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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Oligo Modification By Application
September 18, 2025 by Dawn Lam
Antibodies have unique targeting specificity for different cell- and tissue-types. This has led to rapid growth in Antibody-Drug Conjugates (ADCs) for the treatment of cancer, which can deliver a cytotoxic payload to a particular cell-type or to cancerous cells that overexpress specific receptors. Oligonucleotides, such as antisense oligos (ASOs) and small-interfering RNA oligos (siRNAs), have […]
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