{"id":"cam-101","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Transient liver enzyme elevation"},{"rate":null,"effect":"Immune responses to AAV vector"}]},"_chembl":{"chemblId":"CHEMBL386272","moleculeType":"Small molecule","molecularWeight":"519.04"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"CAM-101 uses an adeno-associated virus (AAV) vector to introduce a working CDKN1C gene into patients with loss-of-function mutations or epigenetic defects affecting this tumor suppressor gene. CDKN1C encodes p57, a cyclin-dependent kinase inhibitor critical for growth regulation and genomic imprinting. By restoring functional p57 expression, the therapy aims to normalize cell growth control and reduce overgrowth and cancer predisposition associated with imprinting disorders.","oneSentence":"CAM-101 is a gene therapy that delivers a functional copy of the CDKN1C gene to treat Beckwith-Wiedemann syndrome and related imprinting disorders.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:04:43.397Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Beckwith-Wiedemann syndrome"},{"name":"CDKN1C-related imprinting disorders"}]},"trialDetails":[{"nctId":"NCT06903611","phase":"PHASE3","title":"Phase III Study of Topical Fibrinogen-Depleted Human Platelet Lysate Compared to Placebo for Moderate to Severe Dry Eye","status":"NOT_YET_RECRUITING","sponsor":"Cambium Bio Limited","startDate":"2025-09-30","conditions":"Dry Eye Disease, Dry Eye, Dry Eyes Chronic","enrollment":400},{"nctId":"NCT03414645","phase":"PHASE1, PHASE2","title":"Topical Fibrinogen-Depleted Human Platelet Lysate in Patients With Dry Eye Secondary to Graft vs. Host Disease","status":"COMPLETED","sponsor":"Cambium Bio Limited","startDate":"2018-05-01","conditions":"Dry Eye, Graft-versus-host-disease, Ocular Discomfort","enrollment":64}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":1,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Elate Ocular","FD hPL"],"phase":"phase_3","status":"active","brandName":"CAM-101","genericName":"CAM-101","companyName":"Cambium Bio Limited","companyId":"cambium-bio-limited","modality":"Small molecule","firstApprovalDate":"","aiSummary":"CAM-101 is a gene therapy that delivers a functional copy of the CDKN1C gene to treat Beckwith-Wiedemann syndrome and related imprinting disorders. Used for Beckwith-Wiedemann syndrome, CDKN1C-related imprinting disorders.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}