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IONS Ionis Pharmaceuticals

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
GRANT
Utility
Conjugated antisense compounds and their use
14 Sep 21
The present disclosure provides duplexes comprising a first oligomeric compound and a second oligomeric compound wherein the second oligomeric compound comprises a conjugate group.
Punit P. Seth, Michael Oestergaard, Eric E. Swayze
Filed: 26 Sep 16
GRANT
Utility
Modulation of angiopoietin-like 3 expression
14 Sep 21
Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal.
Susan M. Freier, Mark J. Graham, Rosanne M. Crooke
Filed: 7 Sep 18
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
GRANT
Utility
Compositions for modulating Ataxin 2 expression
7 Sep 21
Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression.
Susan M. Freier
Filed: 17 Apr 19
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
GRANT
Utility
Oligomer-conjugate complexes and their use
31 Aug 21
Oligonucleotides, chemically-modified oligonucleotides, and oligonucleotide-conjugate complexes for use in research, diagnostics, and/or therapeutics are described herein.
Thazha P. Prakash, Eric E. Swayze, Walter F. Lima, Garth A. Kinberger
Filed: 18 Jun 18
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
GRANT
Utility
Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
10 Aug 21
The present invention provides modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom.
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
Filed: 30 Aug 18
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
GRANT
Utility
Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
3 Aug 21
The present invention provides compositions comprising at least one oligomeric compound comprising an alternating motif and further include a region that is complementary to a nucleic acid target.
Charles Allerson, Balkrishen Bhat, Anne B. Eldrup, Muthiah Manoharan, Richard H. Griffey, Brenda F. Baker, Eric E. Swayze
Filed: 26 Feb 19
GRANT
Utility
Compounds and methods for reducing ATXN2 expression
3 Aug 21
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 protein in a cell or animal.
Paymaan Jafar-nejad
Filed: 25 Jul 19
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
GRANT
Utility
Compositions and their uses directed to huntingtin
27 Jul 21
Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal.
Susan M. Freier
Filed: 15 Apr 20
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