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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.

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

Last updated on January 10, 2024