- Guide to PHARMACOLOGY
Synonyms: ASO-10-27 | Ionis-SMNrx | ISIS 396443 | ISIS-SMNRx | ISIS396443 | Spinraza®
nusinersen is an approved drug (FDA (2016), EMA (2017))
Compound class: Synthetic organic
Comment: Nusinersen (ISIS 396443) is an antisense oligo(ribo)nucleotide (ASO) which induces survival motor neuron (SMN) protein expression from SMN2 genes with the exon 7-skipping mutation .
Nusinersen was initially available for use as a designated orphan drug (by the US FDA and EMA), and was the first drug to be fully approved for the treatment of spinal muscular atrophy (SMA).
The sequence contains 2'-O-(2-methoxyethyl) (2'-MOE)-oligoribonucleotides to reduce nuclease degradation and enhance binding affinity towards the complementary RNA. The full sequence is [2'-O-(2-methoxyethyl)](3'-5')(P-thio)(mU-mC-A-mC-mU-mU-mU-mC-A-mU-A-A-mU-G-mC-mU-G-G) as detailed in the INN record for the agent. A full structural rendering using SMILES from Chemspider is available here (CSID:34983394). The sequence for nusinersen is claimed in patent WO2010148249A1 as SEQ ID NO: 1 .
|Compound class||Synthetic organic|
|Approved drug?||Yes (FDA (2016), EMA (2017))|
|International Nonproprietary Names|
|ASO-10-27 | Ionis-SMNrx | ISIS 396443 | ISIS-SMNRx | ISIS396443 | Spinraza®|
|CAS Registry No.||1258984-36-9 (source: WHO INN record)|
|GtoPdb PubChem SID||328083518|
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