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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Discovering New Therapeutics Uses for Existing Molecules (NTU) program, National Center for Advancing Translational Sciences (NCATS)
Published by IRDiRC
Drug RepurposingFundingNCATSNTU-NCATSOrphan DrugResearch and Drug DiscoveryThe NTU program aims to improve the process of developing new treatments for diseases by finding new uses for existing therapies that already have cleared several key steps along the development path. Existing or partially developed therapeutic candidates can be repurposed for use in new disease indications
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NCATS and NHGRI Genetic and Rare Diseases Information Center
Published by IRDiRC
Database or Tool GARDGenetic DiseaseInformation CenterNCATSNHGRIResearch and Drug DevelopmentGARD provides the public with access to current, reliable, and easy-to-understand information about rare or genetic diseases their families, health care providers, researchers, and the public in English or Spanish.
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Tissue Chip for Drug Screening program and Consortium
Published by IRDiRC
BiomarkerDrug DiscoveryDrug Screening ProgrammeHigh Throughput ScreeningLibraryNCATSResearch and Drug DevelopmentTissue ChipNCATS, in collaboration with other NIH Institutes and Centers and the Food and Drug Administration (FDA), is leading the Tissue Chip for Drug Screening program to develop human tissue chips that accurately model the structure and function of human organs — such as the lungs, liver and heart — to help predict drug Safety in humans more rapidly and effectively
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The Assay Guidance Manual
Published by NIH
Drug Discovery GuidelineDrug RepurposingNCATSNIHResearch and Drug DevelopmentSmall MoleculeTarget Validation Drug DevelopmentThis manual is a comprehensive resource for early drug discovery, providing guidelines for developing therapeutic hypotheses, validating targets and pathways, and establishing proof of concept criteria. It includes analyses of costs at various stages of drug development. The content encompasses critical decision-making points in New Chemical Entity (NCE) development, exploratory Investigational New Drug (IND) processes, and orphan drug designation. Furthermore, it delves into Drug Repurposing and drug delivery technologies, making it an invaluable guide for professionals initiating new drug discovery and development programs.
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