Inotersen sodium

• CAT Number: I045363
• CAS Number: 1432726-13-0
• Molecular Weight: 7601.00
• Purity: ≥95%

Inotersen sodium (Cat No.: I045363) is an antisense oligonucleotide designed to treat hereditary transthyretin-mediated (hATTR) amyloidosis, a rare genetic disorder. It selectively binds to transthyretin (TTR) mRNA in the liver, promoting its degradation and reducing production of both mutant and wild-type TTR protein. This lowers amyloid deposits in tissues, alleviating neuropathy and organ dysfunction. Administered via subcutaneous injection, inotersen sodium has demonstrated efficacy in improving neurological function and quality of life. It represents a targeted RNA-based therapy approved for managing hATTR amyloidosis.

Reference

[1]. Ackermann EJ, et, al. Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides. Amyloid. 2016 Sep;23(3):148-157.
[Content Brief]

[2]. Mathew V, et, al. Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis. Drug Des Devel Ther. 2019 May 6;13:1515-1525.
[Content Brief]

[3]. Benson MD, et, al. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):22-31.
[Content Brief]