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Synonyms: BIIB-115 | BIIB115 | ION-306 | ION306
Compound class:
Nucleic acid
Comment: Salanersen (BIIB115/ION306) is proposed as a survival motor neuron 2 (SMN2) synthesis inducer. It is a splice-switching anti-sense oligonucleotide (ASO) that is designed to restore normal SMN2 gene expression and translation of the SMN2 protein. This mechanism is intended to slow neurodegeneration and improve motor function in patients with infantile-onset spinal muscular atrophy, a condition that is caused by loss of functional SMN protein [1]. Salanersen is synthesised with 2'-O-[2-(methylamino)-2-oxoethyl] (NMA) ribose modifications to enhance its pharmacological properties compared to its predecessor nusinersen [2]. The full sequence is [2'-O-(2-methylamino)-2-oxoethyl](m5C-sp-A-m5C-sp-m5U-m5U-sp-m5U-sp-m5C-sp-A-sp-m5U-sp-A-sp-A-sp-m5U-sp-G-sp-m5C-sp-m5U-sp-G-sp-G-sp-m5C), as detailed in the INN record for the agent.
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| References |
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1. Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, Swoboda KJ, Reyna SP, Sakonju A, Darras BT et al.. (2017)
Natural history of infantile-onset spinal muscular atrophy. Ann Neurol, 82 (6): 883-891. [PMID:29149772] |
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2. Ling K, Prakash TP, Yu J, Jackson M, Chun SJ, Bachmann G, Post N, Greenlee S, Soriano A, Hunyara JL et al.. (2026)
Enhanced splicing modulation by NMA-modified antisense oligonucleotides. Nucleic Acids Res, 54 (10). [PMID:42165132] |
