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 ArticleChildren’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)
Medical Miracles at Penn Medicine: Breakthrough With Customized CRISPR Treatment for Patient With CPS1
December 16, 2025 - Grantee/Partner News
- Somatic Cell Genome Editing (SCGE)
Mapping ‘Dark’ Regions of the Genome Illuminates How Cells Respond to Their Environment
September 25, 2025 - Grantee/Partner News
- Somatic Cell Genome Editing (SCGE)
Research Spotlight: Laying the Foundation for Gene Editing for Inherited Progressive Deafness in Adults, DFNA41
August 14, 2025 - Grantee/Partner News
- Somatic Cell Genome Editing (SCGE)
Gene Editing Reverses Symptoms of Rare Brain Disorder in Mice
July 22, 2025 - Grantee/Partner News
- Somatic Cell Genome Editing (SCGE)
Genome Editing Corrected Rare Brain Mutations in Mice. Could It Help Fight Neurological Diseases?
July 21, 2025 - Grantee/Partner News
- Somatic Cell Genome Editing (SCGE)
A Gene Editing First Augurs an Era of Bespoke Therapies
June 12, 2025 - Grantee/Partner News
- Bespoke Gene Therapy Consortium (BGTC)