Cutting-edge AI-driven models and computational tools are enhancing drug target identification and nanoparticle characterization. OutSee’s Nomaly platform applies structural and functional genomic analysis to predict phenotypes and mechanisms, aiding target discovery complementary to traditional genomic approaches. NanoFCM’s single-molecule NanoAnalyzer enables refined quality control of lipid nanoparticle-based in vivo CAR-T therapies by quantifying mRNA loading and surface ligand decoration, improving therapeutic efficacy and safety. These technological integrations exemplify transformative impacts on biotechnology pipelines and therapeutic development.