NCATS conducts and supports collaborative research across the pre-clinical phase of the translational science spectrum to develop new methods, tools and technologies to catalyze pre-clinical research and make it more efficient. In doing so, the Center effectively “de-risks” novel and promising therapeutic and diagnostic discoveries and enables the translation of these innovations to a state where they can attract outside investment for further clinical development. In this way, NCATS serves as an adaptor between academia, nonprofit research institutions, and private sector companies, each with complementary resources and skills to bring new medical interventions to patients faster.
Importantly, NCATS selects and carries out collaborative research projects to not only jump-start new therapeutic or diagnostic development, but also to help uncover general principles of translational science, or to test a new technology or approach. In addition, NCATS creates innovative methods to overcome translational bottlenecks in areas including therapeutic/diagnostic target identification, assay development, medicinal chemistry, drug screening, efficacy and toxicology testing, and implementation of new medical interventions.
NCATS broadly disseminates the innovative tools and technologies it develops to enable all participants in the biomedical science ecosystem to conduct pre-clinical research more efficiently and effectively. By improving our understanding of the principles underlying successful translational science, NCATS makes translational research more likely to deliver upon the ultimate goal of patient benefit.
- Accelerate therapeutic target identification and validation through advanced gene editing technologies and screening with newly developed small molecule probes.
- Develop innovative biochemical and cellular assay platforms and chemical library screening approaches in the context of disease biology to overcome translational barriers in therapeutic development for underserved diseases.
- Use NCATS expertise in medicinal chemistry and informatics to develop and demonstrate innovations in hit-to-lead optimization of therapeutic candidates.
- Collaborate with academic, nonprofit and industry partners in drug synthesis, formulation, pharmacokinetics and toxicology to support initial regulatory review of interventions in development.
- Develop and integrate new models of human biology to better understand disease physiology and predict candidate drug safety, toxicity and efficacy.
- Develop and disseminate more efficient and effective approaches to predict how chemicals may affect human health, and organize the data collected from such approaches into predictive computational models for toxicity assessment.
- Provide collaborative access to NCATS’ state-of-the-art resources and expertise in automation, compound management, analytical chemistry and informatics, among others.
- Make research data, tools and technologies produced by NCATS broadly accessible and available to the entire translational community.