salanersen

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Ligands
salanersen

GtoPdb Ligand ID: 14557

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.

Bioactivity Comments
Salanersen was found to induce expression of hSMN2 three to four times more potently than nusinersen (which has 2'-O-methoxyethyl/MOE ribose modified bases) in human SMN2 transgenic mice [2].

Bioactivity Comments

Salanersen was found to induce expression of hSMN2 three to four times more potently than nusinersen (which has 2'-O-methoxyethyl/MOE ribose modified bases) in human SMN2 transgenic mice [2].