Drug Discovery Identifying Targets and Lead Molecules to Fight Disease

The drug discovery phase marks the transition from basic research to bona-fide drug development efforts and this is when momentous decisions on the strategy of your drug development programs are made: From formulating a hypothesis on how disease indications could be targeted to effect a positive outcome (Target ID), to exploratory phenotypic screening efforts to identify promising lead molecules which elicit a desirable effect.

Generating a sound strategy is crucial to ensure efficient use of R&D time and resources. Pursuing weak leads and failing late will drive up costs and lengthen your time to market or even jeopardize successful completion of the program.

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Target Identification

Finding a Disease’s Vulnerabilities

When devising a strategy to prevent, treat or cure a disease, a thorough understanding of the pathophysiology is required. This includes cellular pathways and disease-specific tissue biomarkers which can provide information on possible drug targets that could elicit a positive outcome.

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Lead Identification

Who Hits the Target?

As you focus on the right target, you must also take care to identify the most promising lead molecules to be developed into breakthrough medicines, so as to minimize the costs associated with the high attrition rate commonly found in the later stages of drug development.

To find and develop highly specific and efficacious drugs, drug discovery efforts are increasingly shifting from simple assays to elaborate, high-content screening approaches which capture a high number of parameters. Additional complexity is introduced by expanding your sample types from traditional 2D cell cultures to physiologically more relevant 3D cell models and organoids, as well as by shifting from endpoint to live-cell assays. As promising as these changes are in terms of increasing the odds of identifying better drug candidates, they also impose new challenges on imaging technology.

Sample: courtesy of P. Denner, Core Research Facilities, German, Center of Neurodegenerative Diseases (DZNE), Bonn, Germany.