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Research Activities

Our programs, projects and other research activities tackle ongoing challenges in translational research so that new treatments and other health solutions can reach people faster.

Research Activities at NCATS

Through our research activities, we overcome roadblocks that slow progress on developing treatments for many different diseases and that can lead to inequities in clinical outcomes. Explore the range of initiatives we support at NCATS and beyond.

Learn About NCATS Research Topics

Bespoke Gene Therapy Consortium (BGTC)

We help lead this public-private partnership program that focuses on developing platforms and standards to speed the development and delivery of gene therapies for rare diseases.

Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes (CTR) Grants Program

Through this program, we offer grants for projects focused on collecting data needed to advance promising therapies and diagnostics for rare diseases toward clinical trials.

Multidisciplinary Machine-Assisted, Genomic Analysis and Clinical Approaches to Shortening the Rare Diseases Diagnostic Odyssey

This grant program funds the development of innovative approaches for reducing the time it takes to accurately diagnose rare diseases.

Platform Vector Gene Therapy (PaVe-GT)

Our pilot project is testing the feasibility of increasing the efficiency of gene therapy clinical trials by using the same gene delivery system and manufacturing methods for multiple gene therapies.

Rare Diseases Clinical Research Network (RDCRN)

We oversee this NIH-wide grant program that supports medical research on over 200 rare diseases through clinical studies, including collaborations, study enrollment and data sharing.

Somatic Cell Genome Editing

We help lead this NIH Common Fund program that aims to create and make available high-quality tools for safe and effective genome editing to decrease the time and cost of creating new therapies.

Therapeutics for Rare and Neglected Diseases (TRND)

Our experts help move new treatments for rare and neglected diseases through the later stages of drug development by collaborating with external scientists and companies.

Last updated on November 8, 2023