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

> **NCT07070999** · PHASE1,PHASE2 · RECRUITING · sponsor: **Gemma Biotherapeutics** · enrollment: 22 (estimated)

## Conditions studied

- Spinal Muscular Atrophy Type I

## Interventions

- **BIOLOGICAL:** GB221

## Key facts

- **NCT ID:** NCT07070999
- **Lead sponsor:** Gemma Biotherapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2026-01-06
- **Primary completion:** 2028-12
- **Final completion:** 2029-04
- **Target enrollment:** 22 (ESTIMATED)
- **Last updated:** 2026-03-02


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07070999

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07070999, "Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1". Retrieved via AI Analytics 2026-07-19 from https://api.ai-analytics.org/clinical/NCT07070999. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*