Patents Assigned to Ionis Pharmaceuticals, Inc.
  • COMPOSITIONS AND METHODS FOR MODULATING GROWTH HORMONE RECEPTOR EXPRESSION
    Publication number: 20230118177
    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
  • Linkage modified oligomeric compounds and uses thereof
    Patent number: 11629348
    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
  • COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
    Publication number: 20230114429
    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.
    Type: Application
    Filed: August 31, 2022
    Publication date: April 13, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Frank Rigo, Chrissa A, Dwyer
  • Compounds and Methods for Reducing LRRK2 Expression
    Publication number: 20230114930
    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
  • Selective Antisense Compounds and Uses Thereof
    Publication number: 20230113863
    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
  • Compositions for Modulating C9ORF72 Expression
    Publication number: 20230112920
    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
  • METHODS FOR REDUCING HTT EXPRESSION
    Publication number: 20230108340
    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
  • OLIGOMERIC COMPOUNDS COMPRISING BACKBONE CONSTRAINED MACROCYCLES
    Publication number: 20230095696
    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
  • Compositions and methods for modulating PKK expression
    Patent number: 11613752
    Abstract: Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.
    Type: Grant
    Filed: May 11, 2020
    Date of Patent: March 28, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Eric E. Swayze
  • Modified Compounds and Uses Thereof
    Publication number: 20230068063
    Abstract: The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.
    Type: Application
    Filed: March 17, 2022
    Publication date: March 2, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Punit P. Seth, Michael Oestergaard, Michael T. Migawa, Xue-hai Liang, Wen Shen, Stanley T. Crooke, Eric E. Swayze
  • COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION
    Publication number: 20230057718
    Abstract: 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.
    Type: Application
    Filed: May 7, 2021
    Publication date: February 23, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Tamar R. Grossman, Michael L. McCaleb, Andrew T. Watt, Susan M. Freier
  • COMPOUNDS AND METHODS FOR REDUCING APP EXPRESSION
    Publication number: 20230055405
    Abstract: 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 or disorder. Such symptoms and hallmarks include cognitive impairment, including a decline in memory and language skills, behavioral and psychological symptoms such as apathy and lack of motivation, gait disturbances and seizures, progressive dementia, and abnormal amyloid deposits.
    Type: Application
    Filed: July 28, 2021
    Publication date: February 23, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Huynh-Hoa Bui, Susan M. Freier, Hien Thuy Zhao, Holly Kordasiewicz, Ruben E. Valas, Thazha P. Prakash, W. Brad Wan
  • Compounds and methods for reducing ATXN3 expression
    Patent number: 11583548
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 mRNA in a cell or animal, and in certain instances reducing the amount of Ataxin-3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to prevent or 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 SCA3.
    Type: Grant
    Filed: November 10, 2017
    Date of Patent: February 21, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • MODULATORS OF DIACYGLYCEROL ACYLTRANSFERASE 2 (DGAT2)
    Publication number: 20230047452
    Abstract: 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.
    Type: Application
    Filed: February 22, 2022
    Publication date: February 16, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Sanjay Bhanot, Susan M. Freier, Eric E. Swayze
  • COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
    Publication number: 20230043648
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for modulating SCN1A RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a developmental or epileptic encephalopathic disease, such as Dravet Syndrome. Such symptoms include seizures, sudden unexpected death in epilepsy, status epilepticus, behavioral and developmental delays, movement and balance dysfunctions, orthopedic conditions, motor and cognitive dysfunctions, delayed language and speech issues, visual motor integration dysfunctions, visual perception dysfunctions, executive dysfunctions, growth and nutrition issues, sleeping difficulties, chronic infections, sensory integration disorders, and dysautonomia.
    Type: Application
    Filed: February 26, 2021
    Publication date: February 9, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Paymaan Jafar-Nejad, Susan M. Freier, Frank Rigo
  • Oligomeric compounds comprising bicyclic nucleotides and uses thereof
    Patent number: 11566245
    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: Grant
    Filed: January 21, 2020
    Date of Patent: January 31, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Eric E. Swayze
  • COMPOUNDS AND METHODS FOR REDUCING IFNAR1 EXPRESSION
    Publication number: 20230025910
    Abstract: 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.
    Type: Application
    Filed: June 17, 2022
    Publication date: January 26, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Fredrik Carl Kamme
  • COMPOUNDS AND METHODS FOR MODULATING C90RF72
    Publication number: 20230020192
    Abstract: Disclosed herein are compounds and methods for modulating C9orf72 transcript. Such compounds and methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof.
    Type: Application
    Filed: February 4, 2022
    Publication date: January 19, 2023
    Applicants: IONIS PHARMACEUTICALS, INC., BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
    Inventors: Thazha P. PRAKASH, Frank RIGO, David COREY
  • Modulators of FOXP3 expression
    Patent number: 11547718
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting FOXP3 expression, which may be useful for treating, preventing, or ameliorating cancer.
    Type: Grant
    Filed: November 14, 2019
    Date of Patent: January 10, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Alexey Revenko, Robert A. MacLeod, Susan M. Freier
  • SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
    Publication number: 20230002763
    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. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.
    Type: Application
    Filed: January 27, 2022
    Publication date: January 5, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Michael Oestergaard, Punit P. Seth, Eric E. Swayze