The TRND program supports preclinical development of therapeutic candidates intended to treat rare or neglected disorders, with the goal of enabling an Investigational New Drug application.
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NCATS’ Functional Genomics Lab is designed to develop and improve RNAi Screening approaches to better understand gene function and identify treatment targets. Gene silencing through RNAi has emerged as a powerful tool for understanding gene function. Over the past several years, high-throughput RNAi screens have illuminated a wide variety of biological processes, ranging from genes that affect the activity of therapeutic agents to novel components of signaling pathways.
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Rare Diseases Clinical Research Network (RDCRN)
Published by NCATS
Clinical DevelopmentNCATSNetworkRDCRNThe RDCRN program is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.
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Assay Development and Screening Technology (ADST)
Published by NCATS
High Throughput ScreeningNCATSSmall MoleculeOne of the first steps in the drug development process is creating test systems — called assays — on which researchers assess the effects of chemical compounds on cellular, molecular or biochemical processes of interest. At NCATS, the experts in the Assay Development and Screening Technology (ADST) program work to optimize assays requested or submitted by the biomedical research community for high-throughput small-molecule screening
View this resource Bookmark this resourceNCATS launched the Biomedical Data Translator (Translator) program to accelerate biomedical translation for the research community. Through this program, NCATS will integrate multiple types of existing data sources and reveal potential relationships across the spectrum of data types.
View this resource Bookmark this resourceThis NCATS-led pilot project will test whether it is possible to increase the efficiency of Gene Therapy clinical trial startup by using the same Gene Delivery System and manufacturing methods for multiple gene therapies.
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