BBM-F101

BBM-F101 is an investigational adeno-associated virus (AAV) vector-based gene therapy developed by Belief BioMed for the treatment of Fabry disease, an X-linked lysosomal storage disorder. The therapy is designed to deliver a functional copy of the *GLA* gene, which encodes the enzyme alpha-galactosidase A (α-Gal A). In patients with Fabry disease, mutations in this gene lead to a deficiency of α-Gal A, resulting in the systemic accumulation of glycosphingolipids, primarily globotriaosylceramide (Gb3), which causes progressive damage to the kidneys, heart, and nervous system. BBM-F101 aims to achieve stable, long-term endogenous expression of the functional enzyme following a single intravenous infusion, potentially eliminating the need for regular enzyme replacement therapy (ERT). It is currently being evaluated in clinical trials for both adult and pediatric patient populations.