CRISPR treatment shows promise in rare muscle disease
Disease control
Completed
This early-phase trial tested a CRISPR-based gene therapy (HG302) in 4 boys aged 4 to 8 with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. The therapy aims to restore the missing dystrophin protein in muscle cells using a single intravenous injection. The ma…
Phase: EARLY_PHASE1 • Sponsor: HuidaGene Therapeutics Co., Ltd. • Aim: Disease control
Last updated May 06, 2026 16:13 UTC