Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

In this phase 1 study, a chemically modified RNA interference therapy designed to target antithrombin was administered to participants with hemophilia A or B. Antithrombin levels decreased and the generation of thrombin increased.

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Bibliographic Details
Published inThe New England journal of medicine Vol. 377; no. 9; pp. 819 - 828
Main Authors Pasi, K. John, Rangarajan, Savita, Georgiev, Pencho, Mant, Tim, Creagh, Michael D, Lissitchkov, Toshko, Bevan, David, Austin, Steve, Hay, Charles R, Hegemann, Inga, Kazmi, Rashid, Chowdary, Pratima, Gercheva-Kyuchukova, Liana, Mamonov, Vasily, Timofeeva, Margarita, Soh, Chang-Heok, Garg, Pushkal, Vaishnaw, Akshay, Akinc, Akin, Sørensen, Benny, Ragni, Margaret V
Format Journal Article
LanguageEnglish
Published United States Massachusetts Medical Society 31.08.2017
Subjects
Adult
Alloantibodies
Antithrombin
Antithrombin III - antagonists & inhibitors
Antithrombins - blood
Body weight
Clotting
Dose-Response Relationship, Drug
Drug dosages
Healthy Volunteers
Hematology
Hemophilia
Hemophilia A - blood
Hemophilia A - therapy
Hemophilia B - blood
Hemophilia B - therapy
Humans
Injection
Injections, Subcutaneous
Intravenous administration
Liver
Male
Middle Aged
Pharmacodynamics
Plasma
Plasma levels
RNA-mediated interference
RNAi Therapeutics
Single-Blind Method
Thrombin
Thrombin - biosynthesis
Thromboembolism
Young Adult
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Summary:In this phase 1 study, a chemically modified RNA interference therapy designed to target antithrombin was administered to participants with hemophilia A or B. Antithrombin levels decreased and the generation of thrombin increased.
Bibliography:ObjectType-Article-1
SourceType-Scholarly Journals-1
ObjectType-Feature-2
ObjectType-News-3
content type line 23
ISSN:0028-4793
1533-4406
DOI:10.1056/NEJMoa1616569