Brazil Treats First Infant in In‑Country Gene Therapy Trial for Severe Spinal Muscular Atrophy

Overview

• The Health Ministry disclosed that the first infusion occurred in January at Hospital de Clínicas de Porto Alegre, with the baby’s identity protected and neurological monitoring underway.
• Fiocruz is conducting a phase 1/2 study with GemmaBio for up to 22 infants aged two weeks to one year, with a second participant anticipated in the coming months and completion targeted by late 2028 before a potential phase 3.
• The experimental product, identified as GB221, uses an AAV vector carrying a corrected gene and is delivered via cisterna magna to reach the central nervous system.
• Clinicians say this delivery route can treat patients even with pre‑existing antibodies to the viral vector, and the single‑dose procedure was accompanied by MRI and CT imaging.
• Brazil negotiated reduced pricing for future access, with reports of under half the market cost and Fiocruz indicating prices in Brazil could reach about 25% of current U.S. levels to enable possible SUS availability.