First-Choice Medicines for Hereditary Disorders
Combining high-speed experimentation and modern computational methods with the accelerated clinical development path available in genetic disease to provide first-choice therapies for patients, providers and the healthcare system.
Lead Programs
Sharp targets its discovery engine (affectionately, “Disco”) to three hereditary disorders with high unmet need: Gaucher’s Disease, Familial Frontotemporal Dementia and Niemann Pick Type-C. We expect to nominate our first clinical candidate compound from among these programs in Q2 of 2025 with patient testing to begin in 2026 with early-readout efficacy in that year.
The Discovery Engine
By combining industry-scale automated experimentation with advanced molecular-physics and mathematical modeling we created the Disco(tm) platform to rapidly and predictably discover small molecules that can correct the protein defects in hereditary disorders. Delivering small molecule therpies gives patients a first-choice oral medicine for the first time in genetic disease treatment.
Connecting Small Molecule Therapies to Rapid Clinical Development
Traditionally, pharmaceutical clinical development has been extremely costly, slow, and had a low probability of success. Sharp’s focus on hereditary diseases (where the cause of the disease is directly treated) allows our small molecule therapies to take advantage of faster, less expensive trials with much greater certainty of clinical success. This provides a faster, much more cost-effective path to deliver medicines that patients will choose first over biologics treatment (i.e recombinant proteins or gene therapies, the traditional approaches in these areas)