Frontera Therapeutics Doses First Patient in Phase 1 Clinical Trial for Gene Therapy FT-001 for the Treatment of Leber Congenital Amaurosis-2

Frontera Therapeutics announced that it has dosed the first patient in a phase 1 clinical trial of its lead gene therapy program, FT-001, for the treatment of Leber Congenital Amaurosis-2, a severe inherited retinal disease related to a mutation in the RPE65 gene. FT-001 is administered by a one-time injection into the subretinal space of the eye that delivers a functional copy of the human RPE65 gene to the nuclei of the patient’s retinal cells.

“This is an exciting achievement for Frontera Therapeutics as we begin our first clinical trial and take a significant step forward as a company,” said Yong Dai, PhD, CEO and founder of Frontera Therapeutics.

In September 2022, Frontera successfully obtained an IND from the Center for Drug Evaluation (CDE), NMPA, in China. Additionally, the FDA cleared an IND for FT-001 earlier this year in April. This phase 1 clinical trial is designed to evaluate the safety, tolerability, pharmacokinetics, and therapeutic effects of FT-001 as a single dose in patients with LCA-2, and is being conducted in China with an initial data readout expected in the second half of 2023.