HemoShear is developing proprietary new treatments for rare inborn errors of metabolism. We are also utilizing our transformational models of complex diseases to accelerate our partners’ programs in NASH, gout and an undisclosed rare liver disease.
There are currently no effective treatments for many inborn errors of metabolism, which can lead to severe organ damage, developmental deficits and premature death.
“When they looked at my son’s blood work, they thought he drank nail polish remover or antifreeze. They accused me of poisoning my kid. They decided to admit him, and his toxicity kept going up.”
Propionic Acidemia (PA) is a rare inborn error of metabolism caused by an enzymatic defect that results in a build-up of harmful acids that can damage the heart, liver, brain, and bones. The disease can cause seizures, delays to normal development like walking and talking, and other health problems. Although usually diagnosed soon after birth, there is currently no medical treatment for PA. Instead, patients are treated by managing the symptoms and by following a very restrictive diet.
“We worry about the implications of not being diagnosed. What did all those decompensations do to his brain and where is he going to go?”
Methylmalonic acidemia (MMA) is a rare inborn error of metabolism caused by an enzymatic defect in metabolism that results in an abnormally high level of acid in the blood and body tissues. In the acute form of the disease, drowsiness, coma, and seizures may occur. Mental retardation is a long-term consequence. Although usually diagnosed soon after birth, there is currently no medical treatment for MMA. Instead, patients are treated by managing the symptoms and by following a very restrictive diet.
Maple syrup urine disease (MSUD) is a rare inborn error of metabolism caused by the deficiency of an enzyme required to metabolize the chemical "building blocks" of proteins which leads to the buildup of life-threatening toxins in the body. If left untreated, it can result in brain damage and death. The condition gets its name from the characteristic odor, reminiscent of maple syrup that can be detected in the urine and earwax. While some symptoms can be controlled by dietary restrictions, there are currently no effective treatments for MSUD.
Urea cycle disorders (UCD) are rare inborn errors of metabolism resulting in a deficiency of the enzymes in the urea cycle that are responsible for removing toxic ammonia from the bloodstream. Infants with a urea cycle disorder often appear normal initially but rapidly develop brain swelling and potentially irreversible brain damage, coma and death. The treatment of urea cycle disorders consists of dietary management to limit ammonia production in conjunction with medical interventions, medications and/or supplements to remove of ammonia from the bloodstream.
HemoShear’s exclusive partners are accelerating their drug discovery initiatives by leveraging our REVEAL-Tx™ disease modeling platform to identify and validate novel approaches to diseases.
Takeda Pharmaceutical Company has an exclusive partnership with HemoShear, which has generated several novel therapeutic drug targets for liver diseases, including nonalcoholic steatohepatitis (NASH)/fibrosis. 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 these novel biological targets demonstrate disease responses that are superior to established mechanisms of fibrosis currently being targeted by other companies in clinical trials. Under the agreement, HemoShear is eligible to receive milestone payments of potentially $470 million plus royalties, and Takeda will receive the right to license and commercialize any therapeutic that results from the partnership. More information can be found in the press release here.
HemoShear is also developing a new human tissue-based model of a rare liver disease using the company’s REVEAL-Tx™ Platform. This partnership will enable Takeda to interrogate their drug targets in the genetic background of human disease and accelerate the selection of lead candidates. “This new collaboration gives us the opportunity to build on [NASH] momentum and expand the reach of our combined expertise to a challenging, severe rare liver disease of high unmet need,” said Gareth Hicks, Ph.D., GI Drug Discovery Unit Head at Takeda. More information can be found in the press release here.
Horizon Therapeutics has entered into an exclusive collaboration with HemoShear to discover new drugs for gout. The partnership combines HemoShear’s REVEAL-Tx™ platform and drug discovery capabilities with Horizon’s rheumatology development and commercialization expertise. Under the terms of the agreement, HemoShear will receive upfront payments and R&D funding, and Horizon will receive exclusive access to the company’s platform to discover new therapeutics for gout. Successful development and commercialization of multiple therapies by Horizon will make HemoShear eligible to receive milestone payments of potentially more than $500 million plus royalties. More information can be found in the press release here.
HemoShear Therapeutics, Inc.
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Charlottesville, Virginia 22902