News
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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.
AMP® BGTC Announces FDA Clearance of Investigational New Drug Application for Gene Therapy Targeting Multiple Sulfatase Deficiency
July 6, 2026 - Grantee/Partner News
- Bespoke Gene Therapy Consortium (BGTC)
The FDA cleared the Investigational New Drug (IND) application for a gene therapy designed to treat children with multiple sulfatase deficiency (MSD), an ultra-rare disease.
Read ArticleA Gene Editing First Augurs an Era of Bespoke Therapies
June 12, 2025 - Grantee/Partner News
- Bespoke Gene Therapy Consortium (BGTC)
P.J. Brooks, Ph.D., on Working to Bring Gene Therapy Development to More Neuromuscular Disorders
March 5, 2024 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
- Platform Vector Gene Therapy (PaVe-GT)