Skip to main content
HHS Logo U.S. Department of Health & Human Services Divider arrow NIH logo National Institutes of Health Alt desc
TRND Pompe image viewed on tablet

NCATS Collaboration Enables Gene Therapy to Move to Clinical Trial

NCATS scientists and NIH-funded researchers at Duke University collaborate to advance a gene therapy for Pompe disease into clinical testing for the first time.

Work with Us

NCATS pre-clinical programs and resources focus on key obstacles and inefficiencies in the translational process, overcoming bottlenecks that slow the development of new treatments for patients.

Learn more about how to access NCATS programs and expertise.

Improving the Drug Development Process

Creating and testing innovative methods to improve and accelerate the drug development process and find new treatments for patients faster. Learn more.

Find NCATS Programs & InitiativesAccess NCATS Expertise & Resources

Target Identification & Validation

Developing new approaches and technologies to identify and validate potential drug targets in a more efficient and predictable way.

Assay Development & Screening

Enhancing testing systems to assess the effects of chemical compounds on cellular, molecular or biochemical processes of interest.

Probe Development & Lead Optimization

Creating probes to investigate biological processes and optimizing them to become potential drug candidates.

Core Technologies

Using state-of-the-art resources to enable the ongoing operation of all NCATS translational research activities.

Translational Science Spectrum

Learn more about the science of translation.

Innovation in the Drug Development Process

NCATS strives to create and test innovative methods and technologies that will enhance the development, testing and implementation of new drugs, diagnostics and other interventions. By improving the process by which these interventions are developed, NCATS aims to make translational science more efficient, less expensive and less risky. In this way, NCATS complements the work of the private sector and other NIH Institutes and Centers.