Details of the Drug

General Information of Drug (ID: DMGGJAR)

Drug Name

QR-421a

Indication

Disease Entry	ICD 11	Status	REF
Retinitis pigmentosa	9B70	Phase 2/3	[1]
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Drug Type

Antisense oligonucleotide

Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

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

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

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.