Changing Lives for Those with Genetic Disease
Turning Diagnosis into Hope
Patient Science
Many genetic diseases involve the loss of function of one or both copies of a gene leading to less functional protein being made within cells. This loss of function is what causes the imbalance in genetic disease. At Sharp we create small molecule drugs (the kind that can be taken as a pill) that restore the function of these mutated proteins to correct this fundamental imbalance. We make drugs that treat genetic disease at its core cause, turning diagnosis into hope.
Our Priorities
We focus in areas where our technologies provide the most benefit for patients. This means diseases involving loss of function mutations and haploinsufficiency. We also focus in areas where a small molecule pill would lead to improved patient quality of life (and possibly better control of disease).
Our Company
Sharp is a dedicated group of scientists, clinicians, data scientists and genetic disease experts with decades of experience bringing therapies to patients. The team is supported by investors that share our mission of changing lives for patients.