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IONS Ionis Pharmaceuticals

188 patents

Page 5 of 10
APP
Utility
Conjugated Antisense Compounds and Their Use
16 Dec 20
The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, wherein the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound.
Michael Oestergaard, Punit P. Seth, Frank Rigo, Chrissa A. Dwyer
Filed: 13 Dec 18
APP
Utility
Modulators of DNM2 Expression
16 Dec 20
The present embodiments provide methods, compounds, and compositions useful for inhibiting DNM2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DNM2.
Susan M. FREIER, Huynh-Hoa BUI, Susan F. MURRAY, Brett P. MONIA, Shuling GUO
Filed: 14 Jan 19
GRANT
Utility
Oligomeric compounds comprising α-β-constrained nucleic acid
14 Dec 20
The present disclosure provides oligomeric compounds comprising at least one α-β-constrained nucleic acid as provided herein.
Michael Oestergaard, Eric E. Swayze, Punit P. Seth
Filed: 3 Jan 19
GRANT
Utility
Modulators of DNM2 expression
14 Dec 20
The present embodiments provide methods, compounds, and compositions useful for inhibiting DNM2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DNM2.
Susan M. Freier
Filed: 14 Jan 19
APP
Utility
5' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
9 Dec 20
The present invention provides 5′ modified nucleosides and oligomeric compounds prepared therefrom.
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
Filed: 29 Apr 20
GRANT
Utility
Methods for treating hypercholesterolemia
7 Dec 20
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: 6 Aug 18
APP
Utility
Selective Reduction of Allelic Variants
2 Dec 20
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP).
C. Frank Bennett, Michael Hayden, Susan M. Freier, Sarah Greenlee, Jeffrey Carroll, Simon Warby, Eric E. Swayze
Filed: 13 Jan 20
GRANT
Utility
Modulation of SMN expression
30 Nov 20
Certain embodiments are directed to methods and compounds for inhibiting SMN-NAT, the natural antisense transcript of SMN.
Frank Rigo, C. Frank Bennett, Constantin Van Outryve D'Ydewalle, Charlotte J. Sumner
Filed: 10 Apr 16
GRANT
Utility
Conjugated antisense compounds and their use
23 Nov 20
Provided herein are oligomeric compounds with conjugate groups.
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
Filed: 25 Dec 18
APP
Utility
Modulating Apolipoprotein (A) Expression
18 Nov 20
Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)].
Nicholas J. Viney, Richard S. Geary, Yanfeng Wang, Zhengrong Yu
Filed: 29 Dec 19
APP
Utility
Modulation of Dystrophia Myotonica-Protein Kinase (DMPK) Expression
18 Nov 20
Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal.
C. Frank Bennett, Susan M. Freier, Robert A. MacLeod, Sanjay K. Pandey, Charles A. Thornton, Thurman Wheeler, Seng H. Cheng, Andrew Leger, Bruce M. Wentworth
Filed: 19 Dec 19
GRANT
Utility
Modulation of huntingtin expression
16 Nov 20
Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal.
Gene Hung, C. Frank Bennett, Susan M. Freier, Holly Kordasiewicz, Lisa Stanek, Don W. Cleveland, Seng H. Cheng, Lamya Shihabuddin
Filed: 25 Feb 20
GRANT
Utility
Compounds and methods for the modulation of proteins
2 Nov 20
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 Nov 15
GRANT
Utility
Modulation of alpha synuclein expression
26 Oct 20
Disclosed herein are antisense compounds and methods for decreasing alpha-synuclein mRNA and protein expression.
Susan M. Freier
Filed: 17 Apr 17
GRANT
Utility
Compositions for modulating C9ORF72 expression
26 Oct 20
Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal.
Frank Rigo
Filed: 16 Oct 18
APP
Utility
Compositions and Methods for Modulating Ataxin 3 Expression
14 Oct 20
Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides.
Frank RIGO, Thazha P. PRAKASH, Wilhelmina Maria Clasina VAN ROON-MOM, Lodewijk Julius Anton TOONEN
Filed: 25 Nov 19
APP
Utility
Cancer Treatment
7 Oct 20
In certain embodiments, methods, compounds, and compositions for treating B-cell lymphoma or hepatocellular carcinoma by inhibiting expression of STAT3 mRNA or protein in an animal are provided herein.
Stanley T. CROOKE, Mason YAMASHITA
Filed: 27 Feb 20
APP
Utility
Compositions and Their Uses Directed to Huntingtin
7 Oct 20
Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal.
Susan M. Freier
Filed: 14 Apr 20
GRANT
Utility
Compositions for modulating expression of C9ORF72 antisense transcript
5 Oct 20
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: 5 Jan 16
GRANT
Utility
Compositions and methods for modulating growth hormone receptor expression
5 Oct 20
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: 23 Jul 19
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