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.
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.
Read ArticleSharing Science at the NIH Research Festival
October 22, 2024 - Grantee/Partner News
- Our Impact on Rare Diseases
- Platform Vector Gene Therapy (PaVe-GT)
Propelling Cures With Genetics Research: Interview With Joni L. Rutter, PhD, Director of the National Center for Advancing Translational Sciences at NIH
December 14, 2023 - Grantee/Partner News
- 3-D Tissue Bioprinting
- Bespoke Gene Therapy Consortium (BGTC)
- Clinical and Translational Science Awards (CTSA) Program
- New Therapeutic Uses (NTU)
- Platform Vector Gene Therapy (PaVe-GT)
- Rare Diseases Clinical Research Network (RDCRN)
- Somatic Cell Genome Editing (SCGE)
- Therapeutics for Rare and Neglected Diseases (TRND)
- Tissue Chip for Drug Screening
NIH Gene Therapy Team Reveals Its Path to FDA Orphan Drug and Rare Pediatric Disease Designations
March 29, 2023 - NCATS News
- Our Impact on Rare Diseases
- Our Impact on Research Operations
- Platform Vector Gene Therapy (PaVe-GT)
When NCATS received an Orphan Drug Designation (ODD) from the U.S.
Developers of Gene Therapies Can Utilize FDA Guidance to Prepare ODD or RPDD Applications
March 27, 2023 - Media Coverage
- Platform Vector Gene Therapy (PaVe-GT)
NIH, FDA and 15 Private Organizations Join Forces to Increase Effective Gene Therapies for Rare Diseases
October 28, 2021 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
- Platform Vector Gene Therapy (PaVe-GT)
NIH, FDA and 15 Private Organizations Join Forces to Increase Effective Gene Therapies for Rare Diseases
October 27, 2021 - NCATS News
- Bespoke Gene Therapy Consortium (BGTC)
- Platform Vector Gene Therapy (PaVe-GT)
NIH, FDA and 15 private organizations partnered to create the Bespoke Gene Therapy Consortium to speed development of FDA-approved gene therapies for rare diseases.
PaVe-GT: Collaborative NIH Effort Aimed at Creating a Gene Therapy Playbook, Making Rare Disease Treatments More Accessible
November 30, 2020 - NCATS News
- Our Impact on Rare Diseases
- Platform Vector Gene Therapy (PaVe-GT)
The Platform Vector Gene Therapy (PaVe-GT) initiative aims to make gene therapy development and clinical testing more streamlined and less expensive — and potentially more accessible to millions of