Tadnersen

• CAT Number: I045374
• CAS Number: 2170507-65-8
• Purity: ≥95%

Tadnersen (Cat No.: I045374) is an investigational antisense oligonucleotide developed to treat epilepsy caused by SCN1A gene mutations, such as Dravet syndrome. It works by selectively modulating RNA splicing to increase the production of functional Nav1.1 sodium channels, which are deficient in patients with SCN1A loss-of-function mutations. By restoring proper sodium channel expression, tadnersen aims to reduce seizure frequency and severity. Administered intrathecally, it represents a targeted, genetic-based approach to treating severe, drug-resistant epilepsies linked to SCN1A dysfunction. Clinical trials are ongoing.

Reference

[1]. Smeyers J, et al. C9ORF72: What It Is, What It Does, and Why It Matters. Front Cell Neurosci. 2021;15:661447. Published 2021 May 5.
[Content Brief]

[2]. Aoki Y, et al. Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases [published online ahead of print, 2021 Jun 3]. J Neuromuscul Dis. 2021;10.3233/JND-200560.
[Content Brief]