Sharp Therapeutics GBA program

Sharp Therapeutics is developing an unnamed small molecule therapeutic program targeting the GBA (glucosylceramidase) pathway for the treatment of Gaucher's disease and Parkinson's disease. The program utilizes Sharp's proprietary SHARP (Small-molecule High-throughput Automated RNA-targeting Platform) to identify small molecules that modulate RNA to address loss-of-function mutations or haploinsufficiency of the GBA enzyme. In Gaucher's disease, deficient GBA activity leads to the accumulation of glucosylceramide, while in Parkinson's disease, GBA mutations are a significant genetic risk factor associated with alpha-synuclein aggregation. By restoring or enhancing GBA activity through RNA modulation, this program aims to mitigate underlying biochemical defects and slow disease progression in GBA-related disorders.

Other names

GBA programGlucosylceramidase program