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.
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 and Translational Science Awards (CTSA) Program
This program funds a network of more than 60 leading medical institutions to improve the health of individuals and communities through research, resources and training.
Clinical Trial Readiness for Rare Diseases, Disorders and Sy…
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.
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.
The Helping to End Addiction Long-term® Initiative, or NIH H…
As part of the NIH HEAL Initiative, we are applying our translational science approaches to accelerate the development of new treatments for opioid misuse, addiction and pain management.
Tissue Chip for Drug Screening
This program funds the development and testing of models built from human cells for predicting drug safety and toxicity before clinical testing in people.