Innovations in targeted drug delivery: From nanotechnology to clinical applications

Abstract

Targeted drug delivery systems (TDDS) have emerged as transformative platforms for enhancing therapeutic precision while minimizing systemic toxicity. By exploiting mechanisms such as the enhanced permeability and retention (EPR) effect, ligand-based active targeting, and stimulus-responsive release, these systems offer improved pharmacokinetics and site-specific action. Clinical success stories underscore their translational impact—liposomal formulations like Doxil® reduce doxorubicin-related cardiotoxicity by nearly 50 %, while lipid nanoparticle-based Onpattro® became the first FDA-approved RNA interference therapy, paving the way for mRNA vaccine technologies. Despite such milestones, challenges persist, including tumor heterogeneity, immunogenicity, and regulatory complexities, which hinder broader clinical adoption. Emerging strategies—hybrid nanocarriers, AI-driven drug release, and integration with digital health—are reshaping the future of personalized medicine. This review provides a comprehensive analysis of current nanocarrier platforms, clinical progress, regulatory trends, and innovations driving the next generation of targeted drug delivery.