Gemma Biotherapeutics (GEMMABio), a US-based clinical-stage global genetic medicines company, announced on Thursday that it has dosed its first parent in the Phase 1/2 CHARISMA clinical trial of GB221, an investigational, next-generation gene therapy for Spinal Muscular Atrophy Type 1 (SMA1).

The CHARISMA trial (NCT07070999) is the first clinical evaluation of a next-generation gene therapy for SMA1 delivered directly to the cerebrospinal fluid by intracisterna-magna injection. Using proprietary, central nervous system platform technology licensed from the University of Pennsylvania, GB221 was developed to express a functional, codon-optimised SMN1 gene from a modified transgene expression cassette designed to reduce overexpression-related toxicities and sensory neurotoxicity. The trial will evaluate safety, tolerability, and efficacy in paediatric participants from two weeks to younger than twelve months of age. Cohorts will include symptomatic and presymptomatic participants.

GEMMABio CEO James M. Wilson, MD, PhD, who also serves as a Member of the International Expert Advisory Board at Brazil's Casa dos Raros (House of Rare), said, "I would like to express our gratitude for the young child and family who were courageous in being the first to accept our investigational gene therapy for SMA1. We appreciate our steadfast partners at Fiocruz / Bio-Manguinhos, Brazil Ministry of Health, ANVISA, Casa dos Raros, Intrials, and the Hospital de Clínicas de Porto Alegre medical team who worked with us and who demonstrated the tremendous potential of international public-private partnerships with designated regional Research and Treatment Centres. We anticipate expanding the CHARISMA clinical trial in Brazil and working together on future clinical trials for other rare disease gene therapies."