Suzhou GenAssist Therapeutic Co., Ltd, a biopharmaceutical company based in China, has announced the commencement of an investigator-initiated-trial (ITT) for its base editing drug candidate, GEN6050X, in patients afflicted with Duchenne muscular dystrophy (DMD). DMD is a rare genetic disorder linked to the X-chromosome, typically caused by mutations in the dystrophin gene, which is essential for muscle cell membrane stability. The absence of functional dystrophin leads to the progressive muscle degeneration characteristic of DMD.
GEN6050X, an innovative intravenous cytosine base editing drug employing a dual AAV9 vector, targets DMD patients with exon 50跳跃. As the first of its kind to enter clinical trials globally, GEN6050X leverages second-generation CRISPR-Cas9 technology to perform a single systemic correction of the mutated DMD genes, aiming to permanently restore dystrophin expression. This approach holds the potential to transform the treatment landscape for DMD by offering a one-time treatment option. The company anticipates filing Investigational New Drug (IND) applications for GEN6050X in both the United States and China in November, marking a significant step towards bringing this groundbreaking therapy to patients. – Flcube.com
