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 Article
New FDA Designations May Jumpstart Gene Therapy Development
October 18, 2024 - NCATS News
- Bespoke Gene Therapy Consortium (BGTC)
- Platform Vector Gene Therapy (PaVe-GT)
New FDA designations granted to NCATS for rare disease therapies provide financial incentives for companies to further develop treatments.
Read ArticleEx Vivo vs In Vivo Gene Therapy
February 2, 2023 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
‘I Thought I Had Woken Up to an Earthquake’: What We Heard This Week
January 22, 2023 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
NIH Project Aims to Make Gene Therapy ‘Playbook’ Public
January 18, 2023 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
As more families raise money for and partner with researchers to develop tailored, personalized treatments for rare diseases, some NIH agencies are trying to help by streamlining the process.
The Bespoke Gene Therapy Consortium
November 30, 2022 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
Avrobio Receives Orphan Drug Designation for Hunter Syndrome Gene Therapy
July 14, 2022 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
The Sino-American Pharmaceutical Professionals Association-Greater Philadelphia (SAPA-GP) Hosted the Inaugural @Philly Cell and Gene Therapy Annual Conference
July 8, 2022 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)