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October 6, 2026 by Dawn Lam
Introduction The clinical success of GalNAc-conjugated siRNAs established a powerful paradigm for nucleic acid therapeutics (NATs): effective delivery through receptor-mediated endocytosis (RME) can transform a promising nucleic acid into a viable medicine. GalNAc succeeded because it leveraged the asialoglycoprotein receptor (ASGPR), which is highly expressed on hepatocytes and undergoes efficient receptor recycling. As a result, […]
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Rare Disease Industry Insights
July 23, 2026 by Dawn Lam
From Fragile Oligonucleotides to an Established Therapeutic Modality Nucleic acid therapeutics (NATs) have evolved from an intriguing scientific concept into a major pharmaceutical modality. More than two decades after the first FDA approval of an oligonucleotide drug, therapeutic oligonucleotides now treat rare diseases, such as spinal muscular atrophy and hereditary transthyretin amyloidosis, to widespread diseases […]
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