Sepofarsen
Sepofarsen is an oligonucleotide pharmaceutical. It is currently being investigated in clinical studies.
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Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
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Indications
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Clinical
Clinical Trials
4 clinical trials
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Indications Phases 4
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Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Leber congenital amaurosis | D057130 | Orphanet_65 | H35.5 | 2 | 4 | 2 | — | — | 4 |
| Neurologic manifestations | D009461 | 1 | 3 | 2 | — | — | 3 | ||
| Retinal diseases | D012164 | HP_0000479 | H35.9 | 1 | 3 | 2 | — | — | 3 |
| Vision disorders | D014786 | HP_0000505 | H53.11 | 1 | 3 | 2 | — | — | 3 |
| Eye diseases | D005128 | EFO_0003966 | H44 | 1 | 3 | 2 | — | — | 3 |
| Blindness | D001766 | HP_0000618 | H53.12 | 1 | 3 | 2 | — | — | 3 |
| Hereditary congenital and neonatal diseases and abnormalities | D009358 | 1 | 3 | 2 | — | — | 3 | ||
| Sensation disorders | D012678 | H02.59 | 1 | 3 | 2 | — | — | 3 | |
| Hereditary eye diseases | D015785 | 1 | 2 | 1 | — | — | 2 | ||
| Retinal degeneration | D012162 | HP_0001105 | — | 1 | 1 | — | — | 1 |
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Indications Phases 2
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Indications Phases 1
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Indications Without Phase
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Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | SEPOFARSEN |
| INN | sepofarsen |
| Description | Sepofarsen is an oligonucleotide pharmaceutical. It is currently being investigated in clinical studies. |
| Classification | Oligonucleotide |
| Drug class | antisense oligonucleotides |
| Image (chem structure or protein) | |
| Structure (InChI/SMILES or Protein Sequence) | — |
Identifiers
| PDB | — |
| CAS-ID | — |
| RxCUI | — |
| ChEMBL ID | CHEMBL4594497 |
| ChEBI ID | — |
| PubChem CID | — |
| DrugBank | — |
| UNII ID | 7CAZ46X8EL (ChemIDplus, GSRS) |
Target
Agency Approved
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Variants
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