Emerging trends in nanomedicine: The role of RNAi-based therapies and onpattro’s clinical journey
TL;DR
Onpattro, the first FDA-approved RNAi therapy, is highlighted, Onpattro’s clinical development is highlighted, compares delivery platforms, discusses translational challenges, and emerging technologies that will guide the next generation of RNAi nanomedicines in personalized therapy are examined.
Abstract
Nanomedicine has transformed therapeutic strategies by enabling precise delivery of nucleic acid-based drugs, including small interfering RNA (siRNA), messenger RNA (mRNA), and antisense oligonucleotides. A landmark achievement is Onpattro (patisiran), the first FDA-approved RNAi therapy, which employs lipid nanoparticles (LNPs) to silence transthyretin in hereditary amyloidosis. Its approval validates RNAi as a viable therapeutic modality and underscores the central role of nanocarriers in clinical translation. Despite this success, barriers such as nanoparticle stability, targeted delivery, immunogenicity, and manufacturing scalability remain. Recent advances in mRNA vaccines, CRISPR-based gene editing, and stimuli-responsive nanoparticles are addressing these challenges, supported by growing clinical case studies and real-world data. This review highlights Onpattro’s clinical development, compares delivery platforms, discusses translational challenges, and examines emerging technologies that will guide the next generation of RNAi nanomedicines in personalized therapy.