Read our latest research news or follow coverage of our translational science 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.

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Spectradyne: SBIR Funding Spurs Advances in Nanoparticle Technology

November 19, 2024 - NCATS News

Small Business (SBIR)

NCATS small business innovation research funding helped Spectradyne, LLC, commercialize an improved nanoparticle analyzer critical to research and drug development.

Easing the Burden of Rare Diseases Through Translational Science

November 12, 2024 - NCATS News

Our Impact on Rare Diseases

NCATS Director Joni Rutter discusses how NCATS is addressing scientific and operational roadblocks that slow progress for rare diseases research and treatments.

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.

Explore our research labs, projects and collaborations that are advancing translational science. Our largest program is the Clinical and Translational Science Awards (CTSA) Program.

Enhance your knowledge of translational science through programs, activities and resources offered by NCATS and our partner institutions.

Whether you're a researcher, an aspiring translational scientist or an interested member of the community, find information and materials important to you.

For Researchers Learn about our latest opportunities for funding and collaboration and connect with our staff.

Easing the Burden of Rare Diseases Through Translational Science

November 12, 2024

NCATS Director Joni Rutter kicks off a new message series on progress and priorities in rare diseases research across the translational pipeline.

Joni L. Rutter, Ph.D., is the director of NCATS at NIH. Rutter oversees the planning and execution of NCATS complex, multifaceted programs.

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