GEMMABio’s Next-Gen SMA1 Gene Therapy Cleared for Clinical Trials in Brazil

A major step forward for spinal muscular atrophy type 1 (SMA1) treatments is underway in Brazil. GEMMABio has secured approval from ANVISA to begin clinical trials for GB221, a next-generation gene therapy designed to improve survival, reduce toxicity, and make treatment more affordable for patients across Latin America.

The announcement came during the INAME 2025 Spinal Muscular Atrophy Congress in São Paulo, where GEMMABio’s CEO, Dr. James M. Wilson, shared the program’s promising preclinical results.

Inside GB221: A New Approach to SMA1 Gene Therapy

GB221 is designed as a one-time gene therapy delivered directly into the cerebrospinal fluid through intra-cisterna magna injection (ICM). This approach aims to deliver potent benefits with fewer side effects.

Key Features of GB221

• Uses AAVhu68, a human-derived vector engineered for safer CNS delivery.
• Contains a codon-optimized SMN1 gene to restore motor neuron function.
• Uses a modified transgene cassette built to reduce motor and sensory neurotoxicity.
• Delivered directly to the CNS to avoid systemic exposure and lower required doses.

What the Preclinical Data Shows?

The therapy has delivered encouraging results across both animal models and non-human primates.

Preclinical Highlights

• Improved survival and neuromotor function in SMA1 mouse models.
• Achieved high motor neuron transduction in NHPs without toxicity.
• Dramatically reduced off-target liver delivery, a common risk in other gene therapies.
• Lower vector doses via ICM delivery may significantly cut manufacturing costs.

These outcomes support GB221’s potential to offer a safer, more accessible treatment—crucial in a disease where most untreated infants do not survive past their second birthday.

Why Brazil Matters for This Trial?

Brazil will serve as GEMMABio’s regional hub for the SMA1 program. The country’s public healthcare system, SUS, covers over 200 million people and provides a powerful platform for access-driven innovation.

A Multi-Institution Partnership

• Fiocruz, the country’s premier biomedical institution, is working with GEMMABio on clinical readiness.
• Intrials, a Brazil-based CRO, will help manage trial operations.
• A technology-transfer agreement will enable local vector production at Bio-Manguinhos.
• This supports Brazil’s national goal to strengthen its health and biotech industrial ecosystem.

The focus is clear: bring next-generation gene therapies closer to patients while reducing dependence on costly imported treatments.

Why Affordability Is a Central Theme?

Existing SMA1 therapies are life-saving but extremely expensive, limiting access across Latin America.

Dr. Wilson highlights the urgency:

“Without treatment, patients will not survive to their second birthday.”

Direct CNS delivery allows for lower vector doses, reducing both toxicity concerns and cost of goods. If clinical results match preclinical performance, GB221 could become a more affordable, scalable alternative.

Brazilian neurologist Dr. Jonas Morales Saute adds:

“A one-time, more affordable treatment could transform SMA1 care across LATAM.”

GEMMABio expects to open three clinical sites in Brazil and dose the first patient within months. A parallel site will launch in Abu Dhabi in 2026, expanding access across the Middle East.

What to Watch For

• Safety data from CNS-targeted delivery in human infants.
• Durability of gene expression and motor improvements.
• Real-world cost impact from lower vector doses.
• How technology transfer accelerates regional manufacturing capacity.

Trial design details will be available on ClinicalTrials.gov, though enrollment has not yet opened.

About SMA1

SMA is a genetic disorder caused by mutations in SMN1, leading to rapid degeneration of motor neurons. SMA1 is the most severe form, with onset before six months and a global incidence of 1:17,000 births. Without intervention, survival beyond 18 months is rare.

About GEMMABio

GEMMABio develops advanced therapies for rare diseases with a focus on global access, affordability, and translational speed. Led by renowned gene therapy pioneer Dr. Jim Wilson, the company works to move treatments from research labs into clinical care faster and more efficiently. Learn more at gemmabiotx.com.

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