casimersen   Click here for help

GtoPdb Ligand ID: 11444

Synonyms: Amondys 45® | SRP-4045 | SRP4045
Approved drug
casimersen is an approved drug (FDA (2021))
Compound class: Synthetic organic
Comment: Casimersen is an antisense phosphorodiamidate morpholino oligomer (PMO) that is approved to treat certain patients with Duchenne muscular dystrophy (DMD). It promotes functional dystrophin synthesis in patients who have dystrophin gene variants that are amenable to exon 45 skipping, that is sufficient to restore some muscle function and slow disease progression.
The INN record for casimersen provides chemical identity as all-P-ambo-[2',3'-azanediyl-P-(dimethylamino)-P,2',3'- trideoxy-2',3'-seco](2'-N→5')(C-A-A-T-G-C-C-A-T-C-C-T-G-G-A-G-T-T-C-C-T-G) 5'-{P-[4-({2-[2-(2-hydroxyethoxy)ethoxy]ethoxy}carbonyl)piperazin-1-yl]-N,Ndimethylphosphonamidate}, but we have been unable to resolve this to a SMILES or full IUPAC descriptor.
Classification Click here for help
Compound class Synthetic organic
Approved drug? Yes (FDA (2021))
International Nonproprietary Names Click here for help
INN number INN
10354 casimersen
Synonyms Click here for help
Amondys 45® | SRP-4045 | SRP4045
Database Links Click here for help
CAS Registry No. 1422958-19-7 (source: WHO INN record)
ChEMBL Ligand CHEMBL4297566
DrugBank Ligand DB14984
GtoPdb PubChem SID 440816813
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