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salanersen   Click here for help

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.
Classification Click here for help
Compound class Nucleic acid
Compound subclass Antisense oligonucleotide (ASO)
Target SMN2
International Nonproprietary Names Click here for help
INN number INN
13182 salanersen
Synonyms Click here for help
BIIB-115 | BIIB115 | ION-306 | ION306
Database Links Click here for help
CAS Registry No. 2701584-16-7 (source: WHO INN record)
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