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Sharp Therapeutics to Present Pipeline and Program Updates at World Orphan Drug Congress 2025
Globenewswire· 2025-10-08 20:00
PITTSBURGH, Pa. and TORONTO, Oct. 08, 2025 (GLOBE NEWSWIRE) -- Sharp Therapeutics Corp. ("Sharp" or the "Company") (TSX-V: SHRX) (OTCQB: SHRXF), a biotechnology company developing small-molecule therapeutics for genetic diseases, today announced that it will present preclinical data from its lead Phase 1 ready ‘901 program being developed to treat Gaucher disease and Glucocerebrosidase (GBA) Parkinson's disease at the World Orphan Drug Congress 2025. The meeting will take place from October 27 to 29, 2025, ...
Sharp Therapeutics to Present Pipeline and Program Updates at World Orphan Drug Congress 2025
Globenewswire· 2025-10-08 20:00
Core Insights - Sharp Therapeutics Corp. is set to present preclinical data for its lead program '901, aimed at treating Gaucher disease and GBA Parkinson's disease, at the World Orphan Drug Congress 2025 in Amsterdam from October 27 to 29, 2025 [1][2] Company Overview - Sharp Therapeutics is a preclinical-stage biotechnology company focused on developing small-molecule therapeutics for genetic diseases, with a discovery platform that produces compounds to restore activity in mutated proteins [4] Clinical Pipeline - The company’s pipeline includes programs targeting Gaucher disease, Niemann-Pick disease type C, and familial frontotemporal dementia, with '901 being the most advanced candidate designed to address lysosomal storage disorders caused by GBA enzyme deficiencies [2][3] Leadership Statement - The CEO of Sharp Therapeutics expressed the company's commitment to developing pill-based medicines that restore function in defective proteins, aiming to improve the lives of patients with genetic diseases [3]