{"id":"aln-at3sc","_chembl":null,"modality":"Small molecule","_dailymed":null,"mechanism":{"target":"Antithrombin (SERPINC1)","explanation":"ALN-AT3SC is an RNAi therapeutic that targets messenger RNA encoding antithrombin, a natural anticoagulant protein. By reducing antithrombin levels, the drug aims to rebalance the coagulation system in patients with hemophilia and other bleeding disorders, potentially reducing bleeding episodes and the need for factor replacement therapy.","oneSentence":"ALN-AT3SC uses RNA interference to silence the antithrombin gene, reducing antithrombin levels to promote blood clotting in bleeding disorders."},"_scrapedAt":"2026-03-28T05:52:50.587Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"_claudeStub":{"ts":1779123527,"model":"claude-sonnet-4-5-20250929"},"competitors":[{"name":"fitusiran","brand":null,"company":"Sanofi","rationale":"RNAi therapeutic targeting antithrombin for hemophilia treatment"},{"name":"emicizumab","brand":"Hemlibra","company":"Roche","rationale":"Bispecific antibody for hemophilia A prophylaxis"},{"name":"concizumab","brand":null,"company":"Novo Nordisk","rationale":"Anti-TFPI antibody for hemophilia prophylaxis"}],"description":"ALN-AT3SC is an investigational RNA interference (RNAi) therapeutic developed by Sanofi for the treatment of hemophilia and rare bleeding disorders. The drug is designed to silence antithrombin expression to rebalance hemostasis in patients with bleeding disorders. It is currently in Phase 1 clinical development.","indications":{"approved":[{"status":"investigational","condition":"Hemophilia","patientPopulation":"Adult patients with hemophilia"}]},"trialDetails":[{"nctId":"NCT03417245","phase":"PHASE3","title":"A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2018-03-01","conditions":"Hemophilia A, Hemophilia B","enrollment":120},{"nctId":"NCT03417102","phase":"PHASE3","title":"A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2018-02-14","conditions":"Hemophilia A, Hemophilia B","enrollment":60},{"nctId":"NCT02035605","phase":"PHASE1","title":"A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients","status":"COMPLETED","sponsor":"Sanofi","startDate":"2014-01-20","conditions":"Hemophilia A, Hemophilia B","enrollment":51}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"phase_1","status":"active","brandName":"ALN-AT3SC","genericName":"ALN-AT3SC","companyName":"Sanofi","companyId":"sanofi","firstApprovalDate":"","aiSummary":"ALN-AT3SC is an RNAi inhibitor that targets Antithrombin mRNA. It is being studied in clinical trials for the treatment of Hemophilia A and Hemophilia B.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":false,"trials":true,"score":3}}