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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.

Cures 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.

Last updated on March 31, 2026