{"id":"aav2-hrpe65v2","safety":{"commonSideEffects":[{"rate":"null","effect":"Vision improvement"}]},"_chembl":{"chemblId":"CHEMBL3707275","moleculeType":"Gene"},"_dailymed":null,"mechanism":{"_ai_source":"groq-llama-8b","explanation":"This gene therapy works by using an adeno-associated virus (AAV) to deliver a functional copy of the RPE65 gene to the retinal pigment epithelium (RPE) cells in the retina. The RPE65 gene is responsible for producing an enzyme that helps convert light into electrical signals in the retina. By delivering a healthy copy of this gene, AAV2-hRPE65v2 aims to restore vision in individuals with inherited retinal dystrophy caused by RPE65 gene mutations.","oneSentence":"AAV2-hRPE65v2 is a gene therapy that uses a viral vector to deliver a healthy copy of the RPE65 gene to the retina, treating inherited retinal dystrophy.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T02:20:19.488Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Leber congenital amaurosis type 10, inherited retinal dystrophy"}]},"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":14,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Voretigene Neparvovec-rzyl"],"phase":"phase_2","status":"active","brandName":"AAV2-hRPE65v2","genericName":"AAV2-hRPE65v2","companyName":"Spark Therapeutics, Inc.","companyId":"spark-therapeutics-inc","modality":"Biologic","firstApprovalDate":"","aiSummary":"AAV2-hRPE65v2 is a gene therapy that uses a viral vector to deliver a healthy copy of the RPE65 gene to the retina, treating inherited retinal dystrophy. Used for Leber congenital amaurosis type 10, inherited retinal dystrophy.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}