324
Views
66
CrossRef citations to date
0
Altmetric
Review

An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B

&
Pages 135-140 | Published online: 22 Aug 2018

References

  • MannucciPMTuddenhamEGThe hemophilias–from royal genes to gene therapyN Engl J Med2001344231773177911396445
  • SrivastavaABrewerAKMauser-BunschotenEPGuidelines for the management of hemophiliaHaemophilia2013191e1e4722776238
  • OladapoAOEpsteinJDWilliamsEItoDGringeriAValentinoLAHealth-related quality of life assessment in haemophilia patients on prophylaxis therapy: a systematic review of results from prospective clinical trialsHaemophilia2015215e344e35826390060
  • GouwSCvan den BergHMFischerKIntensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN studyBlood2013121204046405523553768
  • ShueyDJMccallusDEGiordanoTRNAi: gene-silencing in therapeutic interventionDrug Discov Today20027201040104612546893
  • GaoKHuangLAchieving efficient RNAi therapy: progress and challengesActa Pharm Sin B201334213225
  • HausseckerDCurrent issues of RNAi therapeutics delivery and developmentJ Control Release2014195495425111131
  • ShettySVoraSKulkarniBContribution of natural anticoagulant and fibrinolytic factors in modulating the clinical severity of haemophilia patientsBr J Haematol2007138454154417659055
  • FranchiniMMontagnanaMTargherGInterpatient phenotypic inconsistency in severe congenital hemophilia: a systematic review of the role of inherited thrombophiliaSemin Thromb Hemost200935330731219452406
  • BolligerDSzlamFSuzukiNMatsushitaTTanakaKAHeterozygous antithrombin deficiency improves in vivo haemostasis in factor VIII-deficient miceThromb Haemost201010361233123820352163
  • PatnaikMMMollSInherited antithrombin deficiency: a reviewHaemophilia20081461229123919141163
  • SehgalABarrosSIvanciuLAn RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophiliaNat Med201521549249725849132
  • NairJKWilloughbyJLChanAMultivalent N-acetylgalactos-amine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencingJ Am Chem Soc201413649169581696125434769
  • RagniMVTargeting antithrombin to treat hemophiliaN Engl J Med2015373438939126200986
  • RagniMVGeorgievPMantTFitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia: updated results from a Phase 1 and Phase 1,2 extension study in patients without inhibitorsBlood20161282572
  • PasiKJGeorgievPMantTFitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia: updated results from a phase 1 and phase 1,2 extension study in patients with inhibitorsBlood20161281397
  • PasiKJRangarajanSGeorgievPTargeting of antithrombin in hemophilia A or B with RNAi therapyN Engl J Med2017377981982828691885
  • PasiKJGeorgievPFitusiranMTAn investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia: interim results from a phase 2 extension study in patients with hemophilia A or B with and without inhibitorsRes Pract Thromb Haemost20171Suppl 125
  • RochaPCarvalhoMLopesMAraújoFCosts and utilization of treatment in patients with hemophiliaBMC Health Serv Res20151548426502954
  • AlnylamAlnylam Reports Patient Death in Fitusiran Clinical StudyNational Hemophilia Foundation2017 Available from: https://www.hemophilia.org/Newsroom/Industry-News/Alnylam-Reports-Patient-Death-in-Fitusiran-Clinical-StudyAccessed August 8, 2018
  • QianKLiuJHuangSAkinAThrombin generation response with the addition of bypassing agents in plasma of patients treated with fitusiranHemophilia201824S5MP-219
  • NegrierCRagniMVGeorgievPPerioperative management in patients with hemophilia receiving fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophiliaBlood2017130Suppl 12366
  • SridharanGLiuJQianKGoelVHuangSAkincAIn silico modeling of the impact of antithrombin lowering on thrombin generation in rare bleeding disordersBlood20171303659
  • LiuJQianKHuangSColbergTEffect of antithrombin lowering on thrombin generation in rare bleeding disorder patient plasmaHemophilia201824S5TP-121