Webinar – Target Validation and Efficacy

MDC Connects Webinar Series

Medicines Discovery Catapult

MDC Connects is a series of informative webinars covering the drug discovery process, from our own experts and experts at our CRO network partners.

Project failure due to lack of clinical efficacy during development remains an issue and can in part be attributed to inadequate pre-clinical target validation or clinical translation data.

Target validation is required to build confidence in the biological hypothesis, and the strength of the hypothesis is increased as the complexity of the system increases; from validation in cell lines, to primary cells, to complex cell systems and animal disease models before ultimately efficacy is tested in human clinical studies.

In this webinar, Matt Burnham from MDC describes the importance of confirming target engagement, the ligand-target interaction and its mechanism, and provides examples of methods to measure this. He explains the importance of understanding target engagement in the event of a lack of efficacy to establish whether this is due to the compound not engaging with the target or a failure of hypothesis. Amanda Woodrooffe from Precision for Medicine discusses the advantages and disadvantages of in vitro cell models using primary cells and the importance and relevance of these approaches in modelling native biology in human systems prior to the clinic; and finally Lorraine Mooney from Sygnature Discovery outlines the considerations for in vivo proof of concept studies with reference to experimental design and model choice based on the molecule’s mode of action.


YouTube video


Strategies for target and pathway engagement in cellular assays

Go native… Characterising therapeutic effect in primary cellular models at Precision for Medicine

Use of pre-clinical models to deliver proof of concept efficacy at Sygnature Discovery