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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] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca. [4] [5] [6] [7]
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]
The use of Morpholino antisense oligonucleotides for gene knockdowns in vertebrates, which is now a standard technique in developmental biology and is used to study altered gene expression and gene function, was first developed by Janet Heasman using Xenopus. [13] FDA-approved Morpholino drugs include eteplirsen and golodirsen. The antisense ...
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]
Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping.
Imetelstat is an oligonucleotide telomerase inhibitor. [ 1 ] [ 2 ] [ 3 ] By blocking telomerase activity, imetelstat causes telomere shortening, inhibits the proliferation of malignant stem and progenitor cells and induces cell death, ultimately leading to a reduction in malignant clones.
Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics.The company has three commercially approved medicines: Spinraza (), Tegsedi (), and Waylivra (Volanesorsen), and has four drugs in pivotal studies: tominersen for Huntington's disease (together with Roche), tofersen for SOD1-ALS, AKCEA ...
Olezarsen, sold under the brand name Tryngolza, is a medication used in the treatment of familial chylomicronemia syndrome. [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.