Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D21URW
|
|||
| Drug Name |
QR-421a
|
|||
| Drug Type |
Antisense oligonucleotide
|
|||
| Indication | Retinitis pigmentosa [ICD-11: 9B70; ICD-10: H35.5] | Phase 2/3 | [1] | |
| Company |
ProQR Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Usherin messenger RNA (USH2A mRNA) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 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. | |||
| REF 2 | Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations. Mol Ther. 2021 Aug 4;29(8):2441-2455. | |||
If You Find Any Error in Data or Bug in Web Service, Please Kindly Report It to Dr. Zhou and Dr. Zhang.