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Sharp Therapeutics to Present Pipeline and Program Updates at World Orphan Drug Congress 2025

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, in Amsterdam, the Netherlands, and the Company will present at 2:45 p.m. (CET).

At the Congress, Sharp will highlight the status of its clinical targets and program pipeline, centered around Gaucher disease, Niemann-Pick disease type C, and familial frontotemporal dementia. The Company’s most advanced program, ‘901, is a differentiated small-molecule candidate designed to address a lysosomal storage disorder caused by a deficiency in the GBA product enzyme. Mutations in GBA are known to result in the development of both Gaucher disease and GBA Parkinson's disease.

“We are pleased to share Sharp’s progress with the rare-disease community at the World Orphan Drug Congress,” said Scott Sneddon, Ph.D., J.D., Chief Executive Officer of Sharp Therapeutics. “Our mission is to develop pill-based medicines that restore function in defective proteins and meaningfully improve the lives of patients with genetic diseases. With ‘901 for Gaucher disease and a growing pipeline of programs in other high-need genetically defined indications, we aim to deliver transformative therapies for patients. 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, while offering a favorable safety profile and the convenience of an oral medicine. We look forward to initiating clinical studies in the near term that are designed to efficiently establish proof of biology and lay the foundation for future registrational trials.”

About Sharp Therapeutics Corp.

First-Choice Therapies for Genetic Diseases

Sharp Therapeutics is a preclinical-stage company developing first-choice small-molecule therapeutics for genetic diseases. The Company’s discovery platform produces small molecule compounds that restore activity in mutated proteins giving the potential to treat genetic disorders with conventional pill-based medicines.

Caution Regarding Forward-Looking Information

Certain statements contained in this press release constitute "forward-looking information" as such term is defined in applicable Canadian securities legislation. The words "may", "would", "could", "should", "potential", "will", "seek", "intend", "plan", "anticipate", "believe", "estimate", "expect" and similar expressions are intended to identify forward-looking information. All statements other than statements of historical fact may be forward-looking information. Such statements reflect Sharp's current views and intentions with respect to future events, and current information available to Sharp, and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements that may be expressed or implied by such forward-looking information to vary from those described herein should one or more of these risks or uncertainties materialize. Should any factor affect Sharp in an unexpected manner, or should assumptions underlying the forward-looking information prove incorrect, the actual results or events may differ materially from the results or events predicted. Any such forward-looking information is expressly qualified in its entirety by this cautionary statement. Moreover, Sharp does not assume responsibility for the accuracy or completeness of such forward-looking information. The forward-looking information included in this press release is made as of the date of this press release and Sharp undertakes no obligation to publicly update or revise any forward-looking information, other than as required by applicable law.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

For additional information on Sharp, please visit: www.sharptx.com.

Sharp Therapeutics Corp.
Scott Sneddon, PhD, JD
CEO/CSO
Email: scott@sharptx.com


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