One-Shot gene therapy trial offers hope for infants with devastating muscle disease
Disease control
Recruiting now
This study is testing a single dose of an experimental gene therapy called GB221 for babies with spinal muscular atrophy type 1 (SMA1). The goal is to see if delivering a working copy of the missing SMN1 gene directly to the nervous system is safe and can help improve muscle func…
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC