Last reviewed · How we verify
AAV5-hRKp.RPGR
AAV5-hRKp.RPGR is a gene therapy that delivers a functional copy of the RPGR gene into retinal cells to restore vision in patients with X-linked retinitis pigmentosa.
AAV5-hRKp.RPGR is a gene therapy that delivers a functional copy of the RPGR gene into retinal cells to restore vision in patients with X-linked retinitis pigmentosa. Used for X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations.
At a glance
| Generic name | AAV5-hRKp.RPGR |
|---|---|
| Also known as | JNJ-74765340, botaretigene sparoparvovec |
| Sponsor | Janssen Research & Development, LLC |
| Drug class | Gene therapy (AAV vector) |
| Target | RPGR gene |
| Modality | Biologic |
| Therapeutic area | Ophthalmology |
| Phase | Phase 2 |
Mechanism of action
This adeno-associated virus (AAV) vector carries the human RPGR (retinitis pigmentosa GTPase regulator) gene, which is mutated in X-linked retinitis pigmentosa (XLRP). The therapy is administered via subretinal injection to transduce photoreceptor cells and restore the missing or defective RPGR protein, thereby halting or slowing photoreceptor degeneration and preserving or improving vision.
Approved indications
- X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations
Common side effects
- Retinal inflammation
- Subretinal fluid accumulation
- Cataract progression
- Injection-related complications
Key clinical trials
- Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (PHASE3)
- Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (PHASE3)
- A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa (PHASE3)
- A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) (PHASE2)
- Long-term Follow-up Gene Therapy Study for RPGR- XLRP
- Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- AAV5-hRKp.RPGR CI brief — competitive landscape report
- AAV5-hRKp.RPGR updates RSS · CI watch RSS
- Janssen Research & Development, LLC portfolio CI