Business
Sharp Therapeutics Corp. Nominates Clinical Candidate for Gaucher's Disease
Pittsburgh, Pennsylvania and Toronto, Ontario--(Newsfile Corp. - May 13, 2025) - Sharp Therapeuti...
About this update from Sharp Therapeutics Corp.
[{"type":"text","content":"Sharp Therapeutics Corp. Nominates Clinical Candidate for Gaucher's DiseasePittsburgh, Pennsylvania and Toronto, Ontario--(Newsfile Corp. - May 13, 2025) - Sharp Therapeutics Corp. (TSXV: SHRX) (\"Sharp\" or the \"Company\"), a pre-clinical-stage biotechnology company developing small molecule therapies to treat genetic diseases, announces that it has nominated a small molecule compound from its GBA program for clinical development in Gaucher's disease. Sharp has launched its clinical development program for Gaucher's disease by nominating a compound from the SEL-148,721 series of GBA1-restoring small molecules to enter IND-enabling studies. The company plans to begin compound scale-up and formal safety studies during the second half of 2025. If successful, the Company expects to file an IND application with the Food and Drug Administration and enter Phase I clinical trials in 2026.About the SHRX Gaucher's Program.Sharp discovered the SEL-148,172 series by applying its Disco™ discovery platform to identify compounds that enhance mutant GBA functional activity. Gaucher's disease is caused by mutation(s) in the GBA enzyme that reduce enzymatic function leading to disease. The candidate compound restores enzymatic activity, which has been shown to be an effective means of treating Gaucher's. Current Gaucher's treatments include recombinant replacement enzyme therapies, which require regular infusions with some patients developing allergic resistance to therapy. Sharp's candidate compound is an orally-available small molecule making it much more convenient for patients, and much more efficient to produce and distribute reliably.\"This is the first program from our platform to enter FDA-reportable studies and is a milestone for Sharp as it transitions to a clinical stage company,\" said Scott Sneddon, CEO/CSO of Sharp. \"The compounds show robust activity in animal models, and more importantly, in cells taken from Gaucher's patients containing several of the most common GBA mutations,\" he added. \"These compounds are also brain penetrant, leaving the prospect for treating CNS manifestations of Gaucher's, a market not effectively treated by existing therapies.\"The scientific data supporting the compounds will be presented at the GBA1 Conference in Montreal starting June 5, 2025 of which Sharp is also a meeting sponsor.About S...