{"id":"genetic-aav5-hrkp-rpgr-intermediate-dose","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 (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 reverse photoreceptor degeneration. This addresses the genetic defect underlying X-linked retinitis pigmentosa, a progressive inherited retinal dystrophy.","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":"2026-03-28T01:13:58.286Z","_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":187,"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 Intermediate Dose","genericName":"Genetic: AAV5-hRKp.RPGR Intermediate Dose","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}}