On May 30, 2024, Suzhou GenAssist Therapeutical Co., Ltd, the operation entity of GenAssist Ltd (GenAssist), announced that the IIT(NCT06392724) kickoff meeting for its first base editing DMD drug, GEN6050X, has be successfully held in Peking Union Medical College Hospital (PUMCH).
This program is sponsored by Suzhou GenAssist Therapeutics and led by Professor Yi Dai, the Deputy Director of Neurology Department at PUMCH. Professor Dan Guo, the Director of Clinical Biobank, also attended this meeting along with other participants from clinical research ward and the Department of laboratory medicine.
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. Pre-IND meeting for GEN6050, pre-optimized version of GEN6050X, has already been held in April 2023.
The kickoff of this program marks a substantial milestone for China-based innovation in DMD treatment area. More close collaborations between biotech companies and top clinical medical centers are needed to address these unmet clinical needs. We look forward to the successful completion of this program.
About Neurology Department of Peking Union Medical College Hospital (PUMCH)
Established in 1921, the Department of Neurology at Peking Union Medical College Hospital is the earliest neurology department in China. Over the past century, it has remained as the top-class department in the field of clinical neurology in China and has been ranked No.1 in the Fudan Neurology Specialist Rankings for years.
The department owns comprehensive professional settings, with strong technical capabilities and solid basic clinical skills. It emphasizes conducting clinically guided scientific research, aiming to explore innovative diagnostic and therapeutic methods, improving clinical diagnosis and treatment levels, and providing high-level medical services to numerous patients with neurological disorders.
