General Information of Drug (ID: DMGGJAR)

Drug Name
QR-421a Drug Info
Indication
Disease Entry ICD 11 Status REF
Retinitis pigmentosa 9B70 Phase 2/3 [1]
Cross-matching ID
TTD Drug ID
DMGGJAR

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Usherin messenger RNA (USH2A mRNA) TTVCLLA USH2A_HUMAN Not Available [2]

References

1 ClinicalTrials.gov (NCT05176717) A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss. U.S.National Institutes of Health.
2 Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations. Mol Ther. 2021 Aug 4;29(8):2441-2455.