Gene therapy SPOT-03 enters human testing for duchenne muscular dystrophy
Disease control
Recruiting now
This early-phase trial tests a gene therapy called SPOT-03 in 9 boys with Duchenne muscular dystrophy (DMD), aged 2 to 8. The main goal is to see if the treatment is safe and tolerable. Researchers will also check if it increases dystrophin protein levels in muscles, which could …
Phase: EARLY_PHASE1 • Sponsor: Shanghai Siponuoyin Biotechnology Co Ltd • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC