alpha-galactosidase A chaperone

Relay Therapeutics is developing an investigational small molecule non-inhibitory chaperone for the treatment of Fabry disease, a rare genetic disorder. The drug targets alpha-galactosidase A (αGal), an enzyme that is deficient in Fabry disease patients due to mutations in the GLA gene. This deficiency leads to the accumulation of globotriaosylceramide (Gb3) in blood cells and tissues throughout the body. The small molecule chaperone is designed to stabilize the αGal protein without inhibiting its activity, thereby enabling greater Gb3 clearance across organs. The program was disclosed in June 2024 as part of Relay's expansion beyond precision oncology into genetic diseases, utilizing the company's Dynamo platform. The drug is intended as a potential chronic treatment for Fabry disease, either alone or in combination with enzyme replacement therapy (ERT).

Other names

αGalRelay Therapeutics alpha-galactosidase A program