Shanghai-based genome medicines specialist HuidaGene Therapeutics has announced the initiation of the MUSCLE study for its CRISPR-based DNA-editing therapy, HG302, which targets Duchenne muscular dystrophy (DMD), with the first subject now dosed.
Understanding Duchenne Muscular Dystrophy and HG302
Duchenne muscular dystrophy is a severe X-linked recessive disorder and a prevalent form of progressive muscular dystrophy. It is caused by mutations in the DMD gene, leading to the absence of functional dystrophin protein. The disease’s progression results in increasing muscle weakness, muscle degeneration, loss of ambulation by adolescence, life-threatening complications in early adulthood, and a shortened lifespan. Current treatment options are limited to symptom management. HG302, utilizing HuidaGene’s proprietary hfCas12Max, a high-fidelity CRISPR nuclease developed through the AI-guided HG-PRECISE platform, precisely edits the DMD exon51 splice-donor site, enabling exon skipping to restore dystrophin production. This one-time treatment option for DMD patients amenable to exon51 skipping or reframing has previously obtained Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) in the US.
MUSCLE Trial Design and Objectives
The MUSCLE (Multidose study to Understand the Safety of Crispr editing therapy and its long-Lasting Effects in DMD) trial is an open-label, multidose dose-escalation, first-in-human study. It is expected to enroll ambulatory boys between 4 and 8 years old with DMD who exhibit impaired muscle function. The study aims to evaluate the safety, tolerability, and preliminary efficacy of HG302. Key outcomes include dystrophin restoration, functional muscle improvements, and quality of life metrics.-Fineline Info & Tech
