Tominersen

• CAT Number: I045376
• CAS Number: 1709886-74-7
• Molecular Weight: 7078.00
• Purity: ≥95%

Tominersen (Cat No.: I045376) is an investigational antisense oligonucleotide developed to treat Huntington’s disease by targeting and reducing mutant huntingtin (mHTT) protein production. It binds to huntingtin (HTT) mRNA, promoting its degradation and thereby lowering levels of both mutant and wild-type HTT. Administered via intrathecal injection, tominersen aims to slow disease progression by addressing the underlying genetic cause. Although early trials showed reduced mHTT levels, later studies revealed mixed results, leading to further evaluation of optimal dosing and patient selection strategies.

Reference

[1]. Tabrizi SJ, et, al. Targeting Huntingtin Expression in Patients with Huntington’s Disease. N Engl J Med. 2019 Jun 13;380(24):2307-2316.
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[2]. Kordasiewicz HB, et al. Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis. Neuron. 2012 Jun 21;74(6):1031-44.
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