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...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Ocugen Inc | OCGN | 3 | PHASE3 | OCU400, OCU410ST |
| Johnson & Johnson | JNJ | 1 | PHASE3 | Botaretigene sparoparvovec |
| Belite Bio, Inc. ADR | BLTE | 1 | PHASE2 | Tinlarebant |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE2 | SAR422459 |
| Kiora Pharmaceuticals, Inc. | KPRX | 1 | PHASE2 | KIO-301 |
| ProQR Therapeutics N.V. | PRQR | 1 | PHASE1 | QR-1123 |
QR-421a is an investigational therapy being developed for retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene. It is also being studied in conditions linked to USH2A mutations, including Usher syndrome type 2. It is not approved for any use and remains in clinical development.
QR-421a targets exon 13 of the USH2A gene. USH2A mutations in this exon are a cause of retinitis pigmentosa and Usher syndrome type 2. By acting on this genetic target, the therapy is designed to address the underlying cause of vision loss in patients with these mutations.
QR-421a is being developed by ProQR Therapeutics N.V., which trades on the Nasdaq under the ticker PRQR. The company is the sponsor of the clinical program evaluating the therapy in patients with retinitis pigmentosa due to USH2A mutations.
QR-421a is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA or any other regulatory agency. The Phase 1 program has been completed, and no trials of QR-421a are currently active.
QR-421a has been evaluated in one completed Phase 1 trial, NCT03780257, titled a study to evaluate the safety and tolerability of QR-421a in subjects with retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. The trial enrolled 20 participants across the United States, Canada, and France.
The Phase 1 trial NCT03780257 was a randomized, double-blind, sham-controlled study of QR-421a in 20 participants with retinitis pigmentosa due to USH2A exon 13 mutations. It enrolled adults 18 years and older of any sex and was conducted in the United States, Canada, and France. The trial is completed.