Patents Assigned to Ionis Pharmaceuticals, Inc.
  • 5? modified nucleosides and oligomeric compounds prepared therefrom
    Patent number: 11268094
    Abstract: The present invention provides 5? modified nucleosides and oligomeric compounds prepared therefrom. More particularly, the present invention provides modified nucleosides having at least one 5?-substituent and an optional 2? substituent, oligomeric compounds comprising at least one of these modified nucleosides and methods of using the oligomeric compounds. In some embodiments, the oligomeric compounds provided herein are expected to hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.
    Type: Grant
    Filed: April 30, 2020
    Date of Patent: March 8, 2022
    Assignee: Ionis Pharmaceuticals, Inc
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
  • COMPOUNDS AND METHODS FOR MODULATING SMN2
    Publication number: 20220064638
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 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 neurodegenerative disorder. Such symptoms include reduced muscle strength; inability or reduced ability to sit upright, to stand, and/or walk; reduced neuromuscular activity; reduced electrical activity in one or more muscles; reduced respiration; inability or reduced ability to eat, drink, and/or breathe without assistance; loss of weight or reduced weight gain; and/or decreased survival.
    Type: Application
    Filed: February 26, 2021
    Publication date: March 3, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Frank Rigo, Thazha P. Prakash, Kar Yun Karen Ling, W. Brad Wan, William John Drury, III
  • COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
    Publication number: 20220064637
    Abstract: 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. 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 motor dysfunction, aggregation formation, and neuron death. Such neurodegenerative diseases include spinocerebellar ataxia type 3 (SCA3).
    Type: Application
    Filed: May 9, 2019
    Publication date: March 3, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • Compounds and Methods for Reducing ATXN2 Expression
    Publication number: 20220064639
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 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 spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.
    Type: Application
    Filed: April 23, 2021
    Publication date: March 3, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Priyam Singh, Frank Rigo, Paymaan Jafar-nejad, Holly Kordasiewicz
  • MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
    Publication number: 20220064636
    Abstract: The present disclosure provides oligomeric compounds comprising a modified oligonucleotide having at least one stereo-non-standard nucleoside.
    Type: Application
    Filed: October 4, 2019
    Publication date: March 3, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Punit P. Seth, Michael T. Migawa, Graeme C. Freestone
  • Compounds and methods for modulating C90RF72
    Patent number: 11260073
    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: Grant
    Filed: November 2, 2016
    Date of Patent: March 1, 2022
    Assignees: IONIS PHARMACEUTICALS, INC., BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
    Inventors: Thazha P. Prakash, Frank Rigo, David Corey
  • Compounds and methods for modulating UBE3A-ATS
    Patent number: 11261446
    Abstract: 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. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delays, ataxia, speech impairment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman Syndrome.
    Type: Grant
    Filed: April 21, 2021
    Date of Patent: March 1, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Paymaan Jafar-nejad
  • COMPOUNDS AND METHODS FOR MODULATING PLP1
    Publication number: 20220056445
    Abstract: 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. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include hypotonia, nystagmus, optic atrophy, respiratory distress, motor delays, cognitive dysfunction, speech dysfunction, spasticity, ataxia, seizures, choreiform movements, and death. Such leukodystrophies include Pelizaeus-Merzbacher disease.
    Type: Application
    Filed: June 29, 2021
    Publication date: February 24, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Berit Elissa Powers
  • Modulators of IRF4 expression
    Patent number: 11241451
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting IRF4 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with IRF4.
    Type: Grant
    Filed: March 1, 2019
    Date of Patent: February 8, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Tianyuan Zhou, Youngsoo Kim, Robert MacLeod, Huynh-Hoa Bui, Susan M. Freier
  • COMPOSITIONS AND METHODS FOR MODULATION OF LMNA EXPRESSION
    Publication number: 20220031731
    Abstract: The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the LMNA gene. Such compounds are useful for modulating the expression of LMNA in a cell or animal, and in certain instances reducing the amount of progerin mRNA and/or progerin protein. Progerin mRNA results from aberrant splicing of LMNA and is translated to generate progerin protein. Accumulation of progerin protein causes Hutchinson-Gilford progeria syndrome (HOPS), a premature aging disease. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the LMNA gene results in a decrease in the amount of progerin mRNA and/or progerin protein. In certain embodiments, oligonucleotides are used to treat Hutchinson-Gilford Progeria Syndrome.
    Type: Application
    Filed: September 20, 2019
    Publication date: February 3, 2022
    Applicants: Ionis Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc.
    Inventors: Priyam Singh, Frank Rigo, Tom Misteli, Madaiah Puttaraju
  • Selective antisense compounds and uses thereof
    Patent number: 11236335
    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: Grant
    Filed: February 18, 2020
    Date of Patent: February 1, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Michael Oestergaard, Punit P. Seth, Eric E. Swayze
  • Antisense compounds and uses thereof
    Patent number: 11236330
    Abstract: The present invention provides compounds comprising oligonucleotides complementary to a pyruvate kinase M transcript. Certain such compounds are useful for hybridizing to a pyruvate kinase M transcript, including but not limited to a pyruvate kinase M transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the pyruvate kinase M transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with cancer.
    Type: Grant
    Filed: October 2, 2018
    Date of Patent: February 1, 2022
    Assignees: Ionis Pharmaceuticals, Inc., Cold Spring Harbor Laboratory
    Inventors: Frank Rigo, C. Frank Bennett, Adrian R. Krainer, Zhenxun Wang
  • Modulation of GYS1 expression
    Patent number: 11236339
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of GYS1 in an individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a glycogen storage disease or disorder in an individual in need.
    Type: Grant
    Filed: June 19, 2017
    Date of Patent: February 1, 2022
    Assignees: Ionis Pharmaceuticals, Inc., The Hospital for Sick Children
    Inventors: Tamar R. Grossman, Susan M. Freier, Berge Minassian, Saija Ahonen
  • COMPOUNDS AND METHODS FOR REDUCING PRION EXPRESSION
    Publication number: 20220025366
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PRNP RNA in a cell or animal, and in certain instances reducing the amount of PrP 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 spongiform changes in the brain, development of abnormal protein aggregates, neuronal loss, markers of neuronal loss, rapidly progressing dementia, and death. Such neurodegenerative diseases include prion diseases, Creutzfeldt-Jakob disease (CJD), variant Creutzfeldt-Jakob Disease (vCJD), familial Creutzfeldt-Jakob Disease (fCJD), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, kuru, Alzheimer's disease, or Parkinson's disease.
    Type: Application
    Filed: November 21, 2019
    Publication date: January 27, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Huynh-Hoa Bui, Hien Thuy Zhao
  • CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE
    Publication number: 20220023429
    Abstract: The present disclosure provides duplexes comprising a first oligomeric compound and a second oligomeric compound wherein the second oligomeric compound comprises a conjugate group. In certain embodiments, the duplex modulates the amount or activity of a target nucleic acid in extra hepatic tissues and/or extra hepatic cells. In certain embodiments, the duplex modulates the amount or activity of a target nucleic acid in hepatic tissues and/or hepatic cells.
    Type: Application
    Filed: August 10, 2021
    Publication date: January 27, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Punit P. Seth, Michael Oestergaard, Eric E. Swayze
  • OLIGOMER-CONJUGATE COMPLEXES AND THEIR USE
    Publication number: 20220025370
    Abstract: Oligonucleotides, chemically-modified oligonucleotides, and oligonucleotide-conjugate complexes for use in research, diagnostics, and/or therapeutics are described herein. In some embodiments, oligonucleotides comprising a stabilized phosphate moiety covalently attached to the 5?-terminal nucleoside are provided.
    Type: Application
    Filed: July 1, 2021
    Publication date: January 27, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Eric E. Swayze, Walter F. Lima, Garth A. Kinberger
  • Compounds and methods for reducing SNCA expression
    Patent number: 11230712
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein 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 motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia with Lewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.
    Type: Grant
    Filed: November 9, 2018
    Date of Patent: January 25, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Holly Kordasiewicz, Tracy A. Cole
  • Modulation of angiopoietin-like 3 expression
    Patent number: 11225664
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing plasma lipids, plasma glucose and atherosclerotic plaques in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease or metabolic disease, or a symptom thereof.
    Type: Grant
    Filed: December 20, 2019
    Date of Patent: January 18, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Rosanne M. Crooke, Mark J. Graham, Richard Lee, Kenneth W. Dobie
  • COMPOUNDS AND METHODS FOR MODULATING ANGIOTENSINOGEN EXPRESSION
    Publication number: 20220000901
    Abstract: Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating AGT and modulating a RAAS pathway related disease, disorder and/or condition in an individual in need thereof. A RAAS pathway related disease, disorder and/or condition in an individual such as hypertension can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to AGT.
    Type: Application
    Filed: November 23, 2020
    Publication date: January 6, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Adam Mullick, Mark J. Graham, Punit P. Seth, Susan M. Freier
  • Modulators of YAP1 expression
    Patent number: 11214803
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting YAP1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with YAP1.
    Type: Grant
    Filed: January 31, 2020
    Date of Patent: January 4, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Youngsoo Kim, Xiaolin Luo, Robert MacLeod, Susan M. Freier, Huynh-Hoa Bui