Using our powerful REVEAL-TxTM drug discovery platform, our scientists are identifying and validating promising approaches to develop novel treatments aiming to extend and improve the quality of life of patients with rare diseases. In our most advanced program, the company is conducting a phase 2 clinical trial of HST5040, an oral small molecule investigational drug for the treatment of patients with methylmalonic acidemia (MMA) and propionic acidemia (PA). These genetic disorders are caused by deficiencies of certain metabolic enzymes that lead to buildup of life-threatening toxins.
HemoShear’s REVEAL-TxTM drug discovery platform combines the power of biological and computational human disease models to gain deep insights into disease drivers, uncover novel targets and translate these discoveries into promising drug candidates.
Click below to learn more about HemoShear's platform.
HemoShear has built an extensive network for obtaining explanted healthy and diseased tissue under IRB-approved protocols for our drug discovery research. We partner with leading transplant centers within the organ donor system in real time to obtain tissue for targeted rare diseases. Our scientists isolate viable cells that are utilized for understanding and recapitulating diseases in our proprietary disease modeling system.
We leverage powerful computational techniques to incorporate data from our disease models and other sources to identify novel therapeutic targets. We then validate the computationally predicted targets in our disease models.
The HemoShear drug discovery team is comprised of more than 30 scientists. These experts apply their years of experience across biology, pharmacology, biomedical engineering, computational science, and medicinal chemistry to validate targets and characterize biological mechanisms to ultimately design and optimize drug candidates to move rapidly into the clinic.
We have developed translational models of human diseases by applying principles of physiological blood flow to human tissue obtained through our procurement network to recapitulate diseases for our programs. Our models enable valuable insights into complex disease pathways and allow for interrogation of drug responses in a more meaningful human-relevant context.
The REVEAL-Tx™ drug discovery platform has been integral to our success in identifying and validating novel drug targets for nonalcoholic steatohepatitis (NASH) and gout through collaborations with Takeda Pharmaceutical Company and Horizon Therapeutics, respectively.
HemoShear Therapeutics, Inc.
501 Locust Avenue
Suite 301
Charlottesville, Virginia 22902