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.
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.
Gene-Targeted Therapies
We are developing generalizable approaches to advance gene-targeted therapies for many rare diseases.
MMA-101
This project will test a gene therapy as a potential treatment for a rare genetic disorder.
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.
More About Multidisciplinary Machine-Assisted, Genomic Analysis
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.
More About the PaVe-GT
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.
More About the RDCRN
Somatic Cell Genome Editing [NIH Common Fund Programs]
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.
More About Somatic Cell Genome Editing