Tackling Rare Metabolic Diseases

HemoShear’s transformational models of complex diseases are accelerating the discovery of new treatments for several rare inborn errors of metabolism. There are currently no effective treatments for many of these tragic disorders, which can lead to significant developmental delays, frequent hospitalizations, and mortality.

Propionic Acidemia (PA) is a rare inborn error of metabolism characterized by deficiency of an enzyme involved in the breakdown of the chemical "building blocks" of certain proteins. Symptoms most commonly become apparent during the first weeks of life and may include poor feeding, vomiting, listlessness, and seizures. Although usually diagnosed soon after birth or through newborn screening, there is currently no medical treatment for PA. Instead, patients are treated by managing the symptoms and by following a very restrictive diet.

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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.

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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.

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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.

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The HemoShear Advantage

The biopharmaceutical industry’s drug development success is impeded by a lack of physiological models that reflect the complexity of disease. HemoShear’s proprietary REVEAL-Tx™ platform combines biological and computational models of human disease to accelerate discovery of novel targets and successful new drug treatments.

Advanced Disease ModelsAdvanced Disease Models Computational InsightComputational Insight Accelerated DiscoveryAccelerated Discovery
Advanced Disease Models

We have developed transformational physiological models of human diseases by applying principles of physiological blood flow to tissue from patients to recapitulate their disease. Our models enable valuable insights into complex disease pathways and drug responses in a more meaningful human-relevant context.

Computational Insight

We leverage powerful computational science to extract signaling pathways from our disease models and identify novel therapeutic targets for treating diseases. We then assess our computational target hypotheses in our biological disease models.

Accelerated Discovery

The REVEAL-Tx™ platform enables HemoShear and our partners to gain unprecedented insight into the underlying mechanisms of disease, rapidly translate those discoveries into drug candidates, and reduce risk of failure by reliably assessing which drug candidates will be safer and more effectively treat patients.

Disease Models

HemoShear’s platform has been used to recreate nearly 20 human diseases related to liver, vascular and tumor biology, including those driving our current pipeline in several rare metabolic disorders and partnerships with major biopharma.

Liver System Liver System

HemoShear has created a biologically responsive human liver system that has yielded a number of novel targets that modulate processes associated with children’s inborn errors of metabolism, and inflammation and fibrosis that can lead to nonalcoholic steatohepatitis (NASH), cirrhosis, and liver cancer. Through our partnership with Takeda, we have studied more than 20 NASH drugs in development across the industry to identify better targets and therapeutics.

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Tumor System Tumor System

HemoShear’s tumor models, currently in development, replicate the complex nature and behavior of a tumor, and can be used to interrogate the tumor microenvironment and make novel discoveries in how cancer cells survive, grow, and become resistant to therapy. In addition, a patient’s tumor tissue could be used to create an ‘avatar’ of the tumor in the HemoShear system to assess the effectiveness of different drugs against the patient’s tumor – an important step towards personalized cancer treatment. We look forward to forging partnerships with companies who want to apply our platform to accelerate successful cancer drug discovery and ultimately develop more effective treatments for patients.

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Vascular System Vascular System

Using human tissue and regional hemodynamics, HemoShear has developed and validated numerous models that mimic the biology and physiology of the blood vessel wall throughout the human vascular system. We have collaborated with a number of partners to uncover mechanisms of action and differentiate drug compounds.

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Contact

info@hemoshear.com

501 Locust Avenue
Suite 301
Charlottesville, Virginia 22902