Abstract: 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. Such Fragile X-Spectrum disorders include FXS, FXTAS, and FXPOI.
Type:
Grant
Filed:
March 22, 2019
Date of Patent:
May 30, 2023
Assignees:
IONIS PHARMACEUTICALS, INC., THE REGENTS OF THE UNIVERSITY OF MICHIGAN
Inventors:
Frank Rigo, Peter Todd, Caitlin Rodriguez
Abstract: The present disclosure provides oligomeric compounds comprising a modified oligonucleotide having at least one stereo-non-standard nucleoside. An oligomeric compound comprising a modified oligonucleotide consisting of 12-30 linked nucleosides, wherein at least one nucleoside of the modified oligonucleotide is a stereo-non-standard nucleoside; and wherein the oligomeric compound is selected from among an RNAi compound, a modified CRISPR compound, and an artificial mRNA compound.
Type:
Application
Filed:
August 14, 2020
Publication date:
May 25, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Punit P. Seth, Michael T. Migawa, Graeme C. Freestone
Abstract: The present disclosure provides compounds comprising modified oligonucleotides for use in CRISPR. In certain embodiments, such modified oligonucleotides provide improved properties of crRNA. In certain embodiments, such modified oligonucleotides provide improved properties of scrRNA.
Type:
Application
Filed:
June 27, 2022
Publication date:
May 25, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Meghdad Rahdar, Thazha P. Prakash, Eric E. Swayze, C. Frank Bennett
Abstract: 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.
Type:
Application
Filed:
April 16, 2021
Publication date:
May 18, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Susan M. Freier, Eric E. Swayze, Robert J. Prill
Abstract: Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
Type:
Application
Filed:
October 21, 2022
Publication date:
May 18, 2023
Applicant:
IONIS PHARMACEUTICALS, INC.
Inventors:
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
Abstract: 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. In certain instances, methods are useful for ameliorating at least one symptom of hereditary angioedema. Such symptoms of hereditary angioedema include, but are not limited to, nausea, vomiting, itching, headache, fatigue, abdominal pain, shortness of breath, rhinitis, anaphylaxis, bronchoconstriction, and swelling. In certain instances, methods are useful for ameliorating at least one symptom of macular edema. Such symptoms of macular edema include, but are not limited to, impaired vision and vision loss.
Type:
Application
Filed:
March 12, 2021
Publication date:
May 18, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Eugene Schneider, Nicholas J. Viney, Veronica J. Alexandra, Laura Bordone, Kenneth Newman
Abstract: 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).
Type:
Application
Filed:
May 19, 2022
Publication date:
May 11, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Sanjay Bhanot, Susan M. Freier, Huynh-Hoa Bui
Abstract: Disclosed herein are antisense compounds and methods for decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) or Lp(a). Certain diseases, disorders or conditions related to apo(a) or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The antisense compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
Type:
Grant
Filed:
August 3, 2020
Date of Patent:
April 25, 2023
Assignee:
Ionis Pharmaceuticals, Inc.
Inventors:
Rosanne M. Crooke, Mark J. Graham, Susan M. Freier, Marc Lim, Andrew Dibble
Abstract: 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.
Type:
Grant
Filed:
November 20, 2020
Date of Patent:
April 25, 2023
Assignee:
IONIS PHARMACEUTICALS, INC.
Inventors:
Eric E. Swayze, Susan M. Freier, Huynh-Hoa Bui
Abstract: 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. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of an epileptic encephalopathy. Such symptoms and hallmarks include infantile spasms or seizures, EEC abnormalities, brain MRI abnormalities in the infant, and an associated developmental impairment. Such epileptic encephalopathies include those associated with gain-of-function and dominant negative mutations in KCNQ2.
Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.
Abstract: Provided are compounds, methods, and pharmaceutical compositions for modulating splicing of a pre-mRNA in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a disease or disorder.
Abstract: 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).
Type:
Application
Filed:
March 7, 2022
Publication date:
April 20, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Sanjay Bhanot, Susan M. Freier, Huynh-Hoa Bui
Abstract: 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.
Type:
Grant
Filed:
February 15, 2022
Date of Patent:
April 18, 2023
Assignee:
Ionis Pharmaceuticals, Inc.
Inventors:
Brooke A. Anderson, Xue-hai Liang, William John Drury, III, Michael Oestergaard, Michael T. Migawa, Punit P. Seth
Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.
Type:
Application
Filed:
April 4, 2022
Publication date:
April 13, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Punit P. Seth, Michael Oestergaard, Eric E. Swayze
Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C9ORF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon 1B of a C9ORF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds.
Type:
Application
Filed:
April 15, 2022
Publication date:
April 13, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Susan M. Freier, Frank Rigo, Priyam Singh
Abstract: 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. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.
Type:
Application
Filed:
April 4, 2022
Publication date:
April 13, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Tracy A. Cole, Holly Kordasiewicz, Huynh-Hoa Bui, Susan M. Freier
Abstract: 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. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.
Abstract: 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. In certain instances, methods are useful for ameliorating at least one symptom of Huntington's disease. Such symptoms of Huntington's disease include, but are not limited to, brain atrophy, muscle atrophy, nerve degeneration, uncontrolled movement, difficulty swallowing, difficulty speaking, anxiety and depression.
Type:
Application
Filed:
February 19, 2021
Publication date:
April 6, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
C. Frank Bennett, Daniel A. Norris, Anne V. Smith, Roger Lane
Abstract: The present disclosure provides a trinucleotide comprising the formula below or an oligomeric compound comprising the formula below:
Type:
Application
Filed:
August 25, 2022
Publication date:
March 30, 2023
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Juan Carlos Salinas Hernandez, Stephen Hanessian, Eric E. Swayze, Punit P. Seth, Jenny Lorena Rico Duque, Graeme C. Freestone, Barbara Lugato, Rajasekaran Tamiselvan