Basic Science Workshop, Ribonucleic Acid Nanomedicine: Reprogramming Cells to Transform Liver Outcomes, Part 2—Transforming Chronic Liver Disease: Breakthroughs and Clinical Updates in Ribonucleic Acid Nanotherapeutics   

Description

RNA nanomedicine has emerged as a groundbreaking approach for safely delivering genetic instructions into target cells, enabling their reprogramming without degradation. Its significance was underscored during the COVID-19 pandemic with the successful deployment of mRNA-based vaccines formulated using lipid nanoparticles. These nanoparticles, available in various formulations, act as carriers to transport RNA directly into target cells for therapeutic action. Patisiran and lumasiran—2 FDA-approved RNA interference (RNAi) agents delivered via lipid nanoparticles (RNA nanotherapeutics)—exemplify this new generation of targeted therapies. These treatments have significantly reduced the need for liver transplantation in patients with genetic conditions such as amyloidosis and hyperoxaluria. Consequently, referrals for liver transplantation due to these metabolic disorders have dramatically declined, highlighting the transformative potential of RNA nanomedicine in clinical practice. In this workshop, experts review the transformative world of RNA nanomedicine; explore its role in delivering genetic instructions with unparalleled precision; discover how advancements in lipid nanoparticle (LNP) formulations are enabling breakthrough therapies; and gain insights into the latest clinical applications that are reshaping treatment paradigms, such as reducing the need for liver transplantation in genetic and metabolic disorders.