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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.

Rare Disease Day at NIH 2026: Paving the Way to a Brighter Future for All Americans

March 31, 2026 - NCATS News

  • Our Impact on Rare Diseases

The rare diseases community came together at the NIH Natcher Conference Center and virtually to celebrate Rare Disease Day at NIH, highlighting inspiring stories, impactful research advances, and new possibilities for the future.

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)

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)

New Initiative Aims to Revive Stalled Rare Disease Therapies

February 24, 2026 - Media Coverage

  • Our Impact on Rare Diseases

More Than 30 Million Americans Living With Rare Diseases

February 23, 2026 - Grantee/Partner News

  • Clinical and Translational Science Awards (CTSA) Program

Last updated on September 29, 2026