Translational Research: Lead Optimization

Lead optimization is the process of generating a pre-therapeutic lead candidate by improving the properties of small molecules or non-viral biologics for which positive and negative attributes have been identified and candidates are tractable to modification. Examples of properties to be improved may be potency, efficacy, selectivity, or bioavailability. This process typically involves the generation and testing of multiple new agents in order to gain a scientific understanding of how variations in lead candidates effect these properties. It is expected that lead optimization will involve assessment of efficacy in animal models and/or ex vivo human samples.