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Genetic: AAV5-hRKp.RPGR Low Dose
Genetic: AAV5-hRKp.RPGR Low Dose is a Gene therapy (AAV vector) Biologic drug developed by Janssen Research & Development, LLC. It is currently in Phase 3 development for X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations. Also known as: botaretigene sparoparvovec.
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 | Genetic: AAV5-hRKp.RPGR Low Dose |
|---|---|
| Also known as | botaretigene sparoparvovec |
| Sponsor | Janssen Research & Development, LLC |
| Drug class | Gene therapy (AAV vector) |
| Target | RPGR gene; photoreceptor cells |
| Modality | Biologic |
| Therapeutic area | Ophthalmology / Retinal Genetic Disorders |
| Phase | Phase 3 |
Mechanism of action
This adeno-associated virus (AAV5) vector carries the human RPGR (retinitis pigmentosa GTPase regulator) gene under control of a photoreceptor-specific promoter. The therapy is administered via subretinal injection to transduce photoreceptor cells, enabling them to produce functional RPGR protein and halt or slow photoreceptor degeneration. The low-dose formulation represents a reduced viral vector dose compared to higher-dose variants.
Approved indications
- X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations
Common side effects
- Subretinal hemorrhage
- Retinal detachment
- Cataract
- Inflammation (anterior chamber or vitreous)
- Injection-site 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)
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:
- Genetic: AAV5-hRKp.RPGR Low Dose CI brief — competitive landscape report
- Genetic: AAV5-hRKp.RPGR Low Dose updates RSS · CI watch RSS
- Janssen Research & Development, LLC portfolio CI
Frequently asked questions about Genetic: AAV5-hRKp.RPGR Low Dose
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Related
- Drug class: All Gene therapy (AAV vector) drugs
- Target: All drugs targeting RPGR gene; photoreceptor cells
- Manufacturer: Janssen Research & Development, LLC — full pipeline
- Therapeutic area: All drugs in Ophthalmology / Retinal Genetic Disorders
- Indication: Drugs for X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations
- Also known as: botaretigene sparoparvovec
- Compare: Genetic: AAV5-hRKp.RPGR Low Dose vs similar drugs
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