OUR DRUG DISCOVERY PIPELINE
HemoShear Therapeutics is advancing drug discovery programs in a multi-target approach for rare metabolic disorders and non-alcoholic steatohepatitis (NASH).
OUR METABOLIC RARE DISEASE DRUG DISCOVERY PROGRAM
In late 2014, we initiated a collaboration with Children’s National Medical Center, the largest clinic in the world for children with rare diseases, to target specific children’s diseases and facilitate obtaining surgically removed livers from children with metabolic disorders who are receiving transplants. We are currently focusing on a family of disorders called organic acidemias, which have known biological mutations and strong clinical hypotheses for novel drug targets. No effective drug therapies exist for children with these disorders and to our knowledge little progress is being made to discover new drug treatments. HemoShear and Children’s National have jointly selected a specific sub-group of organic acidemias that share similar biological pathways and potential therapeutic approaches, including propionic acidemia, methylmalonic acidemia, and two additional metabolic disorders.
OUR NASH DISCOVERY PROGRAM
We are additionally focusing efforts on drug discovery in Nonalcoholic Steatohepatitis (NASH), a serious, chronic, progressive liver disease that is estimated to impact over 16 million people in the U.S. alone. The disease is one of the fastest-growing global diseases driven by poor diet, obesity, type 2 diabetes, and metabolic syndrome, characterized by inflammation, dysregulation of the body’s metabolism, and excessive fat accumulation in the liver that can progress to fibrosis, cirrhosis, and eventually liver failure.
With REVEAL-Tx™, we believe that HemoShear has the only human tissue-based platform that can interrogate this disease at a fundamental biological level and improve our probability of successful drug discovery. Currently, we are studying a wide range of drug targets being developed by other companies to identify treatment gaps and opportunities, as well as our own novel target hypotheses. Our collaborator is NASH expert Dr. Arun Sanyal, the Charles Caravati Professor and chair of the Division of Gastroenterology, Hepatology and Nutrition, in the Department of Internal Medicine at Virginia Commonwealth University.