On July 29, 2024, Suzhou GenAssist Therapeutical Co., Ltd, the operation entity of GenAssist Ltd (GenAssist), announced that Pre-IND (PIND) meeting request for its first base editing DMD drug, GEN6050X, has be submitted to NMPA. Previously, the PIND meeting to FDA was held in April 2023. The PIND filing to FDA and NMPA indicated the company has ambition to evaluate its innovative drug in clinical trial globally.
GenAssist dedicates to develop DMD base editing drugs using its proprietary RNA editing-free TAM cytosine base editing technology, which was licensed from Chinese Academy of Nutrition and Health. Currently, the treatment of DMD mainly relies on glucocorticoids to delay disease progression. GEN6050X injection targets DMD patients amenable for exon 50 skipping. Through one-shot systemic administration, GEN6050X can permanently restore the expression of dystrophin through editing the mutated DMD gene. This may provide a curative solution for DMD. At the same time, IND filing of GEN6050X（US and China）will be expected in November 2024.
Related links: https://www.cde.org.cn/main/xxgk/listpage/f1b85f03689d513e5cce1c6a3185af25
