Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
QR-421a · 1 trial · 8 indications
Incidence and severity of ocular AEs
| Arm | Type | Description |
|---|---|---|
| QR-421a | EXPERIMENTAL | Single dose administration |
| Sham-procedure (dose cohort 1&2 only) | SHAM_COMPARATOR | Sham-procedure (no experimental drug administered) |
| Name | Type | Description |
|---|---|---|
| QR-421a | DRUG | RNA antisense oligonucleotide for intravitreal injection |
| Sham-procedure (dose cohort 1&2 only) | OTHER | Sham-procedure (no experimental drug administered) |
Inclusion Criteria: 1. Male or female, ≥ 18 years of age. 2. Clinical presentation consistent with RP with Usher syndrome type 2 or non-syndromic RP (NSRP), based on ophthalmic, audiologic, and vestibular examinations. 3. A molecular diagnosis of homozygosity or compound heterozygosity for 1 or mor...
QR-421a is an investigational small molecule being developed for retinitis pigmentosa, a rare inherited retinal disease. It is being studied in patients with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene, including those with Usher syndrome type 2.
QR-421a is being developed by ProQR Therapeutics N.V., a biopharmaceutical company traded on the Nasdaq under the ticker symbol PRQR. The company is focused on developing therapies for rare diseases.
QR-421a is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 trial evaluating its safety and tolerability has been completed.
QR-421a has been studied in one completed Phase 1 clinical trial, identified as NCT03780257. This trial evaluated the safety and tolerability of QR-421a in 20 subjects with retinitis pigmentosa due to mutations in exon 13 of the USH2A gene.
QR-421a is the primary name for this investigational drug. No alternative names have been reported for this asset in clinical development.