{"id":"gs010","safety":{"commonSideEffects":[{"rate":null,"effect":"Conjunctival hyperemia"},{"rate":null,"effect":"Eye pain or discomfort"},{"rate":null,"effect":"Anterior chamber inflammation"},{"rate":null,"effect":"Elevated intraocular pressure"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"GS010 uses an adeno-associated virus (AAV) vector to deliver a wild-type ND4 gene into the mitochondria of retinal ganglion cells. The ND4 gene encodes a subunit of Complex I in the electron transport chain; mutations in ND4 cause energy depletion and cell death in the optic nerve. By restoring functional ND4 protein, the therapy aims to restore mitochondrial respiration and prevent or reverse vision loss.","oneSentence":"GS010 is a gene therapy that delivers a functional copy of the ND4 gene to restore mitochondrial function in retinal cells affected by Leber hereditary optic neuropathy.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T00:47:40.379Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Leber hereditary optic neuropathy (LHON) due to ND4 mutation"}]},"trialDetails":[{"nctId":"NCT02652780","phase":"PHASE3","title":"Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)","status":"COMPLETED","sponsor":"GenSight Biologics","startDate":"2016-01","conditions":"Optic, Atrophy, Hereditary, Leber","enrollment":37},{"nctId":"NCT02064569","phase":"PHASE1, PHASE2","title":"Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients","status":"COMPLETED","sponsor":"GenSight Biologics","startDate":"2014-02-13","conditions":"Leber Hereditary Optic Neuropathy","enrollment":19},{"nctId":"NCT07303296","phase":"PHASE2","title":"Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients","status":"RECRUITING","sponsor":"GenSight Biologics","startDate":"2026-01-22","conditions":"Leber Hereditary Optic Disease","enrollment":14},{"nctId":"NCT03672968","phase":"","title":"EAP_GS010_single Patient","status":"NO_LONGER_AVAILABLE","sponsor":"GenSight Biologics","startDate":"","conditions":"Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)","enrollment":""},{"nctId":"NCT02652767","phase":"PHASE3","title":"Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation","status":"COMPLETED","sponsor":"GenSight Biologics","startDate":"2016-02-23","conditions":"Optic, Atrophy, Hereditary, Leber","enrollment":39},{"nctId":"NCT03406104","phase":"PHASE3","title":"RESCUE and REVERSE Long-term Follow-up","status":"COMPLETED","sponsor":"GenSight Biologics","startDate":"2018-01-09","conditions":"Leber Hereditary Optic Neuropathy","enrollment":62},{"nctId":"NCT03293524","phase":"PHASE3","title":"Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year","status":"COMPLETED","sponsor":"GenSight Biologics","startDate":"2018-03-12","conditions":"Leber Hereditary Optic Neuropathy","enrollment":98}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":3,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Lenadogene Nolparvovec","rAAV2/2-ND4"],"phase":"phase_3","status":"active","brandName":"GS010","genericName":"GS010","companyName":"GenSight Biologics","companyId":"gensight-biologics","modality":"Biologic","firstApprovalDate":"","aiSummary":"GS010 is a gene therapy that delivers a functional copy of the ND4 gene to restore mitochondrial function in retinal cells affected by Leber hereditary optic neuropathy. Used for Leber hereditary optic neuropathy (LHON) due to ND4 mutation.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":5,"withResults":3},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}