NCT07070999

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

A Phase 1-2, Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna of Pediatric Participants From 2 Weeks to Younger Than 12 Months of Age With Spinal Muscular Atrophy Type 1

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 22 (estimated)
• Sponsor: Gemma Biotherapeutics · Industry
• Sex: All
• Age: 2 Weeks – 12 Months
• Healthy volunteers: Not accepted

Summary

GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1. participants aged from 2 weeks to younger than 12 months presenting with symptoms of SMA Type 1 who have never received a treatment OR are receiving the drug risdiplam 2. participants aged from 2 weeks to younger than 5 months who are at risk of developing SMA Type 1 (presymptomatic) and have never received treatment OR are receiving the drug risdiplam.

Conditions

• Spinal Muscular Atrophy Type I

Interventions

Type	Name	Description
BIOLOGICAL	GB221	GB221

Timeline

• Start date: 2026-01-06
• Primary completion: 2028-12-01
• Completion: 2029-04-01
• First posted: 2025-07-17
• Last updated: 2026-03-02

Locations

1 site across 1 country: Brazil