358 patents
Utility
Compounds and methods for reducing LRRK2 expression
16 Jan 24
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, Susan M. Freier
Filed: 4 Apr 22
Utility
Linkage Modified Oligomeric Compounds and Uses Thereof
4 Jan 24
The present disclosure provides oligomeric compounds (including oligomeric compounds that are antisense agents or portions thereof) comprising a modified oligonucleotide having at least one modified internucleoside linking group.
Brooke A. Anderson, Xue-Hai Liang, William John Drury, III, Michael Oestergaard, Michael T. Migawa, Punit P. Seth
Filed: 25 Jan 23
Utility
Compounds for Modulating CHMP7
4 Jan 24
Provided are compounds and pharmaceutical compositions for reducing the amount or activity of Charged Multivesicular Body Protein 7 (CHMP7) RNA in a cell or subject, and in certain instances reducing the amount of CHMP7 protein in a cell or subject.
Huynh-Hoa Bui
Filed: 1 Oct 21
Utility
Antisense oligonucleotide compositions
2 Jan 24
The present disclosure provides compositions comprising an antisense oligonucleotide and one or more excipients that modulates viscosity, turbidity or both viscosity and turbidity.
Andrew Dibble, Lloyd Tillman, Bret Coldren, Marc Lim
Filed: 9 Jun 20
Utility
Modulation of prekallikrein (PKK) expression
12 Dec 23
Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression.
Susan M. Freier, Huynh-Hoa Bui
Filed: 2 Jul 21
Utility
Method of treating fatty liver disease
12 Dec 23
The present disclosure provides methods of treating or preventing fatty liver disease and/or lowering cholesterol and LDL cholesterol levels in a subject.
Daniel Linden, Richard Lee, Stefano Romeo, Huynh-Hoa Bui
Filed: 21 Sep 21
Utility
Compounds and methods for increasing STMN2 expression
5 Dec 23
Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal.
Huynh-Hoa Bui, Don W. Cleveland, Ze'ev Melamed
Filed: 14 Jun 19
Utility
Oligomeric compounds for reducing DMPK expression
5 Dec 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
Compositions for modulating Ataxin 2 expression
5 Dec 23
Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression.
Susan M. Freier, Gene Hung, C. Frank Bennett
Filed: 4 Aug 21
Utility
Compounds and Methods for Modulating PMP22
23 Nov 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PMP22 RNA in a cell or animal, and in certain instances reducing the amount of PMP22 protein in a cell or animal.
Eric E. Swayze, Holly Kordasiewicz, Punit P. Seth, Hien Thuy Zhao, Michael T. Migawa, Ruben E. Valas, Thazha P. Prakash
Filed: 18 Jun 21
Utility
Compounds and Methods for Reducing Apoe Expression
9 Nov 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of APOE RNA in a cell or animal, and in certain instances reducing the amount of APOE 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.
Tracy A. Cole, Swagatam Mukhopadhyay, Huynh-Hoa Bui, Priyam Singh, Holly Kordasiewicz, Susan M. Freier, Hien Thuy Zhao
Filed: 23 Sep 21
Utility
Compositions and Methods for Modulating Complement Factor B Expression
9 Nov 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: 19 May 23
Utility
Compounds and Methods for Reducing IFNAR1 Expression
9 Nov 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.
Fredrik Carl Kamme
Filed: 26 Jul 23
Utility
Conjugated Oligonucleotides and Uses Thereof
26 Oct 23
Disclosed herein are compounds that comprise an oligonucleotide, a conjugate linker, and a conjugate moiety capable of interacting with a cell surface moiety, wherein the oligonucleotide and the conjugate moiety are connected via the conjugate linker.
Thazha P. Prakash, Mehran Nikan, Michael Tanowitz, Chrissa A. Dwyer, Frank Rigo, Punit P. Seth
Filed: 18 May 21
Utility
Compounds and methods for modulating GFAP
17 Oct 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GFAP RNA in a cell or subject, and in certain instances reducing the amount of GFAP in a cell or subject.
Berit Elissa Powers, Frank Rigo
Filed: 24 Jul 20
Utility
Modulators of PCSK9 Expression
12 Oct 23
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: 20 Feb 23
Utility
Modulators of PNPLA3 expression
10 Oct 23
The present embodiments provide methods, compounds, and compositions useful for inhibiting PNPLA3 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PNPLA3.
Susan M. Freier, Huynh-Hoa Bui
Filed: 22 Jul 20
Utility
Modulators of HSD17B13 Expression
5 Oct 23
Methods, compounds, and compositions useful for inhibiting HSD17B13 expression are provided.
Susan M. Freier, Susan F. Murray
Filed: 14 Jun 23
Utility
Compositions and their uses directed to huntingtin
19 Sep 23
Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal.
Susan M. Freier
Filed: 21 Jun 21
Utility
Compounds and methods for reducing IFNAR1 expression
12 Sep 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