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IONS Ionis Pharmaceuticals

GRANT
Utility
Compositions and methods for modulating Tau expression
26 Oct 21
Disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds.
Frank Rigo
Filed: 28 Mar 17
GRANT
Utility
Modified compounds and uses thereof
19 Oct 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
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Utility
Methods and compositions for inhibiting PMP22 expression
5 Oct 21
The present embodiments provide methods, compounds, and compositions useful for inhibiting PMP22 expression and for treating, preventing, or ameliorating a disease associated with PMP22.
Gene Hung, Holly Kordasiewicz, Hien Thuy Zhao, Eric E. Swayze
Filed: 9 Mar 17
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Utility
Compositions and methods for modulating MECP2 expression
28 Sep 21
Disclosed herein are compounds and methods for decreasing MECP2 mRNA and protein expression.
Susan M. Freier, Huda Y. Zoghbi, Ezequiel Sztainberg
Filed: 8 Aug 19
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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
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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
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
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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
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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
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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
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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
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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Utility
Compositions and methods for modulation of IKBKAP splicing
20 Jul 21
The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene.
C. Frank Bennett, Frank Rigo, Adrian R. Krainer, Rahul Sinha
Filed: 24 May 19
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Utility
Methods of treating breast cancer
13 Jul 21
Provided herein are methods, compounds, and compositions for reducing expression of a MaTAR in an animal.
David L. Spector, Sarah Daniela Diermeier, Frank Rigo, C. Frank Bennett, Susan M. Freier, Gayatri Arun, Kung-Chi Chang
Filed: 5 Dec 16
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Utility
Modulation of prekallikrein (PKK) expression
6 Jul 21
Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression.
Susan M. Freier, Huynh-Hoa Bui
Filed: 7 Sep 18
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Utility
Modulation of Lnc05 expression
15 Jun 21
Provided herein are methods, compounds, and compositions for reducing expression of Inc05 in a cell or individual.
Allen Tingjin Yu, David L. Spector, Frank Rigo, Susan M. Freier, Jan Bergmann, Carmen Berasain
Filed: 1 Dec 17
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Utility
Compounds and methods for reducing FXI expression
1 Jun 21
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of FXI RNA in a cell or subject, and in certain instances reducing the amount of FXI protein in a cell or subject.
Huynh-Hoa Bui
Filed: 6 Nov 20
GRANT
Utility
Allele specific modulators of P23H rhodopsin
25 May 21
The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject.
Susan F. Murray, Punit P. Seth, Michael L. McCaleb, Susan M. Freier, Priyam Singh
Filed: 28 Aug 19
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Utility
Modulators of PCSK9 expression
11 May 21
The present embodiments provide methods, compounds, and compositions useful for inhibiting PCSK9 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PCSK9.
Eric E. Swayze, Susan M. Freier, Huynh-Hoa Bui
Filed: 1 Nov 19
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Utility
Methods of modulating KEAP1
30 Mar 21
Provided herein are methods, compounds, and compositions for reducing expression of KEAP1 in animal.
Richard Lee, Jeffrey R. Crosby
Filed: 16 Mar 17
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