rexaceract

Rexaceract (formerly GT-02287) is an orally administered, brain-penetrant small molecule positive allosteric modulator (PAM) and pharmacological chaperone of beta-glucocerebrosidase (GCase). Developed by Gain Therapeutics using its proprietary Magellan AI drug discovery platform, rexaceract is designed to stabilize misfolded GCase, facilitating its transport from the endoplasmic reticulum to lysosomes and mitochondria, thereby restoring enzymatic activity and reducing lysosomal dysfunction. The drug is primarily in clinical development for the treatment of Parkinson's disease (both GBA1-mutation-associated and idiopathic forms). It is also being investigated preclinically for other neurodegenerative and lysosomal storage disorders, including Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease.

Other names

4-amino-N-(2,3-dihydro-1H-inden-2-yl)-6-(2-fluoroanilino)pyridine-2-carboxamide