Boston, MA, September 23, 2024 – Frontera is excited to announce that its gene therapy product, FT-002, has been cleared by the U.S. Food and Drug Administration (FDA) for Phase II clinical trials in the U.S. This approval marks a major milestone, as FT-002 becomes the first recombinant adeno-associated virus (rAAV) gene therapy to receive FDA clearance for Phase II trials based on clinical data obtained from studies conducted in China.
“The FDA’s clearance for our Phase II clinical trials is a pivotal moment for Frontera,” said Dr. Xinyan Li, co-founder and CEO of Frontera. “It underscores our innovative approach, the strength of our CMC platform, and the robust preclinical and clinical data we’ve gathered. Our focus remains on developing safe, effective, and accessible rAAV gene therapies.”
About Frontera
Frontera is a gene therapy company focused on developing high-quality, affordable rAAV gene therapies for ophthalmic and cardiovascular diseases. The company’s leadership team brings deep expertise in drug development and clinical translation, supported by world-class scientific and clinical advisory committees.
About FT-002
FT-002 is an innovative gene therapy designed to treat X-linked retinitis pigmentosa (XLRP), a severe inherited retinal disorder. The therapy uses a recombinant adeno-associated virus to deliver a codon-optimized gene encoding a GTPase modulator (hRPGRORF15) to retinal cells. The drug is manufactured at Frontera’s GMP facility using an optimized Sf9 baculovirus expression vector system (Sf9 BEVS), a platform that enables high-yield, low-cost production with minimal impurities. Frontera’s innovative processes have reduced the drug substance’s empty vector content to below 1%, setting a new industry standard for quality.
FT-002 was granted Orphan Drug Designation (ODD) by the FDA in January 2024. At the Retina Cell & Gene Therapy Innovation Summit in May 2024, clinical data from 18 patients treated with FT-002, followed for up to a year, showed encouraging results: the treatment demonstrated good safety and tolerability across all patients, with certain dose groups showing significant improvements in retinal sensitivity and visual function.
About XLRP
X-linked retinitis pigmentosa (XLRP) is a severe, inherited retinal disease caused by mutations in the RPGR gene. It primarily affects males and progresses from night blindness in childhood to gradual vision loss, often resulting in severe visual impairment or blindness by middle age. There are currently no approved treatments for XLRP worldwide, leaving approximately 60,000 to 70,000 patients in China and 20,000 in the U.S. with an urgent unmet medical need.