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)
New Initiative Aims to Revive Stalled Rare Disease Therapies
February 24, 2026 - Media Coverage
- Our Impact on Rare Diseases
Children’s Hospital of Philadelphia Marks One-Year Anniversary of World’s First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease
February 24, 2026 - Grantee/Partner News
- Somatic Cell Genome Editing (SCGE)
More Than 30 Million Americans Living With Rare Diseases
February 23, 2026 - Grantee/Partner News
- Clinical and Translational Science Awards (CTSA) Program
Rare Diseases Clinical Research Network Awardees Expand the Reach of NIH Impact
February 5, 2026 - NCATS News
- Rare Diseases Clinical Research Network (RDCRN)
NIH awarded funding to 10 new and five continuing Rare Diseases Clinical Research Network (RDCRN) consortia to help address challenges in rare diseases treatment development.
UNC Researchers Awarded $7.3 Million to Advance Research on Rare Bronchiectatic Diseases
February 4, 2026 - Grantee/Partner News
- Rare Diseases Clinical Research Network (RDCRN)
A Rare Respiratory Disease May Be More Prevalent in Quebec
November 27, 2025 - Grantee/Partner News
- Rare Diseases Clinical Research Network (RDCRN)