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Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. [1]
Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. [3] It is a transthyretin-directed antisense oligonucleotide. [3]
GRN163 is the pharmacological component with telomerase inhibition based on experiments with poly-G oligonucleotides first conducted at the University of Nebraska Medical Center under contract with Lynx Therapeutics. [9] The palmitic acid moiety is conjugated via a phosphothioate linkage to the backbone of the antisense oligonucleotide.
The US Food and Drug Administration (FDA) approved inotersen in October 2018. [6] The application for inotersen was granted orphan drug designation. [10] The FDA approved inotersen based on evidence from one clinical trial (Trial 1/NCT01737398) that included 172 participants with hereditary transthyretin-mediated amyloidosis. [6]
In November 2016, the new drug application was accepted under the FDA's priority review process on the strength of the Phase III trial and the unmet need, and was also accepted for review at the European Medicines Agency (EMA) at that time. [21] [22] It was approved by the FDA in December 2016 and by EMA in May 2017 as the first drug to treat SMA.
[1] [2] Olezarsen is an apolipoprotein C-III-directed antisense oligonucleotide. [1] It is given by injection under the skin. [1] Olezarsen was approved for medical use in the United States in December 2024. [1] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4]
Tegsedi, developed and marketed by Ionis Pharmaceuticals, was approved by the FDA in October 2018 for the treatment of hereditary transthyretin amyloidosis (hATTR). [10] The chemical structure is a 20-mer oligonucleotide with PS backbone modifications and 2'-MOE ribose substitutions. [4]
The most common side effects include upper respiratory tract infection, injection site reaction, cough, and pyrexia (fever). [2] [3] [1]Although kidney toxicity was not observed in the clinical studies, the clinical experience is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides.