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Genetic: 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 | Genetic: AAV5-hRKp.RPGR |
|---|---|
| Also known as | botaretigene sparoparvovec |
| Sponsor | Janssen Research & Development, LLC |
| Drug class | Gene therapy (AAV vector) |
| Target | RPGR (retinitis pigmentosa GTPase regulator) gene |
| Modality | Biologic |
| Therapeutic area | Ophthalmology / Rare Genetic Disorders |
| Phase | Phase 3 |
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 and retinal cells, enabling them to produce functional RPGR protein and halt or slow photoreceptor degeneration. This addresses the underlying genetic defect causing progressive vision loss in XLRP patients.
Approved indications
- X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations
Common side effects
- Retinal detachment
- Cataract
- Endophthalmitis
- Retinal inflammation
- Visual disturbance
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 |