GLA-S Substrate Reduction Therapy
GLA-S substrate reduction therapy uses modulation of gene expression pathways to reduce the amount of substrate available for alpha-galactosidase A (a-gal A) enzyme and lead to reduce formation of toxic metabolites. Screens are planned with GLA enzyme-defective cells to find candidate therapeutics leading to reduction in toxic metabolite formation, such as globotriaosylceramide (Gb3) and lyso-Gb3.
The screening process involves using GLA defective cells as test group and normal cells as control group, with a readout as increase levels of toxic metabolite formation.
Drugs that effectively reduce toxic metabolite formation will be identified as leads for further development.
Drug is administered orally in a pill form.
With GLA-S substrate reduction therapy, strategies for substrate reduction can be combined with the gene therapies, and potentially other existing treatments, to further decrease toxic metabolite production through synergistic drug combinations.
