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
November 22, 2024 by Dawn Lam
What backbone chemistries have been used for stereopure oligonucleotides? Traditional oligonucleotide synthesis using standard phosphoramidite methods does not control stereochemistry, resulting in a stereorandom phosphorothioate (PS) mixture where each linkage may adopt either R or S configuration. Studies in retinal disorders, CNS diseases, and muscular atrophy have demonstrated that stereopure oligonucleotides can significantly impact biological […]
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