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 Property
June 18, 2026 by Dawn Lam
Introduction to Antisense Oligos (ASOs): Antisense Oligonucleotides (ASOs) are fairly short oligonucleotides, typically 15-30 nucleotides in length, which are composed…
Oligo Modification By Application
May 4, 2026 by Dawn Lam
Circular oligonucleotides are an emerging class of nucleic acid therapeutics (NATs) because of their resistance to exonucleases and their reduced immunogenicity.[1] Wesselhoeft et al., demonstrated that exogenous circRNA did not activate Toll-like Receptors (TLRs) nor Retinoic acid-Inducible Gene I (RIG-I), provided that the circRNA was meticulously purified to remove all linear RNA fragments. In addition, […]
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 […]
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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