Polynucleotide agents targeting serpinc1 (AT3) and methods of use thereof

a technology of polynucleotide agents and serpinc1 is applied in the field of polynucleotide agents targeting serpinc1 (at3), which can solve the problems of refractory to replacement coagulation factor, difficult treatment of bleeds in such subjects, and inability to properly control bleeds, etc., to achieve stabilization of hemoglobin levels, decrease in intravascular hemolysis, and decrease in serpinc1 protein levels
US20200102560A1Inactive Publication Date: 2020-04-02GENZYME CORP
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Patent Information

Authority / Receiving Office
US · United States
Patent Type
Applications(United States)
Current Assignee / Owner
GENZYME CORP
Publication Date
2020-04-02
Estimated Expiration
Not applicable · inactive patent

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Abstract

The invention relates to polynucleotide agents targeting the Serpinc1 (AT3) gene, and methods of using such polynucleotide agents to inhibit expression of Serpinc1 and to treat subjects having a bleeding disorder, e.g., a hemophilia.
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Description

RELATED APPLICATIONS

[0001] This is a continuation application of U.S. patent application Ser. No. 15 / 499,981, filed on Apr. 28, 2017, which is a 35 § U.S.C. 111(a) continuation application which claims the benefit of priority to PCT / US2015 / 057717, filed on Oct. 28, 2015, which claims priority to U.S. Provisional Application No. 62 / 072,686, filed on Oct. 30, 2014. The entire contents of each of the foregoing applications are incorporated herein by reference.SEQUENCE LISTING

[0002] The instant application contains a Sequence Listing which has been submitted electronically in ASCII format and is hereby incorporated by reference in its entirety. Said ASCII copy, created on May 8, 2019, is named 117811_02503_SL and is 299,735 bytes in size.BACKGROUND OF THE INVENTION

[0003] Serpinc1 or antithrombin III (AT3) is a member of the serine proteinase inhibitor (serpin) superfamily Serpinc1 is a plasma protease inhibitor that inhibits thrombin as well as other activated serine proteases of the coagu...

Claims

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