Frontera Therapeutics, Inc., a Sino-US biopharmaceutical company, has announced that it has received approval from the US Food and Drug Administration (FDA) to proceed with a Phase II clinical study for its investigational gene therapy, FT-002. This recombinant adeno-associated virus (rAAV) gene therapy candidate marks a significant milestone as the first Chinese rAAV gene therapy to advance to clinical trials in the US.
FT-002 is an innovative ophthalmic gene therapy designed using a recombinant adeno-associated virus serotype 5 (rAAV5) vector and an optimized Sf9 baculovirus expression vector system (Sf9 BEVS). The therapy includes a codon-optimized coding gene for the GTPase regulator of retinal pigment degeneration (hRPGRORF15) and is specifically intended for the treatment of X-linked retinal pigment degeneration (XLRP), a condition caused by mutations in the RPGR gene. XLRP is a progressive and severe form of hereditary retinal disease that leads to vision loss and currently lacks effective therapies globally.- Flcube.com
