{"id":"aav2-hrpe65v2-voretigene-neparvovec-rzyl","safety":{"commonSideEffects":[{"rate":null,"effect":"Conjunctival hyperemia"},{"rate":null,"effect":"Eye pain or discomfort"},{"rate":null,"effect":"Photopsia (flashing lights)"},{"rate":null,"effect":"Anterior chamber inflammation"},{"rate":null,"effect":"Cataract progression"}]},"_chembl":{"chemblId":"CHEMBL3707275","moleculeType":"Gene"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"The drug uses an adeno-associated virus serotype 2 (AAV2) vector to deliver the normal RPE65 gene into the eye. RPE65 encodes a protein essential for the visual cycle, which converts vitamin A into the light-sensitive form needed for photoreceptor function. By restoring RPE65 protein production, the therapy enables photoreceptors to respond to light again, thereby restoring or improving vision in patients with RPE65-mediated inherited retinal disease.","oneSentence":"AAV2-hRPE65v2 is a gene therapy that delivers a functional copy of the human RPE65 gene into retinal pigment epithelium cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T00:58:28.714Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Inherited retinal dystrophy caused by biallelic RPE65 mutations (Leber congenital amaurosis type 2)"}]},"trialDetails":[{"nctId":"NCT03597399","phase":"","title":"A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US","status":"COMPLETED","sponsor":"Spark Therapeutics, Inc.","startDate":"2019-01-10","conditions":"Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy","enrollment":87},{"nctId":"NCT01208389","phase":"PHASE1, PHASE2","title":"Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2","status":"ACTIVE_NOT_RECRUITING","sponsor":"Spark Therapeutics, Inc.","startDate":"2010-11","conditions":"Leber Congenital Amaurosis","enrollment":12},{"nctId":"NCT00999609","phase":"PHASE3","title":"Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis","status":"ACTIVE_NOT_RECRUITING","sponsor":"Spark Therapeutics, Inc.","startDate":"2012-10","conditions":"Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis","enrollment":31},{"nctId":"NCT03602820","phase":"","title":"Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Spark Therapeutics, Inc.","startDate":"2015-06","conditions":"Inherited Retinal Dystrophy Due to RPE65 Mutations","enrollment":41},{"nctId":"NCT00516477","phase":"PHASE1","title":"Safety Study in Subjects With Leber Congenital Amaurosis","status":"COMPLETED","sponsor":"Spark Therapeutics, Inc.","startDate":"2007-09","conditions":"Leber Congenital Amaurosis","enrollment":12}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":2,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["AAV2-hRPE65v2","voretigene neparvovec","gene therapy vector","voretigene neparvovec-rzyl"],"phase":"phase_3","status":"active","brandName":"AAV2-hRPE65v2,voretigene neparvovec-rzyl","genericName":"AAV2-hRPE65v2,voretigene neparvovec-rzyl","companyName":"Spark Therapeutics, Inc.","companyId":"spark-therapeutics-inc","modality":"Biologic","firstApprovalDate":"","aiSummary":"AAV2-hRPE65v2 is a gene therapy that delivers a functional copy of the human RPE65 gene into retinal pigment epithelium cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations. Used for Inherited retinal dystrophy caused by biallelic RPE65 mutations (Leber congenital amaurosis type 2).","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":1,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}