OUR DRUG DISCOVERY PROGRAMS
HemoShear Therapeutics is advancing drug discovery programs in a multi-target approach for rare metabolic disorders and non-alcoholic steatohepatitis (NASH).
INBORN ERRORS OF METABOLISM
HemoShear’s transformational models of complex diseases are accelerating the discovery of new treatments for several rare inborn errors of metabolism, including Propionic Acidemia (PA), Methylmalonic Acidemia (MMA), Maple Syrup Urine Disease (MSUD) and Urea Cycle Disorders (UCDs). These rare genetic disorders are caused by the deficiency of certain enzymes required to metabolize proteins, resulting in the rapid buildup of life-threatening toxins in the body. There are currently no effective treatments for these disorders, which can lead to significant disabilities, frequent hospitalizations, developmental delays and mortality in afflicted children.
HemoShear has recreated the human pathophysiology of these diseases using tissue from patients and our REVEAL-Tx™ disease modeling platform, which combines the power of dynamic human biology and computational science to generate deep understanding of complex diseases. Using these insights, our scientists are identifying and validating promising treatment targets to combat several inborn errors of metabolism.
In partnership with Takeda, HemoShear’s REVEAL-Tx™ platform has identified several promising new treatment targets for nonalcoholic steatohepatitis (NASH), a serious, chronic liver disease that impacts over 16 million people in the United States alone. These targets were shown to inhibit biological processes associated with inflammation and fibrosis that can lead to NASH, cirrhosis, and liver cancer. Analysis using REVEAL-Tx™ suggests that inhibition of certain biological targets identified by HemoShear show disease responses that are superior to established mechanisms of fibrosis currently being targeted by other companies in clinical trials.
There is currently no FDA approved therapy for NASH and liver fibrosis. Our Takeda partnership combines HemoShear’s novel therapeutic targets with Takeda’s drug discovery and development capabilities.