Target validation

Area of Science:

• Genomics and Proteomics
• Drug Discovery and Development

Background:

• The Human Genome Project has spurred anticipation for identifying new disease-specific molecular targets.
• Current methods for gene identification (genotyping, microarrays) are advancing rapidly.

Purpose of the Study:

• To assess the current state and challenges of validating molecular targets for drug discovery.
• To highlight the gap between target identification capabilities and validation realities.

Main Methods:

• Review of current practices in target identification and validation.
• Discussion of proteomic techniques and their role in target validation (targetomics).
• Emphasis on the need for integrated pharmacological approaches and clinical validation.

Main Results:

• Target validation using proteomic techniques is in its early stages, with capabilities lagging behind expectations.
• Established criteria for target validation are lacking, and timelines for success are underestimated.
• Traditional pharmacological approaches are often overlooked in the pursuit of novel targets.

Conclusions:

• The transition from target identification to validation is a critical bottleneck in drug discovery.
• Clinical efficacy of a drug targeting the validated molecule is the definitive measure of success.
• Future drug discovery efforts must integrate rigorous validation strategies to meet clinical needs.