Lentivirus alpha-galactosidase A transduced stem cells

Lentivirus alpha-galactosidase A (α-gal A) transduced stem cells is an investigational gene therapy designed for the treatment of Fabry disease, a rare X-linked lysosomal storage disorder caused by deficiency of α-galactosidase A. The therapy involves harvesting autologous CD34+ hematopoietic stem/progenitor cells (HSPCs) from the patient, which are then genetically modified ex vivo using a recombinant lentiviral vector encoding the human GLA gene (which encodes α-galactosidase A). These engineered HSPCs are infused back into the patient following non-myeloablative conditioning. The modified cells engraft and produce functional α-galactosidase A enzyme systemically, leading to increased enzyme activity in plasma and leukocytes and reduction of toxic substrate accumulation such as globotriaosylceramide (Gb3) and lyso-Gb3. This approach aims to provide sustained systemic correction of the metabolic defect underlying Fabry disease[1][2][6][8]. Clinical trials have demonstrated safety with no serious adverse events attributable to the product and evidence of biochemical phenotype reversal in treated patients[1][2][6].

Other names

autologous CD34+ hematopoietic stem/progenitor cells engineered to express alpha-galactosidase Alentivirus-mediated gene therapy for Fabry disease