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Gemma Biotherapeutics

Clinical trials sponsored by Gemma Biotherapeutics, explained in plain language.

Trials to join now! 1 Not yet finished but already full! 1

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