358 patents
Page 2 of 18
Utility
Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
7 Sep 23
The present invention provides oligomeric compounds.
Susan M. Freier, Eric E. Swayze
Filed: 16 Dec 22
Utility
Allele specific modulators of P23H rhodopsin
5 Sep 23
The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject.
Susan F. Murray, Punit P. Seth, Michael L. McCaleb, Susan M. Freier, Priyam Singh
Filed: 21 May 21
Utility
Compounds and methods for the modulation of amyloid-β precursor protein
22 Aug 23
Certain embodiments disclosed herein are directed to compounds and methods for modulating APP expression.
Frank Rigo, Michelle L. Hastings
Filed: 1 Mar 19
Utility
Selective antisense compounds and uses thereof
22 Aug 23
The present invention provides oligomeric compounds.
Punit P. Seth, Michael Oestergaard, Eric E. Swayze
Filed: 3 Sep 20
Utility
Compounds and methods for modulating PLP1
22 Aug 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PLP1 RNA in a cell or subject, and in certain instances reducing the amount of proteolipid protein 1 in a cell or subject.
Berit Elissa Powers
Filed: 29 Jun 21
Utility
Compositions and methods for modulating complement factor B expression
22 Aug 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
Conjugated antisense compounds and their use
15 Aug 23
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: 14 Dec 18
Utility
Modulation of GYS1 expression
1 Aug 23
Provided herein are methods, compounds, and compositions for reducing expression of GYS1 in an individual.
Tamar R. Grossman, Susan M. Freier, Berge Minassian, Saija Ahonen
Filed: 23 Dec 21
Utility
Compounds and Methods for Reducing ATXN3 Expression
27 Jul 23
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: 29 Jun 22
Utility
Compounds and Methods for Reducing MSH3 Expression
27 Jul 23
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of MSH3 RNA in a cell or subject, and in certain instances reducing the amount of MSH3 protein in a cell or subject.
Kenneth W. Dobie, Huynh-Hoa Bui, Susan M. Freier
Filed: 10 Jun 21
Utility
Methods and Compositions for Modulating ALPHA-1-ANTITRYPSIN Expression
6 Jul 23
Disclosed herein are methods for decreasing AlAT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of fibrosis.
Brett P. Monia, Michael L. McCaleb, Susan M. Freier, Shuling Guo
Filed: 9 May 22
Utility
Modulators of FOXP3 Expression
29 Jun 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 M. REVENKO, Robert A. MACLEOD, Susan M. FREIER
Filed: 29 Nov 22
Utility
Gene Therapy and Targeted Delivery of Conjugated Compounds
29 Jun 23
Provided herein are methods, compounds, and compositions useful for targeted delivery of compounds to non-native cells ectopically expressing cell surface receptors.
Frank Rigo
Filed: 13 Jul 22
Utility
Methods for modulating FMR1 expression
30 May 23
Provided are methods for increasing the amount or activity of FMR1 RNA, and in certain instances of increasing the amount of FMRP protein, in an animal Such methods are useful to prevent or ameliorate at least one symptom of a Fragile X-Spectrum disorder.
Frank Rigo, Peter Todd, Caitlin Rodriguez
Filed: 22 Mar 19
Utility
Modified Crispr Rna and Modified Single Crispr Rna and Uses Thereof
25 May 23
The present disclosure provides compounds comprising modified oligonucleotides for use in CRISPR.
Meghdad Rahdar, Thazha P. Prakash, Eric E. Swayze, C. Frank Bennett
Filed: 27 Jun 22
Utility
Modified Oligomeric Compounds and Uses Thereof
25 May 23
The present disclosure provides oligomeric compounds comprising a modified oligonucleotide having at least one stereo-non-standard nucleoside.
Punit P. Seth, Michael T. Migawa, Graeme C. Freestone
Filed: 14 Aug 20
Utility
Compositions and Methods for Treating and Preventing Prekallikrein-associated Conditions
18 May 23
Provided herein are methods of administering ISIS 721744 for ameliorating edema, and methods of reducing prekallikrein (PKK) RNA, protein or activity in a human subject in need thereof.
Eugene Schneider, Nicholas J. Viney, Veronica J. Alexandra, Laura Bordone, Kenneth Newman
Filed: 12 Mar 21
Utility
Compositions and Methods for Modulating TTR Expression
18 May 23
Provided herein are oligomeric compounds with conjugate groups.
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
Filed: 21 Oct 22
Utility
COMPOSITIONS AND METHODS OF INHIBITING SEVERE ACUTE RESPIRATORY SYNDROME CORONAVIRUS 2 (SARS-CoV-2)
18 May 23
The present embodiments provide methods, compounds, and compositions useful for inhibiting or reducing SARS-CoV-2 replication, infectivity, viral titer, or viral load, which can be useful for preventing or treating COVID-19 in an individual.
Susan M. Freier, Eric E. Swayze, Robert J. Prill
Filed: 16 Apr 21
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