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

Recruiting now · Phase 1/Phase 2

Conditions studied: Spinal Muscular Atrophy Type I

In brief

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.

Key facts

Study ID

NCT07070999

Run by

Gemma Biotherapeutics

People needed

22

Starts

2026-01-06

Expected to finish

2029-04-01

Last updated by the study team

2026-06-29

Who can join

Age: 0 and older, up to 1. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Symptomatic Participants
• Diagnosis of SMA Type 1 based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and up to 3 copies of SMN2
• Participants must be 2 weeks to < 12 months of age at the time of dosing with disease onset of during the first 6 months of life.
• Presymptomatic Participants
• At risk of SMA Type 1 based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and up to 2 copies of SMN2
• Participants must be 2 weeks to < 5 months (< 150 days) of age at the time of dosing.

You may not qualify if…

• Any suspected or confirmed active viral infection at screening baseline (including HIV, Hepatitis B or C, or human T Cell lymphotropic viruses [HTLV])
• History of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry <95% saturation.
• Ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial (e.g. corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab)
• Participation in a recent SMA treatment clinical trial that, in the opinion of the Investigator, creates unnecessary risks for gene transfer.
• Prior history of gene therapy for any indication, hematopoietic transplant or solid organ transplant
• Subjects with severe scoliosis
• Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients.

Where it is running

• Hospital de Clínicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)

Full record on ClinicalTrials.gov

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