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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Early Translation Branch (ETB)
Published by NCATS
BiossayHigh Throughput ScreeningHit IdentificationETB researchers advance small molecule therapeutic development through assay (test) design, High Throughput Screening and medicinal chemistry.
View this resource Bookmark this resourceNCATS’ 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.
View this resource Bookmark this resourceChemistry technology experts at NCATS develop Small Molecules and screening approaches that other scientists can use to pursue innovations in therapeutic development.
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Matrix Combination Screening
Published by NCATS
Hight Throughput ScreeningLead ValidationMatrix CombinationNCATS experts use a technology called Matrix Combination screening to quickly narrow down a long list of potential drug combinations and find those with the most potential to help patients.
View this resource Bookmark this resourceThe goal of the NCATS 3-D Tissue Bioprinting program is to advance the process of discovery and development of new medicines by developing new assay models that better predict the effects of drugs in humans. 3-D Tissue Models that mimic characteristics of live human tissues are produced on microplates to test effectiveness and toxicity of Small Molecules or other therapeutic.
View this resource Bookmark this resourceThe Tox21 initiative is designed to improve toxicity testing methods and enable faster, more efficient evaluation of chemicals’ effects on human health.
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Stem Cell Translation Laboratory (SCTL)
Published by NCATS
Patient-derived Induced Pluripotent Stem Cell (iPSC)Stem CellNCATS is working to advance translational methods in stem cell research. Induced pluripotent stem cells are particularly useful because scientists can transform them into many different cell types to use for research or therapies.
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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.
View this resource Bookmark this resourceRaDaR, formerly known as the Global Rare Diseases Registry Data Repository program, aims to define best practices for patient registries. RaDaR also strives to identify and adopt standards to support high-quality registries for rare diseases therapeutics development.
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