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IONS Ionis Pharmaceuticals

APP
Utility
Compounds and Methods for Modulation of Transcript Processing
14 Oct 21
Provided herein are methods, compounds, and compositions for modulation of transcript processing.
Thazha P. Prakash, Frank Rigo, Punit P. Seth
Filed: 18 Nov 20
APP
Utility
Modulation of LNC05 Expression
23 Sep 21
Provided herein are methods, compounds, and compositions for reducing expression of lnc05 in a cell or individual.
Allen Tingjin Yu, David L. Spector, Frank Rigo, Susan M. Freier, Jan Bergmann, Carmen Berasain
Filed: 3 Jun 21
APP
Utility
Method of Conjugating Oligomeric Compounds
16 Sep 21
Provided herein are solid phase methods for the synthesis of conjugated oligomeric compounds and intermediates used in such methods.
Isaiah E. Cedillo, Recaldo Carty
Filed: 6 Oct 17
APP
Utility
Methods for Treating Hypercholesterolemia
16 Sep 21
Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C.
Susan M. Freier, Rosanne M. Crooke, Mark J. Graham, Kristina M. Lemonidis, Diane Tribble, Sanjay Bhanot, Andrew T. Watt
Filed: 25 Sep 20
APP
Utility
Methods for Safely Reducing Thrombopoietin
9 Sep 21
Provided herein are methods, compounds, and compositions for safely reducing thrombopoietin in a cell or an individual.
Andras Gruber, Erik Tucker, Brett P. Monia, Alexey Revenko
Filed: 16 Jul 19
APP
Utility
Compounds and Methods for Modulating UBE3A-ATS
9 Sep 21
Susan M. Freier, Huynh-Hoa Bui, Paymaan Jafar-nejad, Frank Rigo
Filed: 21 Apr 21
APP
Utility
Compositions and Methods for Modulating PKK Expression
9 Sep 21
Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression.
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Susan M. Freier, Huynh-Hoa Bui
Filed: 11 May 20
APP
Utility
Compounds and Methods for the Modulation of Proteins
2 Sep 21
In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell.
Stanley T. Crooke, Xue-hai Liang, Wen Shen
Filed: 15 Sep 20
APP
Utility
Modified Compounds and Uses Thereof
26 Aug 21
The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications.
Punit P. Seth, Michael Oestergaard, Michael T. Migawa, Xue-hai Liang, Wen Shen, Stanley T. Crooke, Eric E. Swayze
Filed: 10 Feb 21
APP
Utility
Compounds and Methods for Increasing STMN2 Expression
19 Aug 21
Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal.
Huynh-Hoa Bui, Don W. Cleveland, Ze'ev Melamed
Filed: 14 Jun 19
APP
Utility
Linkage Modified Oligomeric Compounds
19 Aug 21
The present invention provides gapped oligomeric compounds comprising from 1 to about 3 internucleoside linkages having one of formulas I to XVI.
Michael T. Migawa, Eric E. Swayze, Punit P. Seth
Filed: 17 Jun 19
APP
Utility
Selective Antisense Compounds and Uses Thereof
5 Aug 21
The present invention provides oligomeric compounds.
Punit P. Seth, Michael Oestergaard, Eric E. Swayze
Filed: 3 Sep 20
APP
Utility
Compositions for Modulating C9ORF72 Expression
29 Jul 21
Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal.
Frank Rigo
Filed: 18 Sep 20
APP
Utility
Compositions for Modulating Expression of C9ORF72 Antisense Transcript
15 Jul 21
Disclosed herein are compositions and methods for reducing expression of C9ORF72 antisense transcript in an animal with C9ORF72 antisense transcript specific inhibitors.
Frank Rigo
Filed: 27 Aug 20
APP
Utility
Compositions and Methods for Modulating Growth Hormone Receptor Expression
8 Jul 21
The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with excess growth hormone using antisense compounds or oligonucleotides targeted to growth hormone receptor (GHR).
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Sanjay Bhanot, Susan M. Freier, Huynh-Hoa Bui
Filed: 4 Aug 20
APP
Utility
MODULATORS OF ENaC EXPRESSION
17 Jun 21
The present embodiments provide methods, compounds, and compositions useful for inhibiting ENaC expression, which may be useful for treating, preventing, or ameliorating a disease associated with ENaC.
Jeffrey R. Crosby, Shuling Guo, Huynh-Hoa Bui, Andrew T. Watt, Susan M. Freier
Filed: 31 Oct 18
APP
Utility
Methods for Reducing C9ORF72 Expression
10 Jun 21
Provided are methods for reducing the amount or activity of C9ORF72 RNA, and in certain instances of reducing the amount of C9ORF72 protein, in an animal.
C. Frank Bennett, Frank Rigo, Don W. Cleveland, Jie Jiang, Qiang Zhu, Clotilde Lagier-Tourene
Filed: 23 Jul 20
APP
Utility
Conjugated Antisense Compounds for Use in Therapy
10 Jun 21
Provided herein are methods of administering gapmer oligomeric compounds with GalNAc conjugate groups to a human.
Nicholas J. Viney, Richard S. Geary, Yanfeng Wang, Zhengrong Yu, Rudy Gunawan
Filed: 28 Jul 20
APP
Utility
Modulation of Factor 11 Expression
10 Jun 21
Disclosed herein are antisense compounds and methods for decreasing Factor 11 and treating or preventing thromboembolic complications in an individual in need thereof.
Susan M. Freier, Chenguang Zhao, Brett P. Monia, Hong Zhang, Jeffrey R. Crosby, Andrew M. Siwkowski
Filed: 29 Jul 20
APP
Utility
Methods of Modulating Antisense Activity
10 Jun 21
Disclosed herein are methods for increasing antisense activity by modulating translation.
Xue-hai Liang, Stanley T. Crooke
Filed: 16 Nov 18
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