{"id":"abo-101","safety":{"commonSideEffects":[{"rate":null,"effect":"Infusion-related reactions"},{"rate":null,"effect":"Transient cytopenias"},{"rate":null,"effect":"Liver enzyme elevation"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"anthropic-haiku","explanation":"ABO-101 uses an adeno-associated virus (AAV) vector to deliver the wild-type FANCC gene into patient hematopoietic stem cells, restoring the function of the Fanconi anemia complementation complex. This correction allows bone marrow cells to repair DNA damage more effectively and reduces the risk of bone marrow failure and malignant transformation associated with Fanconi anemia type C.","oneSentence":"ABO-101 is a gene therapy that delivers a functional copy of the FANCC gene to correct Fanconi anemia complementation group C deficiency.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T02:28:27.392Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Fanconi anemia complementation group C (FA-C)"}]},"trialDetails":[{"nctId":"NCT06839235","phase":"PHASE1, PHASE2","title":"Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)","status":"RECRUITING","sponsor":"Arbor Biotechnologies","startDate":"2025-06-16","conditions":"Primary Hyperoxaluria Type 1 (PH1)","enrollment":23},{"nctId":"NCT04655911","phase":"","title":"A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101","status":"TERMINATED","sponsor":"Abeona Therapeutics, Inc","startDate":"2020-10-28","conditions":"Mucopolysaccharidosis III-B","enrollment":1}],"_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":[],"phase":"phase_2","status":"active","brandName":"ABO-101","genericName":"ABO-101","companyName":"Abeona Therapeutics, Inc","companyId":"abeona-therapeutics-inc","modality":"Biologic","firstApprovalDate":"","aiSummary":"ABO-101 is a gene therapy that delivers a functional copy of the FANCC gene to correct Fanconi anemia complementation group C deficiency. Used for Fanconi anemia complementation group C (FA-C).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}