Sharp Therapeutics Corp. has engaged global contract research organization Rho Inc. to support the preparation and submission of its Investigational New Drug Application (IND) to the U.S. Food and Drug Administration for compound '901, a potential oral therapy for Gaucher disease. The Pittsburgh-based biotechnology company announced this strategic partnership as it prepares to transition from preclinical to clinical-stage development.
Novel Approach to Lysosomal Storage Disorder
Gaucher disease is a genetic disorder caused by deficiency in the enzyme glucocerebrosidase (GBA1), making it the most common lysosomal storage disorder. Without this enzyme, glucocerebroside lipid accumulates within lysosomes of certain cells, particularly macrophages, leading to enlarged and dysfunctional "Gaucher cells." This accumulation primarily affects the spleen, liver, bone marrow, and sometimes the nervous system, causing organ enlargement and characteristic disease symptoms.
Sharp's '901 compound represents a differentiated small-molecule approach designed to restore GBA1 enzyme function. Preclinical studies demonstrate that the compound increases GBA1 activity for numerous mutations in Gaucher patient-derived cells and enhances substrate turnover in rodent models of the disease. The therapy is orally available and CNS-penetrant, offering potential advantages over current treatment approaches.
Clinical Development Timeline
"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, Chief Executive Officer of Sharp Therapeutics. The company intends to meet with the FDA regarding its Phase I clinical plan and file for orphan drug designation this year, with IND-enabling studies planned to commence before the end of 2025.
The '901 compound targets all types of Gaucher disease, with potential application extending to GBA-associated Parkinson's disease. This broad therapeutic scope reflects the compound's ability to address both peripheral and central nervous system manifestations of GBA1 deficiency. Scientific data supporting the '901 series was presented at the GBA1 Conference in Montreal on June 5, 2025.
Broader Pipeline Development
Sharp Therapeutics is developing a pipeline centered around genetic diseases including Gaucher disease, Niemann-Pick disease type C, and familial frontotemporal dementia. The company's discovery platform combines novel high-throughput screening technologies with computationally optimized compound libraries based on cellular trafficking defects and allosteric protein activation.
"Our mission is to develop pill-based medicines that restore function in defective proteins and meaningfully improve the lives of patients with genetic diseases," Sneddon explained. The company aims to deliver transformative therapies through conventional oral medications, potentially offering improved convenience and accessibility compared to existing treatments.
Partnership with Rho Inc.
Rho Inc., a global contract research organization headquartered in Research Triangle Park, North Carolina, brings extensive experience in drug development services from program strategy through clinical trials and marketing applications. Since 1984, Rho has partnered with pharmaceutical, biotechnology, and medical device companies as well as academic and government organizations.
The partnership positions Sharp to efficiently advance '901 through regulatory processes and into clinical development. "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," Sneddon noted.
Sharp plans to present pipeline and program updates at the World Orphan Drug Congress 2025, taking place October 27-29 in Amsterdam, where the company will highlight the status of its clinical targets and development programs.
