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Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
K. John Pasi ,
Savita Rangarajan ,
Pencho Georgiev ,
Tim Mant ,
Michael D. Creagh ,
Toshko Lissitchkov ,
David Bevan ,
Steve Austin ,
Charles R. Hay ,
Inga Hegemann ,
Rashid Kazmi ,
Pratima Chowdary ,
Liana Gercheva-Kyuchukova ,
Vasily Mamonov ,
Margarita Timofeeva ,
Chang-Heok Soh ,
Pushkal Garg ,
Akshay Vaishnaw ,
Akin Akinc ,
Benny Sørensen ,
Margaret V. Ragni
August 31 2017
August 31 2017
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Abstract
Current hemophilia treatment involves frequent intravenous infusions of clotting factors,
which is associated with variable hemostatic protection, a high treatment burden,
and a risk of the development of inhibitory alloantibodies. Fitusiran, an investigational
RNA interference (RNAi) therapy that targets antithrombin (encoded by SERPINC1), is
in development to address these and other limitations.