Integrative drug discovery through molecular, cellular, genomic, and immunological approaches: From gene editing to precision antibody engineering

Abstract

Modern drug discovery is undergoing a transformative shift driven by the integration of molecular, cellular, genomic, and immunological approaches. This review provides a comprehensive overview of cutting-edge methodologies that are reshaping target identification and therapeutic development, including multi-omics profiling, CRISPR–Cas functional genomics, high-throughput sequencing, and bioinformatics-driven network analysis. We highlight how these approaches enable precise dissection of disease-relevant biological mechanisms, facilitate validation of drug targets, and improve experimental efficiency in both in vitro and in vivo systems. Beyond discovery, we emphasize translational applications, illustrating the clinical potential of CRISPR/Cas9 in cancer therapy and lessons learned from rare and complex diseases such as sickle cell disease, transfusion-dependent beta-thalassemia, and Alzheimer’s disease. Additionally, the review explores advances in therapeutic antibodies, engineered biologics, and antibody–drug conjugates, detailing experimental strategies for their design, optimization, and mechanistic evaluation. By integrating experimental innovation with computational modeling, this work bridges fundamental molecular insights with clinical relevance, providing a framework for rational drug development. Designed as both an educational resource for newcomers and a practical guide for experienced researchers, the review emphasizes mechanistically grounded, clinically translatable strategies that accelerate modern drug discovery and precision medicine, offering a forward-looking perspective on the integration of emerging technologies into next-generation therapeutics.