapeutics announces CDE clearance to initiate clinical trial of a First-in-class gene therapy for the treatment of RPGR-associated X-linked Retinitis Pigmentosa

BEDFORD, Mass., and SHANGHAI, China, Nov 6th, 2023 Frontera Therapeutics (Suzhou) Co., Ltd (referred to as Frontera) announced the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has given clearance of the Investigational New Drug (IND) application to begin a clinical trial of an innovative gene therapy drug, FT-002, for the treatment of RPGR (Retinitis Pigmentosa GTPase Regulator) – associated X-linked Retinitis Pigmentosa (XLRP).

No drugs are available to treat XLRP worldwide. FT-002 is the first gene therapy for XLRP moving into clinical stage in China.

Regarding FT-002

FT-002 is a recombinant adeno-associated virus-based gene therapy targeting mutations of the RPGROFR15 gene in retinal photoreceptor cells. FT-002 is specially transduced to retinal cells and expresses active RPGR protein following intraocularly administration to rescue the loss of photoreceptor cells caused by the variants of RPGR gene, and improve the visual function or delay the progression of visual impairment.

“We are strongly encouraged by the CDE IND clearance of this First-In-Class drug. It is the 5th investigational gene therapy receiving IND approval in the 3 years since the company was founded. This achievement further validates the maturity and promise of Frontera’s AAV R&D platform, ” said Dr. Xinyan Li, co-founder and CEO of Frontera. “The preliminary results of the IIT study of FT-002 are impressive. The objective indicators including macular retinal thickness, visual field and visual acuity, have been  improved after the treatment of FT-002 especially that the significant improvements in best corrected visual acuity (BCVA) were observed in some patients at two months compared with baseline (ETDRS visual acuity chart). We look forward to rapidly advancing FT-002 into the clinic and bringing a new treatment for all XLRP patients.”