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.
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the CDK toolkit. All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 16
Hydrogen bond donors 3
Rotatable bonds 16
Topological polar surface area 190.77
Molecular weight 577.57
XLogP -3.68
No. Lipinski's rules broken 3