Gene therapy SPOT-03 hopes to restore muscle protein in boys with DMD
Disease control
Recruiting now
This early-phase study tests a new gene therapy called SPOT-03 in 9 boys aged 2 to 8 with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and tolerable. Researchers will also check if SPOT-03 can increase dystrophin, a key muscle protein missin…
Phase: EARLY_PHASE1 • Sponsor: Shanghai Siponuoyin Biotechnology Co Ltd • Aim: Disease control
Last updated May 17, 2026 18:41 UTC