358 patents
Page 5 of 18
Utility
Compositions and methods for modulation of protein aggregation
8 Nov 22
In certain embodiments, the present disclosure provides methods comprising contacting a cell with a compound comprising a modified oligonucleotide complementary to a nucleic acid transcript.
C. Frank Bennett, Xue-hai Liang, Wen Shen
Filed: 7 Aug 18
Utility
Compositions and Methods for Modulating Angiopoietin-like 3 Expression
13 Oct 22
Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal.
Thazha P. PRAKASH, Punit P. SETH, Eric E. SWAYZE, Susan M. FREIER, Mark J. GRAHAM, Rosanne M. CROOKE
Filed: 1 Dec 20
Utility
Compounds and Methods for Reducing Snca Expression
6 Oct 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease.
Holly Kordasiewicz, Priyam Singh, Susan M. Freier, Tracy A. Cole
Filed: 9 Nov 21
Utility
Modulators of YAP1 Expression
6 Oct 22
The present embodiments provide methods, compounds, and compositions useful for inhibiting YAP1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with YAP1.
Youngsoo Kim, Xiaolin Luo, Robert MacLeod, Susan M. Freier, Huynh-Hoa Bui
Filed: 16 Nov 21
Utility
Modulation of frataxin expression
4 Oct 22
Certain embodiments are directed to methods and compounds for increasing FXN.
Frank Rigo, Thazha P. Prakash, David Corey
Filed: 21 Dec 18
Utility
Compounds and Methods for Modulating Angiotensinogen Expression
22 Sep 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of AGT RNA in a cell or subject, and in certain instances reducing the amount of AGT in a cell or subject.
Adam Mullick, Susan M. Freier
Filed: 2 Jun 22
Utility
Compounds and methods for modulating angiotensinogen expression
20 Sep 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of AGT RNA in a cell or subject, and in certain instances reducing the amount of AGT in a cell or subject.
Adam Mullick, Susan M. Freier
Filed: 18 Nov 21
Utility
2'-METHOXY Substituted Oligomeric Compounds and Compositions for Use In Gene Modulations
15 Sep 22
Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification.
Stanley T. Crooke
Filed: 3 Nov 21
Utility
Compounds and Methods for Modulating ATXN1
15 Sep 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN1 RNA in a cell or subject, and in certain instances reducing the amount of ATXN1 in a cell or subject.
Keming Zhou
Filed: 23 May 22
Utility
Compounds and Methods for Reducing Spdef Expression
15 Sep 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SPDEF RNA in a cell or subject, and in certain instances reducing the amount of SPDEF protein in a cell or subject.
Susan M. Freier, Huynh-Hoa Bui, Shuling Guo, Jeffrey R. Crosby
Filed: 22 Oct 21
Utility
Trans-cyclooctene Labeled Antisense Oligonucleotides, Radio Labeled Tetrazine and Methods
15 Sep 22
Featured are trans-cyclooctene and tetrazine compounds.
Brendon Ellery COOK, Maciej Adam KALISZCZAK, Laurent MARTARELLO, Nathan Edward GENUNG, Sivaraman DANDAPANI, Eric Edward SWAYZE, William John DRURY, III, Chrissa Ann DWYER
Filed: 19 Aug 20
Utility
Compounds and Methods for Modulating CLN3 Expression
8 Sep 22
Provided are compounds, methods, and pharmaceutical compositions for modulating the expression of CLN3 RNA in a cell or animal, and in certain instances modulating the expression of CLN3 protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease.
Michelle L. Hastings, Frank Rigo
Filed: 10 Sep 19
Utility
Compounds and methods for reducing ATXN3 expression
6 Sep 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 RNA in a cell or animal, and in certain embodiments reducing the amount of ATXN3 protein in a cell or animal.
Susan M. Freier
Filed: 9 May 19
Utility
Modulators of HSD17B13 Expression
1 Sep 22
Methods, compounds, and compositions useful for inhibiting HSD expression are provided.
Susan M. Freier, Susan F. MURRAY
Filed: 20 Dec 19
Utility
CorrectedCompositions and Methods for Modulating TTR Expression
1 Sep 22
Provided herein are oligomeric compounds with conjugate groups.
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
Filed: 13 Feb 20
Utility
Modulation of Huntington expression
23 Aug 22
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: 12 Oct 20
Utility
Compounds and Methods for Modulating TMPRSS6 Expression
18 Aug 22
Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof.
Shuling Guo, Mariam Aghajan, Eric E. Swayze
Filed: 24 Sep 21
Utility
Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
16 Aug 22
The present disclosure provides compounds comprising modified oligonucleotides for use in CRISPR.
Meghdad Rahdar, Thazha P. Prakash, Eric E. Swayze, C. Frank Bennett
Filed: 29 Jun 16
Utility
Synthesis of Oligomeric Compounds Comprising Phosphorothioate Diester and Phosphodiester Linkages
11 Aug 22
The present disclosure provides methods for synthesizing oligomeric compounds having at least one phosphorothioate diester linkage and at least one phosphate diester internucleoside linkage.
Andrew K. McPherson, Andrew A. Rodriguez, Daniel C. Capaldi
Filed: 15 May 20
Utility
Methods and Compositions for Inhibiting PMP22 Expression
11 Aug 22
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: 4 Oct 21