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The U.S. National Center for Advancing Translational Sciences CHRISTOPHER P. AUSTIN, M.D. DIRECTOR, NCATS U.S. NATIONAL INSTITUTES OF HEALTH BIO INTERNATIONAL.

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Presentation on theme: "The U.S. National Center for Advancing Translational Sciences CHRISTOPHER P. AUSTIN, M.D. DIRECTOR, NCATS U.S. NATIONAL INSTITUTES OF HEALTH BIO INTERNATIONAL."— Presentation transcript:

1 The U.S. National Center for Advancing Translational Sciences CHRISTOPHER P. AUSTIN, M.D. DIRECTOR, NCATS U.S. NATIONAL INSTITUTES OF HEALTH BIO INTERNATIONAL CONVENTION JUNE 7, 2016

2 NCATS Mission To catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.

3 What is Translational Science? Translational Science is the field of investigation focused on understanding the scientific and operational principles underlying each step of the translational process. NCATS studies translation as a scientific and organizational problem.

4 NCATS Scientific Initiatives  Clinical Translational Science Clinical and Translational Science Awards program Rare Disease Clinical Research Network New Therapeutic Uses program  Preclinical Translational Science NCATS Chemical Genomics Center Therapeutics for Rare and Neglected Diseases program Bridging Interventional Development Gaps program  Re-engineering Translational Sciences Toxicology in the 21st Century Microphysiological Systems (Tissue Chip) program Office of Rare Diseases Research

5 Division of Preclinical Innovation Target Validation through Drug Development to First-in-Human Collaborative project team model  > 300 collaborations with investigators worldwide Each project customized to problem and development stage Each project is “dual use”  Solves specific block in translation  Provides insight of general utility to advance science of translation

6 Therapeutics for Rare/Neglected Dis (TRND) BrIDGs Stem Cell Technology Facility Tox21 (Systems Toxicology) RNAi Paradigm/Technology Development Repurposing Lead Optimization Preclinical Development Probe/Lead Development Target Validation Target FDA approval Clinical Trials I II III Project Entry Point Deliverables Repurposing Unvalidated target Validated target Lead compound Preclinical development candidate Genome-wide RNAi systems biology data Chemical genomics data Small molecule and siRNA research probes More efficient/faster/cheaper translation and therapeutic development Leads for therapeutic development Predictive in vitro toxicology profiles Approved drugs effective for new indications New drugs for untreatable diseases Novel clinical trial designs Drugs suitable for adoption for further development Assay Dev Assay, Chemistry Technologies Target assay DPI Program NCGC NCATS Division of Preclinical Innovation Stem cell tools/data

7 NCATS DPI Staff

8 CSC Developing Aldehyde Dehydrogenase Inhibitors Collaborator: Vasilis Vasiliou, Yale University Target: ALDH1A1 Therapeutic Scope: Cancer, Inflammation, Obesity, Development Objective: Identification and characterization of small molecule inhibitors of Aldehyde Dehydrogenase isoform 1A1 Chemico-Biological Interactions 202 (2013) 2–10 ALDH

9 Developing Aldehyde Dehydrogenase Inhibitors Developed two highly potent series of ALDH1A1 inhibitors, e.g., NCT-501, with excellent selectivity and ADME properties Demonstrated efficacy of candidate inhibitors in 3D-spheroid and organoid (cancer stem cell) models of Glioblastoma multiforme 1A11B13A1ALDH2 Permeability (PAMPA) Aqueous Solubility 40 nM>57 µM 164 (10 -6 cm/s)>60 µg/mL RLM (T 1/2 ) HLM (T 1/2 ) Caco-2: P (B-A) /P (A-B) Protein Binding 84 min> 2 h19.6/19.3 = 1.0269% ALDH inhibition (IC 50 ), selectivity, and early ADME properties of NCT-501

10 NCATS Partnership on Sickle Cell Disease  Collaborator: AesRx, LLC  Compound: Aes-103 (5-hydroxymethyl-2-furfural) Binds to sickle hemoglobin, increases O 2 affinity  TRND partnered with AesRx to de-risk Project initiation to patients in <12 mos Animal toxicology studies CMC (Chemistry, Manufacturing, and Controls) Regulatory: interactions with FDA, IND filing Ph Ia, Ib, IIa trials  Project acquired by Baxter

11 ncats.nih.gov/expertise

12 Learn More About NCATS Website: www.ncats.nih.govwww.ncats.nih.gov Facebook: facebook.com/ncats.nih.gov Twitter: twitter.com/ncats_nih_gov YouTube: youtube.com/user/ncatsmedia E-Newsletter: ncats.nih.gov/news-and- events/e-news/e-news.html Email us! info@ncats.nih.govinfo@ncats.nih.gov

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