Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DM31L9K)

Drug Name
Sepofarsen Drug Info
Synonyms QR-110
Indication
Disease Entry ICD 11 Status REF
Leber congenital amaurosis 9B70 Phase 2/3 [1]
Cross-matching ID
TTD Drug ID
DM31L9K

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas
Download the Complete Interaction Atlas for Visualization in Cytoscape

Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
CEP290 messenger RNA (CEP290 mRNA) TT3XBOV CE290_HUMAN Inhibitor [2]

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.