What is GB221 studying for Spinal Muscular Atrophy Type 1?

GB221 is a gene therapy being studied to see how safe and tolerable it is for infants with SMA Type 1. Researchers want to understand how this therapy might affect motor neuron function in babies with or at risk of SMA. The trial is led by Gemma Biotherapeutics and aims to gather important information on this treatment approach.

Who may be eligible for this Spinal Muscular Atrophy Type 1 clinical trial?

Infants aged between 2 weeks and less than 12 months with SMA Type 1 or those younger than 5 months at risk of developing it may be eligible. Participants can be treatment naive or currently using risdiplam. People with active viral infections or prior gene therapy are excluded. Please review eligibility criteria for more details.

Is there any cost to participate in this Spinal Muscular Atrophy Type 1 clinical trial?

Study-related care, including the gene therapy and all trial visits, is provided at no cost. However, personal travel or accommodation expenses may vary and are not guaranteed to be covered. Participants and their families should plan accordingly.

Actively Recruiting

Phase 1

Phase 2

Age: 2Weeks - 12Months

All Genders

ID07070999

A Phase 1-2 Study to Assess Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna in Infants With Spinal Muscular Atrophy Type 1

Led by Gemma Biotherapeutics · Updated on 2026-03-02

Participants Needed

Research Sites

17 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating GB221, a gene therapy that delivers a working SMN1 gene to motor neurons, for infants with spinal muscular atrophy SMA Type 1. The study focuses on two groups infants aged 2 weeks to less than 12 months who show symptoms of SMA Type 1 and have either never received treatment or are receiving the drug risdiplam, and infants aged 2 weeks to less than 5 months who are presymptomatic but at risk of developing SMA Type 1, also either untreated or receiving risdiplam. This open-label, Phase 1-2 study aims to assess the safety, tolerability, and efficacy of GB221 in these participants. Participants receive a single dose of GB221 delivered into the cisterna magna, a fluid-filled space near the brain and spinal cord. There are four cohorts two with symptomatic infants up to 12 months old and two with presymptomatic infants up to 5 months old, including those who are treatment naive or currently on risdiplam. This design allows researchers to explore safety and early efficacy in both symptomatic and at-risk infants. Throughout the study, lasting up to 18 months with multiple visits, participants will be closely monitored for treatment-related adverse events, changes in physical and neurological functions, vital signs, lab abnormalities, and immunogenicity markers. Assessments include electrocardiograms, serum cardiac troponin I levels, and various laboratory tests. Researchers will also evaluate motor milestones and neuromuscular function scores to measure the therapys impact.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 2Weeks - 12Months

All Genders

Eligibility Criteria

You may qualify if you...

Symptomatic participants diagnosed with SMA Type 1 by gene mutation analysis with bi-allelic SMN1 mutations and up to 3 copies of SMN2
Age between 2 weeks and less than 12 months at dosing with disease onset in the first 6 months of life
Presymptomatic participants at risk of SMA Type 1 by gene mutation analysis with bi-allelic SMN1 mutations and up to 2 copies of SMN2
Age between 2 weeks and less than 5 months (under 150 days) at dosing
Participants must be treatment naive or receiving risdiplam

You will not qualify if you...

Active viral infection (including HIV, Hepatitis B or C, or HTLV) at screening
History of invasive ventilatory support or oxygen saturation less than 95%
Immunosuppressive therapy ongoing or within 3 months prior to trial start
Participation in recent SMA treatment trials posing gene transfer risks
Prior gene therapy, hematopoietic transplant, or solid organ transplant
Severe scoliosis
Known allergy or hypersensitivity to prednisolone, glucocorticosteroids, or their excipients

Research Team

Jenna Tress

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