Details of the Drug

General Information of Drug (ID: DM31L9K)

Drug Name
Sepofarsen
Synonyms QR-110
Indication
Disease Entry ICD 11 Status REF
Leber congenital amaurosis 9B70 Phase 2/3 [1]
ATC Code
S01XA29: Sepofarsen
S01XA: Other ophthalmologicals
S01X: OTHER OPHTHALMOLOGICALS
S01: OPHTHALMOLOGICALS
S: SENSORY ORGANS
Drug Type
Oligonucleotide
Sequence
GGUGGAUCACGAGUUCA
Cross-matching ID
UNII
7CAZ46X8EL
DrugBank ID
DB16327
TTD ID
DS14FO

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
CEP290 messenger RNA (CEP290 mRNA) TT3XBOV CE290_HUMAN Inhibitor [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 ClinicalTrials.gov (NCT04855045) An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN). U.S. National Institutes of Health.
2 Clinical pipeline report, company report or official report of ProQR Therapeutics.