{"id":"aav5-hrkp-rpgr","safety":{"commonSideEffects":[{"rate":null,"effect":"Retinal inflammation"},{"rate":null,"effect":"Subretinal fluid accumulation"},{"rate":null,"effect":"Cataract progression"},{"rate":null,"effect":"Injection-related complications"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"anthropic-haiku","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 cells and restore the missing or defective RPGR protein, thereby halting or slowing photoreceptor degeneration and preserving or improving vision.","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":"2026-03-28T02:28:08.381Z","_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},{"nctId":"NCT05926583","phase":"PHASE3","title":"A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa","status":"RECRUITING","sponsor":"Janssen Pharmaceutical K.K.","startDate":"2023-09-12","conditions":"Retinitis Pigmentosa","enrollment":4},{"nctId":"NCT06646289","phase":"PHASE2","title":"A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Janssen Research & Development, LLC","startDate":"2024-10-10","conditions":"X-Linked Retinitis Pigmentosa","enrollment":24},{"nctId":"NCT04312672","phase":"","title":"Long-term Follow-up Gene Therapy Study for RPGR- XLRP","status":"COMPLETED","sponsor":"Janssen Research & Development, LLC","startDate":"2017-07-31","conditions":"X-Linked Retinitis Pigmentosa","enrollment":42},{"nctId":"NCT03252847","phase":"PHASE1, PHASE2","title":"Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)","status":"COMPLETED","sponsor":"MeiraGTx UK II Ltd","startDate":"2017-07-31","conditions":"X-Linked Retinitis Pigmentosa","enrollment":49}],"_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":["JNJ-74765340, botaretigene sparoparvovec"],"phase":"phase_2","status":"active","brandName":"AAV5-hRKp.RPGR","genericName":"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":3,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}