{"id":"eb-101","safety":{"commonSideEffects":[{"rate":null,"effect":"Infusion-related reactions"},{"rate":null,"effect":"Hematologic toxicity"},{"rate":null,"effect":"Infection"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"EB-101 uses a lentiviral vector to transduce patient hematopoietic stem cells ex vivo with the ABCD1 gene, which encodes the adrenoleukodystrophy protein responsible for transporting very long-chain fatty acids. The modified cells are then reinfused into the patient, where they engraft and produce functional protein to prevent or slow neurological deterioration in cerebral adrenoleukodystrophy.","oneSentence":"EB-101 is a gene therapy that delivers a functional copy of the ABCD1 gene to restore adrenoleukodystrophy protein function in patients with cerebral adrenoleukodystrophy.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:10:32.134Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Cerebral adrenoleukodystrophy (CALD)"}]},"trialDetails":[{"nctId":"NCT05725018","phase":"PHASE3","title":"A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients","status":"ACTIVE_NOT_RECRUITING","sponsor":"Abeona Therapeutics, Inc","startDate":"2023-04-02","conditions":"Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, RDEB","enrollment":12},{"nctId":"NCT05708677","phase":"","title":"A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB","status":"ENROLLING_BY_INVITATION","sponsor":"Abeona Therapeutics, Inc","startDate":"2021-02-09","conditions":"RDEB","enrollment":22},{"nctId":"NCT03183934","phase":"","title":"A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical Trial","status":"COMPLETED","sponsor":"Anterogen Co., Ltd.","startDate":"2017-12-08","conditions":"Dystrophic Epidermolysis Bullosa","enrollment":2},{"nctId":"NCT04227106","phase":"PHASE3","title":"Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)","status":"COMPLETED","sponsor":"Abeona Therapeutics, Inc","startDate":"2020-01-10","conditions":"Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa","enrollment":11}],"_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":["pz-cel","LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]"],"phase":"phase_3","status":"active","brandName":"EB-101","genericName":"EB-101","companyName":"Abeona Therapeutics, Inc","companyId":"abeona-therapeutics-inc","modality":"Biologic","firstApprovalDate":"","aiSummary":"EB-101 is a gene therapy that delivers a functional copy of the ABCD1 gene to restore adrenoleukodystrophy protein function in patients with cerebral adrenoleukodystrophy. Used for Cerebral adrenoleukodystrophy (CALD).","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":1,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}