220 patents
Page 2 of 11
Utility
Modulators of Growth Hormone Receptor
11 May 23
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).
Sanjay Bhanot, Susan M. Freier, Huynh-Hoa Bui
Filed: 19 May 22
Utility
Methods for Treating Hypercholesterolemia
4 May 23
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: 15 Nov 22
Utility
Compounds and Methods for Modulating Splicing of Pre-mrna
20 Apr 23
Provided are compounds, methods, and pharmaceutical compositions for modulating splicing of a pre-mRNA in a cell or subject.
Frank Rigo, Paymaan Jafar-Nejad
Filed: 26 Feb 21
Utility
Compositions and Methods for Modulating Growth Hormone Receptor Expression
20 Apr 23
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: 7 Mar 22
Utility
Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
20 Apr 23
The present invention provides oligomeric compounds.
Susan M. Freier, Eric E. Swayze
Filed: 6 Apr 22
Utility
Compounds and Methods for Modulating KCNQ2
20 Apr 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of KCNQ2 RNA in a cell or subject, and in certain instances reducing the amount of Kv7.2 protein in a cell or subject.
Paymaan Jafar-nejad, Huynh-Hoa Bui
Filed: 5 Mar 21
Utility
Selective Antisense Compounds and Uses Thereof
13 Apr 23
The present invention provides oligomeric compounds.
Punit P. Seth, Michael Oestergaard, Eric E. Swayze
Filed: 4 Apr 22
Utility
Compounds and Methods for Reducing DMPK Expression
13 Apr 23
Provided are oligomeric compounds, methods, and pharmaceutical compositions for DMPK the amount or activity of DMPK RNA in a cell or animal, and in certain instances reducing the amount of DMPK protein in a cell or animal.
Frank Rigo, Chrissa A, Dwyer
Filed: 31 Aug 22
Utility
Compounds and Methods for Reducing LRRK2 Expression
13 Apr 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal.
Tracy A. Cole, Holly Kordasiewicz, Huynh-Hoa Bui, Susan M. Freier
Filed: 4 Apr 22
Utility
Compositions for Modulating C9ORF72 Expression
13 Apr 23
Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors.
Susan M. Freier, Frank Rigo, Priyam Singh
Filed: 15 Apr 22
Utility
Methods for Reducing HTT Expression
6 Apr 23
Provided herein are methods of administering ISIS 443139 for ameliorating Huntington's disease, reducing HTT RNA, reducing mHTT RNA, reducing HTT protein, or reducing mHTT protein in a human subject in need thereof.
C. Frank Bennett, Daniel A. Norris, Anne V. Smith, Roger Lane
Filed: 19 Feb 21
Utility
Oligomeric Compounds Comprising Backbone Constrained Macrocycles
30 Mar 23
Juan Carlos Salinas Hernandez, Stephen Hanessian, Eric E. Swayze, Punit P. Seth, Jenny Lorena Rico Duque, Graeme C. Freestone, Barbara Lugato, Rajasekaran Tamiselvan
Filed: 25 Aug 22
Utility
Modified Compounds and Uses Thereof
2 Mar 23
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: 17 Mar 22
Utility
Compounds and Methods for Reducing App Expression
23 Feb 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of APP RNA in a cell or animal, and in certain instances reducing the amount of APP protein in a cell or animal.
Huynh-Hoa Bui, Susan M. Freier, Hien Thuy Zhao, Holly Kordasiewicz, Ruben E. Valas, Thazha P. Prakash, W. Brad Wan
Filed: 28 Jul 21
Utility
Compositions and Methods for Modulating Complement Factor B Expression
23 Feb 23
The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Tamar R. Grossman, Michael L. McCaleb, Andrew T. Watt, Susan M. Freier
Filed: 7 May 21
Utility
Modulators of Diacyglycerol Acyltransferase 2 (DGAT2)
16 Feb 23
The present embodiments provide methods, compounds, and compositions useful for inhibiting DGAT2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DGAT2.
Sanjay Bhanot, Susan M. Freier, Eric E. Swayze
Filed: 22 Feb 22
Utility
Compounds and Methods for Modulating SCN1A Expression
9 Feb 23
Provided are compounds, methods, and pharmaceutical compositions for modulating SCN1A RNA and/or protein in a cell or subject.
Paymaan Jafar-Nejad, Susan M. Freier, Frank Rigo
Filed: 26 Feb 21
Utility
Compositions and Methods for Modulating Ataxin 3 Expression
9 Feb 23
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: 23 Feb 22
Utility
Compounds and Methods for Reducing IFNAR1 Expression
26 Jan 23
Provided are oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of IFNAR1 RNA in a cell or animal, and in certain instances reducing the amount of IFNAR1 protein in a cell or animal Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat diseases and conditions associated with neuroinflammation, including Aicardi-Goutières Syndrome, stroke, neuropsychiatric systemic lupus erythematosus, neuroinflammation following traumatic brain injury, neuro-autoimmune disorders, Alzheimer's disease, post-operative delirium and cognitive decline, cranial radiation-induced cognitive decline, viral infection-induced cognitive decline, neuromyelitis optica, and ataxia telangiectasia.
Fredrik Carl Kamme
Filed: 17 Jun 22
Utility
Modulation of Huntingtin Expression
26 Jan 23
Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal.
Gene Hung, C. Frank Bennet, Susan M. Freier, Holly Kordasiewicz, Lisa Stanek, Don W. Cleveland, Seng H. Cheng, Lamya Shihabuddin
Filed: 18 Jul 22