Frontera Therapeutics Receives Additional IND Clearance for its Lead Program FT-001

Frontera Therapeutics announced that its lead program, FT-001, a gene therapy product candidate targeting inherited retinal degenerations (IRDs) with a RPE65 mutation, successfully obtained an IND from the Center for Drug Evaluation (CDE), NMPA, in China. The FDA also cleared an IND for FT-001 in April.

Inherited retinal degenerations or dystrophies are a heterogenous group of serious eye conditions that cause visual impairment that may lead to blindness. Currently, nearly 300 genetic mutations that are associated with IRD have been identified, including those in the retinal pigment epithelium 65 kDa protein (RPE65). There is no approved therapy for this condition in China and other countries where there is unmet medical need.

Frontera’s FT-001 is an AAV gene therapy product 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. The addition of a functional copy of the RPE65 gene to the retinal RPE cells allows normal functioning human hRPE65 proteins to be expressed and improves a patient’s functional vision.

“Since Frontera’s founding, we have rapidly progressed a significant number of programs into IND enabling studies. This year, we have advanced our lead program, FT-001, and have received both FDA and China CDE IND clearances. Building on our product development momentum, we intend to advance a number of programs into clinical studies in the next 6 to 9 months. We will also leverage our APEX Technology & Manufacturing platform to create new innovative programs targeting large global markets such as cardiovascular, metabolic, and CNS diseases,” Yong Dai, PhD, founder and CEO of Frontera, said in a company news release.