Other
Sharp Therapeutics to Present Pipeline and Program Updates at World Orphan Drug Congress 2025
SHRX · Price
Executive Summary
- Sharp Therapeutics will present preclinical data on its lead ‘901 program for Gaucher disease and GBA Parkinson’s disease at the World Orphan Drug Congress 2025 in Amsterdam (Oct 27‑29, 2025).
- The presentation will highlight the status of Sharp’s pipeline targeting Gaucher disease, Niemann‑Pick disease type C, and familial frontotemporal dementia.
- CEO Scott Sneddon emphasized that ‘901 shows promise for treating both peripheral and CNS manifestations of Gaucher disease with an oral formulation and a favorable safety profile, and that clinical studies are planned in the near term.
Key Details
- Presentation Slot: World Orphan Drug Congress 2025, Amsterdam – 2:45 p.m. (CET) on one of the conference days (Oct 27‑29).
- Program Highlighted: ‘901, a small‑molecule candidate designed for lysosomal storage disorders caused by GBA deficiency.
- Target Indications: Gaucher disease and GBA‑associated Parkinson’s disease; pipeline also includes Niemann‑Pick disease type C and familial frontotemporal dementia.
- CEO Quote: “Based on our promising preclinical data, we believe ‘901 has the potential to treat both the peripheral and central nervous system manifestations of Gaucher disease… We look forward to initiating clinical studies in the near term…”
Notable Quotes
“Our mission is to develop pill‑based medicines that restore function in defective proteins and meaningfully improve the lives of patients with genetic diseases.” – Scott Sneddon, Ph.D., J.D., CEO
No financial figures or material transaction details were disclosed.
More from Sharp Therapeutics Corp.
May 28, 2026 · 16:00