358 patents
Page 4 of 18
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
Oligomeric compounds comprising bicyclic nucleotides and uses thereof
31 Jan 23
The present invention provides oligomeric compounds.
Susan M. Freier, Eric E. Swayze
Filed: 21 Jan 20
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
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
Compounds and Methods for Modulating C90RF72
19 Jan 23
Disclosed herein are compounds and methods for modulating C9orf72 transcript.
Thazha P. PRAKASH, Frank RIGO, David COREY
Filed: 4 Feb 22
Utility
Modulators of FOXP3 expression
10 Jan 23
The present embodiments provide methods, compounds, and compositions useful for inhibiting FOXP3 expression, which may be useful for treating, preventing, or ameliorating cancer.
Alexey Revenko, Robert A. MacLeod, Susan M. Freier
Filed: 14 Nov 19
Utility
Selective Antisense Compounds and Uses Thereof
5 Jan 23
The present invention provides oligomeric compounds.
Michael Oestergaard, Punit P. Seth, Eric E. Swayze
Filed: 27 Jan 22
Utility
Compounds and Methods for Modulating Factor XII
5 Jan 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of FXII RNA in a cell or subject, and in certain instances reducing the amount of FXII protein in a cell or subject.
Susan M. Freier, Huynh-Hoa Bui, Chenguang Zhao, Brett P. Monia, Jeffrey R. Crosby
Filed: 17 Dec 21
Utility
Compounds and methods for modulating ATXN1
3 Jan 23
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 Modulating UBE3A-ATS
29 Dec 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in a cell or subject.
Susan M. Freier, Huynh-Hoa Bui, Paymaan Jafar-nejad, Frank Rigo
Filed: 14 Jan 22
Utility
Modulation of transthyretin expression
27 Dec 22
Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal.
Brett P. Monia, Susan M. Freier, Andrew M. Siwkowski, Shuling Guo
Filed: 1 Jun 20
Utility
Selective Reduction of Allelic Variants
22 Dec 22
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP).
C. Frank Bennett, Susan M. Freier, Sarah Greenlee, Eric E. Swayze
Filed: 18 Jan 22
Utility
Modulation of Angiopoietin-Like 3 Expression
22 Dec 22
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: 11 Aug 21
Utility
Methods for treating hypercholesterolemia
20 Dec 22
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
Utility
Methods for reducing LRRK2 expression
20 Dec 22
Provided herein are methods for decreasing LRRK2 mRNA expression.
Hien Thuy Zhao, Holly Kordasiewicz
Filed: 17 Dec 20
Utility
Modulators of APOL1 expression
13 Dec 22
The present embodiments provide methods, compounds, and compositions useful for inhibiting APOL1 expression, which may be useful for treating, preventing, or ameliorating a disease associated with APOL1.
Susan M. Freier
Filed: 18 Sep 20
Utility
Compounds and Methods for Reducing App Expression
1 Dec 22
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 Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease.
Susan M. Freier, Huynh-Hoa Bui, Holly Kordasiewicz, Eric E. Swayze
Filed: 29 Jan 20
Utility
Antisense Compounds and Uses Thereof
24 Nov 22
The present invention provides compounds comprising oligonucleotides complementary to a pyruvate kinase M transcript.
Frank Rigo, C. Frank Bennett, Adrian R. Krainer, Zhenxun Wang
Filed: 16 Dec 21
Utility
Antisense compounds and uses thereof
8 Nov 22
The present disclosure provides oligomeric compounds.
Holly Kordasiewicz, Curt Mazur, Punit P. Seth, Eric E. Swayze
Filed: 22 Oct 18