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Tegsedi targets TTR production in hATTR(1,2)
Tegsedi targets TTR production in hATTR1,2
Tegsedi reduces the synthesis of TTR protein in the liver through degradation of TTR mRNA, targeting the disease at its source.1,2
- Tegsedi is a short, 20-base antisense oligonucleotide (ASO) with a sequence complementary to part of the TTR mRNA1
- Tegsedi binds to and causes degradation of TTR mRNA, inhibiting TTR protein synthesis1,2
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References
mRNA, messenger ribonucleic acid; TTR, transthyretin.
OLE, open-label extension; TTR, transthyretin
- Tegsedi Summary of Product Characteristics. April 2021.
- Benson MD et al. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018;379(1):22–31 (Suppl Appendix).
- Brannagan TH, Wang AK, Coelho T, et al. Eur J Neurol. 2020;27(8):1374-1381.
doi:10.1111/ene.14285 - Coelho T et al. Efficacy and safety with >3 years of inotersen treatment for the polyneuropathy of hereditary transthyretin amyloidosis. OPR15, 7th Congress of the European Academy of Neurology 2021, 19-22 June 20.
Serum TTR levels: double-blind trial and open-label extension
- Median serum TTR reductions in the Tegsedi group in NEURO-TTR reached a nadir of 79% below baseline from week 13 to 652
- Median serum TTR reductions in the placebo-Tegsedi group reached a nadir of 78% below OLE baseline from week 13 to 1043
