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Also known as RNA antisense oligonucleotide for intravitreal injection
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 |
|---|---|---|---|---|
| Belite Bio, Inc. ADR | BLTE | 1 | PHASE3 | Tinlarebant |
| Ocugen Inc | OCGN | 3 | PHASE3 | OCU410ST, OCU400 |
| Johnson & Johnson | JNJ | 1 | PHASE3 | Botaretigene sparoparvovec |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE2 | SAR422459 |
| Kiora Pharmaceuticals, Inc. | KPRX | 1 | PHASE2 | KIO-301 |
| ProQR Therapeutics N.V. | PRQR | 1 | PHASE2 | QR-1123 |
| City Therapeutics | CTY | 1 | PHASE1 | CITY-RBP4 |
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 related conditions including Usher syndrome type 2. It is not approved for any indication and remains in clinical development.
QR-421a targets mutations in exon 13 of the USH2A gene, which are a cause of retinitis pigmentosa and Usher syndrome type 2. By addressing this specific genetic defect, the therapy aims to treat the underlying cause of disease in patients with these mutations.
QR-421a is being developed by ProQR Therapeutics N.V., a biopharmaceutical company traded on the Nasdaq under the ticker symbol PRQR. The company focuses on RNA-based therapies for rare genetic diseases.
QR-421a is in Phase 1 clinical development. It has not been approved by the FDA or any regulatory agency and is considered investigational. The Phase 1 program has been completed.
QR-421a has been evaluated in a Phase 1 clinical trial, NCT03780257, titled Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene. This trial enrolled 20 participants across the United States, Canada, and France and is now completed.
No, QR-421a and sepofarsen are distinct investigational therapies. QR-421a targets mutations in exon 13 of the USH2A gene for retinitis pigmentosa, while sepofarsen targets a different genetic mutation. They are separate development programs.