GEMMABio AAV Gene Therapy for SMA1 Cleared for Clinical Trial in Brazil

PHILADELPHIA, PA / SÃO PAULO, BRAZIL – November 24, 2025-  Gemma Biotherapeutics (GEMMABio), a leading global genetic medicines company, today announced a major step toward bringing its novel AAV gene therapy for Spinal Muscular Atrophy type 1 (SMA1) to the clinic. Brazil’s health regulatory agency, ANVISA, has authorized the clinical trial for GB221, a next-generation AAV vector designed for direct central nervous system (CNS) delivery.

The announcement closely follows the presentation of promising preclinical data for GB221 by GEMMABio’s President and CEO, Dr. James M. Wilson, at the INAME 2025 Spinal Muscular Atrophy Congress in São Paulo, Brazil.

Next-Gen AAV Designed for Enhanced Safety and Access

In his address, “Gene Therapy for SMA1: History, R&D on Next Generation Therapy, and the Challenge of Affordability,” Dr. Wilson detailed the design of GB221:

• Vector Composition: GB221 is a one-time AAV gene therapy that utilizes the human AAV isolate, AAVhu68. This AAV encases a functional copy of a codon-optimized SMN1 gene within a modified transgene cassette.

• Delivery: The AAV vector is delivered directly to the cerebrospinal fluid (CSF) via intra-cisterna magna (ICM) injection.

• Safety Profile: Preclinical studies demonstrated high levels of motor neuron transduction and improved survival in SMA1 mouse models without toxicity in non-human primates (NHPs). Crucially, the ICM delivery strategy substantially reduced off-target AAV delivery to the liver in NHP studies.

Dr. Wilson noted that the direct CNS delivery allows for lower AAV vector doses to be used without compromising therapeutic gene expression in the target motor neurons. This strategy mitigates systemic toxicity related to the higher AAV vector doses observed in other intravenously administered AAV gene therapies.

“We look forward to opening SMA1 clinical trial sites in Brazil and moving this program into the clinic,” said Dr. Jim Wilson. “SMA1 is a devastating motor neuron disease with high unmet need, as existing disease-modifying therapies are expensive and the cost sharply restricts access to care. This novel AAV gene therapy aims to change that.”

Brazil Partnership Aims to Boost AAV Affordability

GEMMABio’s clinical activities in Brazil are a core component of a unique partnership agreement with the Oswaldo Cruz Foundation (Fiocruz), announced in October 2024.

The proprietary CNS platform technology, licensed from the University of Pennsylvania, demonstrated a reassuring preclinical safety profile and durable expression in NHP studies. The use of lower AAV doses via ICM delivery is anticipated to significantly reduce the cost of goods sold (COGS), which could enhance the affordability of this novel AAV gene therapy and expand patient access through Brazil’s publicly funded healthcare system, SUS.

The partnership also includes GEMMABio transferring AAV vector production technology to the Institute of Technology on Immunobiologicals (Bio-Manguinhos) at Fiocruz. This aligns with the Brazil Ministry of Health’s directive to strengthen its national health industry, ensuring a local, affordable supply of this cutting-edge AAV technology.

“A more affordable, one-time AAV treatment option distributed through research and treatment centers in Brazil has the potential to revolutionize SMA1 patient care in LATAM in a very short period of time,” stated Dr. Jonas Morales Saute, a principal investigator for the clinical trial.

GEMMABio anticipates opening three clinical sites in Brazil and dosing the first SMA1 patient within the next few months. Brazil will serve as a regional hub for GEMMABio’s AAV gene therapy programs, with an additional international clinical site expected to initiate in the Middle East in 2026.

About SMA1

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease caused by a mutation in the survival motor neuron gene 1 (SMN1). SMA Type 1 (SMA1) is the most severe form; untreated patients typically do not survive past 18 months of age.