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August 6, 2026 by Dawn Lam
Oligonucleotide therapeutics, including antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), act through base pairing between a short sequence and a target transcript. Although sequences can be selected that perfectly match only the intended target, partial complementarity to other transcripts can cause off-target effects (Figure 1). These hybridization-dependent off-target effects have been well established for […]
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Industry Insights Rare Disease
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 […]
April 22, 2026 by Dawn Lam
In May of 2025, a month after the last dose of k-abe was administered, a remarkable paper was published detailing the conception, development and treatment of the infant KJ Muldoon with an in vivo base editor to cure his carbamoyl-phosphate synthetase 1 (CPS1) deficiency.[1] CPS1 deficiency, which affects approximately 1 in 1,300,000 people, is a […]
Oligo Modification By Application
March 23, 2026 by Dawn Lam
Background Ligases are ubiquitous. They are found in every living organism and are required for cell division – stitching together the Okazaki fragments produced during DNA replication and maintaining genetic integrity by performing the final step in every DNA repair pathway – the crucial ligation step – which is the formation of a phosphodiester linkage […]
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