Pipeline Overview
GLA-H Gene Therapy
Transplantation therapy using hematopoietic stem cells (HSCs) engineered to restore alpha-galactosidase A (a-gal A) enzyme activity in patients.
GLA-T Gene Therapy
Cell therapy using rapamycin-attenuated T cells engineered to restore a-gal A enzyme activity in patients.
GLA-S Substrate Reduction Therapy
Modulation of gene expression to alter metabolic pathways and reduced the substrate for a-gal A enzyme.