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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Backbone Oligo Modification by Type
May 6, 2024 by Dawn Lam
Check out our updated section below! What are antisense oligonucleotides (ASOs)? Antisense oligonucleotides (ASOs) are short nucleic acids designed to bind to specific RNA sequences, thereby inhibiting, reducing, restoring, or modifying the expression of target RNA. Over the past two decades, this method of gene silencing at the posttranscriptional stage has become increasingly popular as […]
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