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)
Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment
May 15, 2025 - Grantee/Partner News
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
Infant With Rare Incurable Disease Becomes First to Receive Successful Personalized Gene Therapy
May 15, 2025 - Media Coverage
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
Researchers Identify Pathway Responsible for Calciphylaxis, a Rare and Serious Condition
April 23, 2025 - Grantee/Partner News
- Clinical and Translational Science Awards (CTSA) Program
A Combination of Tests Shows Learning Capabilities of Nonverbal Children with Rare Brain Disorder
February 25, 2025 - NCATS News
- Our Impact on Rare Diseases
- Rare Diseases Clinical Research Network (RDCRN)
NCATS-funded scientists found that a pair of tests can measure nonverbal learning skills and show the cognitive abilities of children with Aicardi-Goutières syndrome. The tests could improve the support and therapy doctors provide.
AI Tool Helps Find Life-Saving Medicine for Rare Disease
February 5, 2025 - Grantee/Partner News
- Biomedical Data Translator (Translator)
- Our Impact on Rare Diseases
New Path for a Gene Therapy Trial at NIH for a Rare Metabolic Disease
November 19, 2024 - NCATS News
- Bespoke Gene Therapy Consortium (BGTC)
- Our Impact on Clinical Trials
- Our Impact on Drug Discovery and Development
- Our Impact on Rare Diseases
- Platform Vector Gene Therapy (PaVe-GT)
Scientists at NCATS and other NIH institutes and centers are teaming up to advance a gene therapy treatment for MMA, a rare and potentially life-threatening genetic disorder.