Brasília – Anvisa authorized, on November 24, a clinical trial led by Fiocruz and the U.S.-based biotechnology company Gemma Biotherapeutics (GEMMABio), marking the first gene-therapy study for a rare disease in Brazil. The trial targets children with the most severe form of spinal muscular atrophy type 1 (AME type 1) and uses a viral vector to deliver a functional copy of the SMN1 gene, responsible for the condition.

The approved study is a phase 1/2 trial designed to evaluate safety, tolerability and initial signs of efficacy. It will be conducted in at least three clinical centers in the country, including Hospital de Clínicas de Porto Alegre (HCPA) and the Hospital das Clínicas at Unicamp. If successful, the therapy may later be registered and produced in Brazil by Bio-Manguinhos, supporting national capacity for advanced therapies and strengthening the health industrial complex.

Source: Anvisa