Our Transformational Drug Discovery Platform

 

A fundamental flaw driving the very low success in drug R&D is that existing models do not reliably represent human biological context.  We have developed a transformational platform, REVEAL-Tx™, which applies principles of physiological blood flow to tissue from patients to recapitulate disease and allow drug candidates to be studied at human concentrations.  REVEAL-Tx™ provides us with unprecedented insights into complex pathophysiological pathways by replicating human disease with great accuracy. 

Our human disease models, in combination with our advanced proprietary computational biology tools, reduce risk of failure by enabling us to deeply interrogate disease pathways, test hypotheses and select meaningful targets in physiologically accurate disease conditions.   REVEAL-Tx™ gives us confidence in the novel targets we have identified for the treatment of metabolic rare diseases.

 

We strongly believe that our focus on deeper understanding of human disease will materially increase success rates of new drug candidates as well as accelerate discovery and development of promising drug candidates for children with rare metabolic diseases.