National Center for Advancing Translational Sciences (NCATS)

We are pleased to announce #RareDiseaseDay 2025 will take place on Feb. 27­­–28, 2025! This year we will co-host with the FDA. Stay tuned for more information about #RDDatFDANIH! https://go.nih.gov/ry9Wbby

As 2024 comes to a close, we're reflecting on the incredible growth of the NCATS SBIR/STTR programs. More and more small businesses are receiving funding to drive breakthroughs in translational science, with a focus on key areas, like preclinical drug discovery, health research informatics and implementation research. We're excited to see this momentum continue into 2025! Learn more about our research priorities, programs, and how to apply: https://go.nih.gov/uMlOl9t #NCATSsbir #STTR #TranslationalScience #Innovation #YearInReview

Attention #TranslationalScience enthusiasts: Today is #FundingFriday! NCATS offers a wide range of #FundingOpportunities. View them here: https://go.nih.gov/G6pdrVT

Scientists in NCATS’ Therapeutics for Rare and Neglected Diseases (#TRND) program helped develop the first #GeneTherapy to be delivered directly to the brain! The recently FDA-approved gene therapy, called Kebilidi, treats aromatic L-amino acid decarboxylase (AADC) deficiency –– a fatal genetic disease affecting the brain and central nervous system –– by restoring a missing AADC enzyme.   Read more about this study: https://go.nih.gov/pbXSoQM #RareDisease

Scientific and technological innovations are transforming the drug development lifecycle—from discovery to clinical trials to commercialization. These breakthroughs could unlock the potential of precision and individualized medicine, offering new ways to prevent and treat diseases. This #MIHealthSummit session explores emerging technologies that are reshaping drug development, reducing R&D costs, and enhancing the health ecosystem. Watch the recording here: https://lnkd.in/e2s78cTn Moderator Conor Hale, Senior Editor, Fierce Biotech Speakers Theresa LaVallee, Chief Development Officer, Coherus BioSciences Joni L Rutter, Director, National Center for Advancing Translational Sciences (NCATS), The National Institutes of Health Laurence S Sperling M.D., FACC, FAHA, FACP, Chief Medical Officer, Family Heart Foundation Peter Stein, Director, Office of New Drugs, FDA

The Rare Diseases Clinical Research Network (RDCRN) supports clinical studies on more than 280 #RareDiseases — enhancing collaboration, data sharing and patient involvement in research! It also works with patient advocacy groups to advance treatments for conditions that lack viable therapies. For more details, visit the #RDCRN page: https://go.nih.gov/ai5Z1hV

The Bridging Interventional Development Gaps (#BrIDGs) #NCATSIntramural program encourages partnerships and helps researchers advance promising therapeutics for #RareDiseases through late-stage preclinical development and toward #ClinicalTrial testing of Investigational New Drugs. Learn more about the program: https://go.nih.gov/GR302cK

Mark your calendars for #RareDiseaseDay! 📅 We’re partnering with FDA for a two-day hybrid #RDDatFDANIH event on Feb. 27–28, 2025! Learn about #RareDiseases, scientific challenges and opportunities to advance research for new treatments. Visit the website to get event details! https://go.nih.gov/ry9Wbby

💡 EATRIS is co-hosting a free workshop in Berlin with National Center for Advancing Translational Sciences (NCATS), EU-OPENSCREEN, and Nuvisan CDD Holding GmbH. Join the "Assay Guidance Workshop for High-Throughput Screening and Lead Discovery" event on 9-10 April 2025. 💊 This in-depth workshop offers cutting-edge insights and practical tools for developing robust, reproducible assays in drug discovery and high-throughput screening. Ideal for researchers aiming to improve translational potential in #DrugDiscovery. Stay a bit longer for Nuvisan and EU-OPENSCREEN facilities tours in Berlin on 11 April. 👉 Find out more here: https://lnkd.in/dFqEx7fR

NCATS’ Therapeutics for Rare and Neglected Diseases (#TRND) program helped develop a recently FDA-approved gene therapy — Kebilidi — to treat aromatic L-amino acid decarboxylase (AADC) deficiency, a genetic #RareDisease affecting the brain and central nervous system. Kebilidi is the first #GeneTherapy to be delivered directly to the brain! Learn more: https://go.nih.gov/pbXSoQM