{"id":"genetic-aav5-hrkp-rpgr-low-dose","safety":{"commonSideEffects":[{"rate":null,"effect":"Subretinal hemorrhage"},{"rate":null,"effect":"Retinal detachment"},{"rate":null,"effect":"Cataract"},{"rate":null,"effect":"Inflammation (anterior chamber or vitreous)"},{"rate":null,"effect":"Injection-site complications"}]},"_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 slow photoreceptor degeneration. The low-dose formulation represents a reduced viral vector dose compared to higher-dose variants.","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":"2026-03-28T01:13:58.102Z","_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":14567,"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 Low Dose","genericName":"Genetic: AAV5-hRKp.RPGR Low 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}}