inotersen
Sign in to saveAlso known as Tegsedi
Inotersen, sold under the brand name Tegsedi, is a 2'-O-(2-methoxyethyl) (2'-MOE) antisense oligonucleotide medication used for the treatment of nerve damage in adults with hereditary transthyretin-mediated amyloidosis. The sequence is 5'-TCTTG GTTACATGAA ATCCC-3', where C is 5-methylcytidine, and the first and third section (bases 1-5 and 16–20, separated from the middle section by spaces) are MOE-modified (i.e. inotersen is a gapmer).
Clinical Trials
4 registered- APPROVED_FOR_MARKETINGATTR Expanded Access Program (EAP) by IonisIonis Pharmaceuticals, Inc. · NCT03400098
- PHASE2/PHASE3COMPLETEDEfficacy and Safety of Inotersen in Familial Amyloid PolyneuropathyIonis Pharmaceuticals, Inc. · NCT01737398
- PHASE2WITHDRAWNION-682884 in Patients With TTR Amyloid CardiomyopathyBrigham and Women's Hospital · NCT04843020
- PHASE2TERMINATED24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy PatientsBrigham and Women's Hospital · NCT03702829
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Inotersen, sold under the brand name Tegsedi, is a 2'-O-(2-methoxyethyl) (2'-MOE) antisense oligonucleotide medication used for the treatment of nerve damage in adults with hereditary transthyretin-mediated amyloidosis. The sequence is 5'-TCTTG GTTACATGAA ATCCC-3', where C is 5-methylcytidine, and the first and third section (bases 1-5 and 16–20, separated from the middle section by spaces) are MOE-modified (i.e. inotersen is a gapmer).
The most common side effects are injection site reactions (redness, swelling, bleeding, pain, rash, and itching at the injection site), nausea, headache, tiredness, low platelet counts, and fever.
Excerpted from Wikipedia’s “inotersen” article, available under the CC BY-SA 4.0 licence.