NEW YORK – Chinese gene therapy developer Suzhou GenAssist Therapeutics on Saturday said the US Food and Drug Administration has accepted a request for a pre-investigational new drug (IND) application meeting for its first base-editing product.
The pre-IND application request is for GEN6050, an investigational treatment for Duchenne muscular dystrophy, a genetic disorder in which mutations in the DMD gene cause the body not to express the protein dystrophin.
GEN6050 uses a gene-editing approach known as base editing in vivo to restore expression of dystrophin by skipping exon 50 in the DMD gene.
Suzhou GenAssist Therapeutics, which was founded in 2020, develops gene-editing therapies to treat genetic neuromuscular diseases. It has a pipeline of five drugs targeting other forms of Duchenne muscular dystrophy, spinal muscular atrophy, and beta thalassemia and has global rights to base-editing technology licensed from the Chinese Academy of Nutrition and Health and Westlake University.
The company is headquartered in Suzhou, China, with subsidiaries in Shanghai and Boston.