China CDE accepts Investigational New Drug (IND) application for lead gene therapy product candidate, FT-001, for the treatment of a rare inherited eye disease.
BEDFORD, Massachusetts, and SHANGHAI, China September 21, 2022 — Frontera Therapeutics, a global clinical-stage biotechnology company that seeks to develop novel and best-in-class gene therapy medicines to improve the lives of patients across multiple disease areas, today 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. Frontera previously announced that the U.S. Food and Drug Administration (FDA) also cleared an IND for FT-001 earlier this year 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 U.S. 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 six 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,” said Yong Dai, Ph.D., founder and CEO of Frontera.
Frontera Therapeutics is a global, fully-integrated, clinical stage biotechnology company leveraging its novel APEX Technology & Manufacturing platform to develop and manufacture superior and novel gene therapy candidates across multiple disease areas. The platform is an innovative adeno-associated virus (AAV) gene expression system that aims to optimize both new and clinically validated AAV vectors to enhance the safety and efficacy profiles of gene therapy products. The in-house GMP manufacturing capabilities enable rapid production across the product lifecycle, and efficiently advance AAV therapies from research to clinical development. Frontera’s development pipeline spans not only orphan diseases, but also targets large patient markets – including ophthalmology, hematology, cardiovascular, neurology and metabolic diseases. Frontera Therapeutics has offices in the United States and China, allowing it to target global patient populations. For additional information about Frontera Therapeutics, please visit the company’s website at www.fronteratherapeutics.com.