Beyond the liver: reimagining delivery systems for the future of gene and RNA medicines

Over the past decade, gene and RNA therapeutics have moved from scientific promise to clinical impact—transforming once-hypothetical interventions into life-changing realities. Yet a persistent challenge continues to define the field: delivery [1]. The liver has served as both a proving ground and a safe harbor for innovation, thanks to its natural receptivity to lipid nanoparticles (LNPs) and AAV vectors [2]. More recently, N-acetylgalactosamine (GalNAc) conjugates have further expanded the liver’s therapeutic potential by enabling highly specific, receptor-mediated delivery to hepatocytes via the asialoglycoprotein receptor (ASGPR) [3]. But most diseases don’t begin in the liver—and the next frontier in genetic medicine will depend on our ability to reach far beyond it (Figure 1).

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