This 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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Assay Development and Screening Technology (ADST)

Published by NCATS

High Throughput ScreeningNCATSSmall Molecule

One 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

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Functional Genomics Lab

Published by NCATS

RNAi ScreeningTarget IdentificationNCATS

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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Discovering New Therapeutics Uses for Existing Molecules (NTU) program, National Center for Advancing Translational Sciences (NCATS)

Published by IRDiRC

Research and Drug DiscoveryOrphan DrugDrug RepurposingNCATSNTU-NCATSFunding

The 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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