药物潜在靶标识别与药物设计方法研究

Drug target identification and validation constitute the fundamental stage in novel drug development. The principal challenge lies in identifying biomolecular targets that concurrently fulfill three critical requirements: relevance to disease mechanisms, structural targetability, and therapeutic potential. Through systematic and comprehensive target validation, researchers can establish definitive causal relationships between target modulation and therapeutic outcomes. By conducting thorough analyses of target three-dimensional structures and functional characteristics, integrated with rational drug design approaches, scientists can effectively discover and optimize target-specific bioactive compounds. This strategy substantially reduces both the drug discovery timeline and associated research and development expenditures. In complex disease therapeutics, the combined implementation of target discovery technologies and drug design methodologies has yielded dual benefits: enhanced drug targeting capability and treatment efficacy, while simultaneously driving the evolution of pharmaceutical research and development from conventional broad-spectrum therapies toward precision medicine interventions. This review comprehensively outlines our research group’s methodological and technological advancements over two decades in multiple areas, including target identification, drug design, and AI-assisted pharmacology, along with corresponding progress in pioneering drug development. Furthermore, based on contemporary research landscapes, we examine pressing scientific challenges in drug design and provide insightful projections regarding future developments and potential obstacles in the field.