FT-002

FT-002 is an investigational gene therapy designed to treat X-linked retinitis pigmentosa (XLRP), a severe inherited retinal disorder caused primarily by mutations in the RPGR gene. FT-002 uses a recombinant adeno-associated virus (rAAV, specifically AAV5) as a vector to deliver a codon-optimized gene encoding a GTPase modulator (hRPGRORF15) directly to retinal cells. The therapy aims to restore or replace functional RPGR protein to prevent photoreceptor cell loss, thereby delaying or reversing vision loss characteristic of XLRP. FT-002 is administered via intraocular (subretinal) injection and is currently in Phase 1/2 and Phase 2 clinical trials, with promising early safety and efficacy data. Frontera Therapeutics manufactures FT-002 using a baculovirus expression vector system to ensure high yield and low impurity. The drug received Orphan Drug Designation from the U.S. FDA in January 2024[1][3][4][5][7][8].