Core Viewpoint - Sharp Therapeutics Corp. is advancing its lead program '901, aimed at treating Gaucher disease and GBA Parkinson's disease, with preclinical data to be presented at the World Orphan Drug Congress 2025 [1][2][3] Group 1: Company Overview - Sharp Therapeutics is a biotechnology company focused on developing small-molecule therapeutics for genetic diseases, particularly targeting lysosomal storage disorders [4] - The company’s discovery platform is designed to create small molecule compounds that restore activity in mutated proteins, potentially allowing for treatment through conventional pill-based medicines [4] Group 2: Clinical Programs and Pipeline - The '901 program is the most advanced in Sharp's pipeline, specifically targeting Gaucher disease and GBA Parkinson's disease, which are linked to mutations in the GBA enzyme [2][3] - The company is also developing therapies for Niemann-Pick disease type C and familial frontotemporal dementia, indicating a broader focus on high-need genetically defined indications [2] Group 3: Future Plans and Expectations - Sharp Therapeutics aims to initiate clinical studies for '901 in the near term, with the goal of establishing proof of biology and laying the groundwork for future registrational trials [3] - The company emphasizes the potential of '901 to treat both peripheral and central nervous system manifestations of Gaucher disease, highlighting its favorable safety profile and oral administration convenience [3]

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]