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.

 

 

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