Sharp Therapeutics Corp. Engages Rho Inc. to Help Advance Compounds into Clinical Trials for Gaucher Disease

Pittsburgh, Pennsylvania and Toronto, Ontario--(Newsfile Corp. - July 29, 2025) - Sharp Therapeutics Corp. (TSXV: SHRX) ("Sharp" or the "Company"), a preclinical-stage biotechnology company developing small molecule therapies to treat genetic diseases, today announced that it has engaged Rho, Inc. ("Rho"), a global contract research organization (CRO), to support Sharp's preparation and planned submission of its Investigational New Drug Application ("IND") to the U.S. Food and Drug Administration (the "FDA") for the evaluation of its clinical candidate compound ("'901") for the treatment of Gaucher disease.

Gaucher disease is a genetic disorder caused by a deficiency in the enzyme glucocerebrosidase (also called beta-glucosidase or GBA1). Sharp's small molecule compound is a potential new orally available CNS-penetrant therapy for the treatment of Gaucher disease.

"We are excited by the preclinical data from our Gaucher candidate, '901, and look forward to Rho's support in preparing our IND and other filings," said Scott Sneddon, PhD, JD and Chief Executive Officer of Sharp. "We intend to meet with FDA on our Phase I clinical plan, and to file for orphan drug designation this year. IND-enabling studies are also planned to commence before end of 2025. This is a key milestone for Sharp as we transition from a preclinical-stage to a clinical-stage company," he added.

About '901

The '901 compound increases GBA1 activity for numerous mutations in Gaucher patient-derived cells, as well as increasing substrate turnover in a rodent model of Gaucher. Preclinical studies show the compound to be orally available and CNS-penetrant with good safety and preclinical pharmacology profiles. The compound is being targeted to all types of Gaucher with an application to GBA-associated Parkinson's disease also possible.

The scientific data supporting the '901 series of compounds was presented at the GBA1 Conference in Montreal on June 5, 2025. That presentation is available on the Company's website at www.sharptx.com.

About Gaucher Disease

Gaucher disease is a lysosomal storage disease caused by a deficiency in the GBA1 enzyme. Gaucher is the most common lysosomal storage disorder.

Without this enzyme, the glucocerebroside lipid accumulates within the lysosomes of certain cells, particularly macrophages (immune cells). These overloaded cells, called "Gaucher cells," become enlarged and dysfunctional. The buildup primarily affects the spleen, liver, bone marrow, and sometimes the nervous system, leading to organ enlargement and the characteristic symptoms of the disease.

This is the classic pattern of lysosomal storage diseases: enzyme deficiency → substrate accumulation → cellular dysfunction → organ pathology.

About Rho, Inc.

Rho is a global, privately held contract research organization (CRO) headquartered in Research Triangle Park, a biotech hub in North Carolina, US. Rho provides a full range of drug development services, from program strategy through to clinical trials and marketing applications. Since 1984, Rho has been a trusted partner to some of the most innovative pharmaceutical, biotechnology and medical device companies as well as academic and government organizations. Dedicated to service excellence and cross-functional collaboration, Rho's therapeutic expertise, employee focus and commitment to strong site relationships change what it means to work with a CRO – accelerating time to market, maximizing ROI, and delivering consistent, smarter and more efficient programs. Experience Rho by following the company on LinkedIn.

About Sharp Therapeutics Corp.

- First-Choice Therapies for Genetic Diseases

Sharp Therapeutics is a preclinical-stage company developing small-molecule therapeutics for genetic diseases. The Company's discovery platform combines novel high throughput screening technologies, with compound libraries computational optimized based on the physics and biology of cellular trafficking defects and allosteric activation of proteins. The platform produces small molecule compounds that restore activity in mutated proteins giving the potential to treat genetic disorders with conventional pill-based medicines.

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

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.

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