{"id":"genetic-aav5-hrkp-rpgr","safety":{"commonSideEffects":[{"rate":null,"effect":"Retinal detachment"},{"rate":null,"effect":"Cataract"},{"rate":null,"effect":"Endophthalmitis"},{"rate":null,"effect":"Retinal inflammation"},{"rate":null,"effect":"Visual disturbance"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"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.","oneSentence":"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.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:13:58.135Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations"}]},"trialDetails":[{"nctId":"NCT04671433","phase":"PHASE3","title":"Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene","status":"COMPLETED","sponsor":"Janssen Research & Development, LLC","startDate":"2020-12-04","conditions":"X-Linked Retinitis Pigmentosa","enrollment":105},{"nctId":"NCT04794101","phase":"PHASE3","title":"Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene","status":"ACTIVE_NOT_RECRUITING","sponsor":"Janssen Research & Development, LLC","startDate":"2020-12-04","conditions":"X-Linked Retinitis Pigmentosa","enrollment":97}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["botaretigene sparoparvovec"],"phase":"phase_3","status":"active","brandName":"Genetic: AAV5-hRKp.RPGR","genericName":"Genetic: AAV5-hRKp.RPGR","companyName":"Janssen Research & Development, LLC","companyId":"johnson-johnson","modality":"Biologic","firstApprovalDate":"","aiSummary":"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.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}