National Center for Advancing Translational Sciences (NCATS)

If you want to read about the latest news from us, including research progress, events and #FundingOpportunities, subscribe to NCATS News: https://bit.ly/15qf4Lf

“By driving this gene therapy trial forward, we’re not only addressing an urgent need for the MMA community, but also building a framework that could accelerate future rare disease trials at NIH,” — NCATS Director Joni Rutter, Ph.D. Explore how NCATS and the National Human Genome Research Institute (NHGRI) are supporting a #ClinicalTrial to test MMA-101 — a gene therapy for methylmalonic acidemia — and how it could inform #PaVeGT & #BGTC: https://go.nih.gov/OT08PVD #RareDisease

Our November Newsletter is out now! Read about: ? A drug screening funnel for finding new therapies for #GaucherDisease and Parkinson’s ? #NCATSsbir funding for Spectradyne to develop a product that can accurately analyze #nanoparticles ? An NCATS-National Human Genome Research Institute (NHGRI)-supported #GeneTherapy advancements for a #RareDisease ? And much more! Read the full newsletter: https://lnkd.in/d6_f8wyz

Methylmalonic acidemia (MMA) is an often life-threatening, rare disease that affects the liver. NCATS and the National Human Genome Research Institute (NHGRI) are working together to support the development of a #ClinicalTrial investigating if MMA-101 — a potential therapeutic — is safe and shows some signs of treating the #RareDisease. Read the article for more details and stay tuned for updates: https://go.nih.gov/OT08PVD The National Institute of Neurological Disorders and Stroke (NINDS), the NIH Clinical Center (CC) and the Eunice Kennedy Shriver?National Institute of Child Health and Human Development (NICHD) will also participate.

With funding from #NCATSsbir, a small startup, Spectradyne developed a new product — the ARC? particle analyzer — that can accurately and quickly analyze the tiniest particles of living things. These #nanoparticles help us understand basic biology and can aid disease diagnosis and treatment. Being able to accurately and quickly identify these particles and their qualities is important for growing the field of nanomedicine. Read about the development of this technology and NCATS’ role: https://go.nih.gov/oiorQ6X

Defects in the enzyme beta-glucocerebrosidase (GCase) are linked to #GaucherDisease and #ParkinsonsDisease. Researchers have identified GCase as an urgent therapeutic target. Learn how researchers at NCATS found promising therapeutics that address GCase defects: https://go.nih.gov/gG5QYdS #NCATSLabs #RareDiseases

Take advantage of this opportunity to elevate your NCATS SBIR/STTR research through I-Corps at The National Institutes of Health! Gain training, mentorship and expert guidance on commercialization. Apply by Nov. 22:?https://lnkd.in/ecCfzd7w #NCATSsbir

Today at the NCATS #WomenInScience leadership panel, remarkable women leaders from The National Institutes of Health and the Women in Science Diplomacy Association (WiSDA) candidly shared their personal stories and career advice for young women who are interested in pursuing careers in #STEM. NASA - National Aeronautics and Space Administration Astronaut Dr. Kate Rubins, who also served as a panelist, joined NCATS Director Dr. Joni Rutter for a fireside chat where they discussed Kate’s experience as a female scientist on Earth and in space. Thank you to the women leaders for sharing your stories and insights and to all who joined us for this inspiring conversation! #AstroKate #SpaceChat

Last week at the Milken Institute Future of Health Summit, NCATS’ Director Joni Rutter, Ph.D., spoke about the future of research and development and the impact NCATS is making on the process of #DrugDevelopment. Take a look at these photos from the Summit!