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HemoShear and CNHS Accelerating Rare Disease Drug Discovery

Wednesday, November 19, 2014  
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HemoShear and Children's National Health System (CNHS) together presented at the 6th annual Partnering for Cures conference held November 16-18, 2014 in New York City. Marshall Summar, MD, Division Chief of Genetics and Metabolism at CNHS, and Brian Wamhoff, PhD, Vice President of R&D and Co-founder of HemoShear, introduced a new paradigm for enabling discovery of new rare disease treatments in less time and with higher success rates.

Drs. Summar and Wamhoff described how Children's knowledge of disease genetics and access to patients, in combination with HemoShear's translational rare disease tissue systems, have the potential to uncover the biology of liver-focused rare diseases and to predict patient responses to new drug candidates. This new paradigm is a major advancement over traditional drug R&D approaches, such as animal models, because they do not mimic human rare disease biology, leading to high failure rates and high costs for very small patient populations.

"At Children's National, we treat more patients with rare diseases than nearly anywhere else in the world," said Dr. Summar. "Because we have few or no available drug therapies for most of the 7,000 rare genetic conditions, we are forced to treat the patients symptomatically, day to day, without addressing the underlying problem. For our metabolic rare disease patients, there simply are few reliable drugs. For many, a liver transplant is often the last therapeutic alternative. Even then our patients' lifespans can be tragically short."

"Our ability to accurately recreate liver rare diseases will be a major step forward in accelerating discovery and development of new treatments," said Dr. Wamhoff. "With the HemoShear platform, we expect to be able to unmask meaningful mechanisms behind these diseases, identify new targets and new drug candidates, and accelerate translation of optimal candidates into the clinic for children in need."

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