GT-GLA-S03

GT-GLA-S03 is an investigational autologous hematopoietic stem cell gene therapy being developed by Glafabra Therapeutics for the treatment of classic Fabry disease. The therapy utilizes the proprietary Live-cel platform to perform ex vivo lentiviral vector-mediated gene transfer of a functional GLA gene into a patient's own hematopoietic stem cells. Following a single intravenous infusion, these genetically modified cells engraft in the bone marrow and differentiate into various blood cell lineages that continuously produce and secrete the alpha-galactosidase A (GLA) enzyme. This mechanism, known as cross-correction, allows for the systemic restoration of enzyme activity, potentially reducing the accumulation of globotriaosylceramide (Gb3) and lyso-Gb3. GT-GLA-S03 aims to provide a durable, one-time therapeutic alternative to lifelong bi-weekly enzyme replacement therapy.