Sharp Therapeutics to Showcase Promising Genetic Disease Therapies at WODC 2025
Sharp Therapeutics Corp., a preclinical-stage company, is set to present its latest findings at the World Orphan Drug Congress 2025. The presentation will focus on the status of the company's clinical targets and pipeline, including Gaucher disease, Niemann-Pick disease type C, and familial frontotemporal dementia.
Sharp's most promising program is '901, a small-molecule candidate designed to treat a lysosomal storage disorder caused by a deficiency in the GBA product enzyme. This disorder is linked to Gaucher disease and GBA Parkinson's disease, both of which are caused by mutations in GBA. '901 has the potential to treat both peripheral and central nervous system manifestations of Gaucher disease.
Sharp's discovery platform produces small molecule compounds that restore activity in mutated proteins. This innovative approach aims to treat genetic disorders using conventional pill-based medicines. The company's goal is to deliver transformative therapies for patients with genetic diseases.
Sharp Therapeutics Corp. will present preclinical data from its lead Phase 1 ready '901 program at the World Orphan Drug Congress 2025. The company's focus on genetic diseases and innovative small-molecule therapeutics offers hope for patients awaiting effective treatments.