MDC Connects: A Guide to Drug Discovery

Published by Medicines Discovery Catapult

Drug DiscoveryTarget IdentificationSmall Molecules

This guideline is a summary presentation from the Medicines Discovery Catapult (MDC) webinars. The aim was to create a resource of modern drug discovery knowledge to support small companies who are developing their own medicines. The guideline is focused on preclinical research with an emphasis on small molecules in the following topics:
• What makes, and how to identify, a good target
• Methods to identify the chemical compound which would become the drug
• How to optimise that drug
• How to test it works in models of disease
• And how to demonstrate that it would be safe to dose in humans

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Genetic-Driven Druggable Target Identification and Validation

Published by Trends Genet.

Target IdentificationGWAS AssociationGenetic ToolboxExpression Quantitative Trait Locus

This article reviews the opportunities and challenges, and infer criteria for the optimal use of genetic findings in the drug discovery pipeline. Choosing the right biological target is the critical primary decision for the development of new drugs. Systematic genetic association testing of both human diseases and quantitative traits, along with resultant findings of coincident associations between them, is becoming a powerful approach to infer drug targetable candidates and generate in vitro tests to identify compounds that can modulate them therapeutically

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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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