National Center for Advancing Translational Sciences (NCATS)

After studying nearly 60,000 electronic health records stored in the #N3C database, researchers found that a person’s health care service use 37 days before a person got #COVID19 was a strong predictor of whether they will get #LongCOVID. Read more: https://go.nih.gov/TKKlRw3

“Developing a platform that enables the discovery and characterization of potential treatments for Gaucher and Parkinson’s is the result of more than a decade of collaborative work,” — NCATS’ Mark Henderson, Ph.D. Explore this newly developed process that could be applied to other conditions: https://go.nih.gov/gG5QYdS

Celebrating Women Leaders in Biotech This #WomensSmallBusinessMonth! This month, we celebrate the remarkable women entrepreneurs shaping the future. From needing a male relative to co-sign for loans in 1988 to generating $1.9 trillion in revenue by 2019, women-owned businesses have shattered barriers and redefined success. We're inspired by leaders like Dr. Ornit Chiba-Falek and Dr. Elaine Hamm of CLAIRIgene, an NCATS-funded company developing groundbreaking gene therapies for neurodegenerative diseases. Their work embodies the spirit of innovation and the power of women in STEM. NCATS is proud to support women-owned and women-led small businesses like CLAIRIgene through programs like SBIR/STTR, providing critical funding and resources to advance their vital research. Learn more about how NCATS empowers women entrepreneurs: https://go.nih.gov/OujjbuE #NCATSsbir #STTR

Train with us! We have onsite training opportunities in our state-of-the-art labs for early-career scientists. If you’re interested in building your #TranslationalScience skills through hands-on research, visit our website today to learn more: https://go.nih.gov/JgNvmAv

🔬 Are You Developing Breakthrough Biotech Solutions? 🌎 NCATS' SBIR & STTR programs provide crucial funding to help small businesses advance innovative research and move toward commercialization. We’re seeking projects in: - Drug discovery tools - Biomedical technology - And more! Learn more and explore funding opportunities: go.nih.gov/sHre8ZB #NCATSsbir #SBIRSTTR #biotech #lifescience #innovation #funding

📣The NCATS Strategic Plan for 2025–2030 is available. This plan is by and for the #TranslationalScience community, guiding us to a future with more treatments for all people more quickly. Read more about the plan and our next steps: https://go.nih.gov/jTfZhSi

“Making gene therapies for rare diseases more attractive to potential commercial sponsors is one of the ultimate goals of the BGTC projects,” — NCATS Division of #RareDiseases Research Innovation Director Dr. Dominique Pichard. The FDA granted NCATS Rare Pediatric Disease designations and Orphan Drug Designations for investigational gene therapies through the Foundation for the National Institutes of Health's Accelerating Medicines Partnership® (AMP®) Bespoke Gene Therapy Consortium (BGTC), opening the door for companies to further develop the therapies and pave the way to future cures! Learn more about this exciting news: https://go.nih.gov/9C2zdBo #AMP #BGTC

What do top regulators, data scientists, and biomedical innovators say we can do to improve #ClinicalTrials for #RareDiseases? Here are multiple ideas we heard from leaders of FDA CDER, National Center for Advancing Translational Sciences (NCATS), Jazz Pharmaceuticals and the National Bleeding Disorders Foundation: - Consider patients as "lived experience experts" and involve them in the process of identifying research gaps, from the very beginning. - Go out of your way to bring diversity to your clinical trials as a requirement; don't leave anyone out. - Incorporate as much "fit for purpose" patient data as possible; not just clinical data, but experiential data. (i.e. What is it like to grow up day to day with a certain #RareDisease?) - Speed up the process of clinical trials delivering results by defining the efficacy of products in multiple ways, with multiple #endpoints. And make sure these endpoints will have a true beneficial impact for patients, otherwise the #ClinicalTrial may not be worth the time. Ask patients first! - Make enrollment easier, rather than requiring significant travel and time. New digital tools can help with this.

It's as easy as 1, 2, 3! A new three-step screening approach created by NCATS, National Human Genome Research Institute (NHGRI), Simon Fraser University and Roche revealed drugs that can rescue and help restore defective versions of an enzyme that’s a common contributor to #ParkinsonsDisease and Gaucher disease — a #RareDisease. Learn how this innovative drug testing process could speed up new therapies: https://go.nih.gov/gG5QYdS