|
Synonyms: SRP-4053 | SRP4053 | VYONDYS 53®
golodirsen is an approved drug (FDA (2019))
Compound class:
Nucleic acid
Comment: Golodirsen (SRP-4053) is an antisense RNA oligonucleotide that uses the phosphorodiamidate morpholino oligomer (PMO) chemistry [2]. It was designed to induce exon 53 skipping in the dystrophin gene as a mechanism to increase expression of functional dystrophin protein in the muscle fibers of appropriately selected Duchenne muscular dystrophy patients [1].
Full sequence as provided in the agent's INN record is: all-P-ambo-[2′,3′-Azanediyl-P-(dimethylamino)-P,2′,3′-trideoxy-2′,3′-seco](2′-N→5′)(G-T-T-G-C-C-T-C-C-G-G-T-T-C-T-G-A-A-G-G-T-G-T-T-C) 5′-{P-[4-({2-[2-(2-hydroxyethoxy)ethoxy]ethoxy}carbonyl)piperazin-1-yl]-N,N-dimethylphosphonamidate}, but we have been unable to resolve this to a SMILES or HELM notation. |
Classification  |
|
| Compound class | Nucleic acid |
| Compound subclass | Antisense oligonucleotide (ASO) |
| Target | dystrophin (DMD) pre-mRNA |
| Approved drug? | Yes. FDA (2019) |
| International Nonproprietary Names |
|
| INN number | INN |
| 10355 | golodirsen |
| Synonyms |
| SRP-4053 | SRP4053 | VYONDYS 53® |
| Database Links |
|
| CAS Registry No. | 1422959-91-8 (source: WHO INN record) |
| GtoPdb PubChem SID | 504705424 |
| Search PubMed clinical trials | golodirsen |
| Search PubMed titles | golodirsen |
| Search PubMed titles/abstracts | golodirsen |
