New gene therapy hopes to stop deadly infant muscle disease
Disease control
Recruiting now
This study tests a one-time gene therapy called GB221 in infants with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The therapy delivers a working SMN1 gene to nerve cells to help them survive and function. The trial includes babies aged 2 weeks to unde…
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated May 13, 2026 16:01 UTC