Patents Assigned to Ionis Pharmaceuticals, Inc.
  • METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
    Publication number: 20230212564
    Abstract: Disclosed herein are methods for decreasing AlAT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of fibrosis. Disclosed herein are methods for decreasing A1AT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of liver disease, such as, A1ATD associated liver disease, and pulmonary disease, such as, A1ATD associated pulmonary disease in an individual in need thereof. Methods for inhibiting AlAT mRNA and protein expression can also be used as a prophylactic treatment to prevent individuals at risk for developing a liver disease, such as, A1ATD associated liver disease and pulmonary disease, such as, A1ATD associated pulmonary disease.
    Type: Application
    Filed: May 9, 2022
    Publication date: July 6, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Brett P. Monia, Michael L. McCaleb, Susan M. Freier, Shuling Guo
  • MODULATORS OF FOXP3 EXPRESSION
    Publication number: 20230201243
    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: Application
    Filed: November 29, 2022
    Publication date: June 29, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Alexey M. REVENKO, Robert A. MACLEOD, Susan M. FREIER
  • GENE THERAPY AND TARGETED DELIVERY OF CONJUGATED COMPOUNDS
    Publication number: 20230201374
    Abstract: Provided herein are methods, compounds, and compositions useful for targeted delivery of compounds to non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions are useful, for example, in gene therapy mediated ectopic expression of cell surface receptors and targeted delivery of compounds, such as conjugated oligonucleotides, to the non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions can be useful, for example, to treat, prevent, delay or ameliorate disease in an individual by targeted reduction of a gene of interest in the non-native cell ectopically expressing cell surface receptors.
    Type: Application
    Filed: July 13, 2022
    Publication date: June 29, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Frank Rigo
  • Modulation of Dystrophia Myotonica-Protein Kinase (DMPK) Expression
    Publication number: 20230174987
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
    Type: Application
    Filed: July 21, 2022
    Publication date: June 8, 2023
    Applicants: Ionis Pharmaceuticals, Inc., University of Rochester
    Inventors: C. Frank Bennett, Susan M. Freier, Robert A. MacLeod, Sanjay K. Pandey, Charles A. Thornton, Thurman Wheeler, Seng H. Cheng, Andrew Leger, Bruce M. Wentworth
  • COMPOUNDS AND METHODS FOR REDUCING PSD3 EXPRESSION
    Publication number: 20230167446
    Abstract: Provided are compositions of matter including oligomeric agents, modified oligonucleotides, oligomeric compounds, and pharmaceutical compositions, and methods of use thereof, for reducing the amount or activity of PSD3 RNA in a cell or animal, and in certain instances reducing the amount of PSD3 protein in a cell or animal. Such compositions are useful to treat liver disease, fatty liver disease (FLD), nonalcoholic fatty liver disease (NAFLD), hepatic steatosis, non-alcoholic steatohepatitis (NASH), liver cirrhosis, hepatocellular carcinoma, alcoholic liver disease, alcoholic steatohepatitis (ASH), HCV hepatitis, chronic hepatitis, hereditary hemochromatosis, or primary sclerosing cholangitis.
    Type: Application
    Filed: October 31, 2022
    Publication date: June 1, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Huynh-Hoa BUI, Susan M. FREIER, Richard LEE
  • Methods for modulating FMR1 expression
    Patent number: 11661601
    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
  • MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
    Publication number: 20230159922
    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
  • MODIFIED CRISPR RNA AND MODIFIED SINGLE CRISPR RNA AND USES THEREOF
    Publication number: 20230159919
    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
  • COMPOSITIONS AND METHODS OF INHIBITING SEVERE ACUTE RESPIRATORY SYNDROME CORONAVIRUS 2 (SARS-CoV-2)
    Publication number: 20230151366
    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
  • COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION
    Publication number: 20230151365
    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
  • COMPOSITIONS AND METHODS FOR TREATING AND PREVENTING PREKALLIKREIN-ASSOCIATED CONDITIONS
    Publication number: 20230149438
    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
  • MODULATORS OF GROWTH HORMONE RECEPTOR
    Publication number: 20230147811
    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
  • Methods and compositions for modulating apolipoprotein (a) expression
    Patent number: 11634711
    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
  • Modulators of PCSK9 expression
    Patent number: 11633483
    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
  • COMPOUNDS AND METHODS FOR MODULATING KCNQ2
    Publication number: 20230124616
    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.
    Type: Application
    Filed: March 5, 2021
    Publication date: April 20, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Paymaan Jafar-nejad, Huynh-Hoa Bui
  • Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
    Publication number: 20230121967
    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 6, 2022
    Publication date: April 20, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Eric E. Swayze
  • COMPOUNDS AND METHODS FOR MODULATING SPLICING OF PRE-MRNA
    Publication number: 20230117089
    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.
    Type: Application
    Filed: February 26, 2021
    Publication date: April 20, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Frank Rigo, Paymaan Jafar-Nejad
  • 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
  • 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