News
Explore the latest stories and publications from NCATS and our partners, see upcoming events, or explore the variety of resources we provide to better understand translational science and our work.
News
Read the latest news from NCATS and its collaborators whose research is supported through the Center’s programs, or follow coverage of NCATS' translational research activities through national and local media.
Transparency and Early Planning Are Key in Increasing Efficiency of Gene Therapy Development for Rare Diseases
February 26, 2026 - NCATS News
- Platform Vector Gene Therapy (PaVe-GT)
PaVe-GT scientists underscore strategy, framework and early interaction with the FDA to align preclinical and clinical efforts in AAV gene therapy translation.
Read ArticleCures Within Reach Selected Three Clinical Trials in Rare/Ultra-Rare Diseases to Validate AI Predictions
February 25, 2026 - Grantee/Partner News
- Biomedical Data Translator (Translator)
Using AI to Improve Detection of Rare Diseases
July 16, 2024 - Grantee/Partner News
- Clinical and Translational Science Awards (CTSA) Program
At a Symposium on Rare Diseases, Smiles Were in Abundance
March 28, 2024 - Grantee/Partner News
- Our Impact on Rare Diseases
Pushing for Better Clinical Trial Design for Rare Diseases
October 17, 2023 - Grantee/Partner News
Genetics Experts Support Adding Hundreds of Treatable Rare Diseases to Newborn Screening
May 9, 2023 - NCATS News
- Clinical and Translational Science Awards (CTSA) Program
A study found that 88% of experts surveyed on rare diseases agreed that DNA testing to reveal treatable genetic disorders should be available for all infants. This additional screening could allow lifesaving treatments to be delivered to babies sooner.
Gene Therapy: The Future Made Present for Addressing Rare Diseases
April 18, 2023 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
NIH, FDA and 15 Private Organizations Join Forces to Increase Effective Gene Therapies for Rare Diseases
October 27, 2021 - NCATS News
- Bespoke Gene Therapy Consortium (BGTC)
- Platform Vector Gene Therapy (PaVe-GT)
NIH, FDA and 15 private organizations partnered to create the Bespoke Gene Therapy Consortium to speed development of FDA-approved gene therapies for rare diseases. The effort is co-led by NCATS and managed by FNIH.