Patents Assigned to Ionis Pharmaceuticals, Inc.
  • Compounds and methods for reducing ATXN3 expression
    Patent number: 11434488
    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: Grant
    Filed: May 9, 2019
    Date of Patent: September 6, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • MODULATORS OF HSD17B13 EXPRESSION
    Publication number: 20220273691
    Abstract: Methods, compounds, and compositions useful for inhibiting HSD expression are provided. Such compounds, compositions, and methods are useful for treating, preventing, or ameliorating a disease associated with HSD 17B 13.
    Type: Application
    Filed: December 20, 2019
    Publication date: September 1, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Susan F. MURRAY
  • COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION
    Publication number: 20220275365
    Abstract: Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
    Type: Application
    Filed: February 13, 2020
    Publication date: September 1, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
  • Modulation of Huntington expression
    Patent number: 11421231
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
    Type: Grant
    Filed: October 12, 2020
    Date of Patent: August 23, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Gene Hung, C. Frank Bennett, Susan M. Freier, Holly Kordasiewicz, Lisa Stanek, Don W. Cleveland, Seng H. Cheng, Lamya Shihabuddin
  • COMPOUNDS AND METHODS FOR MODULATING TMPRSS6 EXPRESSION
    Publication number: 20220259600
    Abstract: Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as polycythemia, hemochromatosis or ?-thalassemia can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to TMPRSS6.
    Type: Application
    Filed: September 24, 2021
    Publication date: August 18, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Shuling Guo, Mariam Aghajan, Eric E. Swayze
  • Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
    Patent number: 11414657
    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: Grant
    Filed: June 29, 2016
    Date of Patent: August 16, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Meghdad Rahdar, Thazha P. Prakash, Eric E. Swayze, C. Frank Bennett
  • SYNTHESIS OF OLIGOMERIC COMPOUNDS COMPRISING PHOSPHOROTHIOATE DIESTER AND PHOSPHODIESTER LINKAGES
    Publication number: 20220251128
    Abstract: The present disclosure provides methods for synthesizing oligomeric compounds having at least one phosphorothioate diester linkage and at least one phosphate diester internucleoside linkage. In certain embodiments, the present disclosure provides oxidation reagents that produce low amounts of unwanted phosphate diester impurities in oligomeric compounds having at least one phosphorothioate diester linkage and at least one phosphate diester internucleoside linkage.
    Type: Application
    Filed: May 15, 2020
    Publication date: August 11, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Andrew K. McPherson, Andrew A. Rodriguez, Daniel C. Capaldi
  • METHODS AND COMPOSITIONS FOR INHIBITING PMP22 EXPRESSION
    Publication number: 20220251553
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting PMP22 expression and for treating, preventing, or ameliorating a disease associated with PMP22.
    Type: Application
    Filed: October 4, 2021
    Publication date: August 11, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Gene Hung, Holly Kordasiewicz, Hien Thuy Zhao, Eric E. Swayze
  • ANGIOTENSIN II TYPE 1 RECEPTOR TARGETED OLIGONUCLEOTIDES AND USES THEREOF
    Publication number: 20220243210
    Abstract: The present disclosure provides compounds and methods for targeting cells expressing AGTR1. In some instances, the compound includes an oligonucleotide and an AGTR1 binding conjugate moiety, and optionally a conjugate linker.
    Type: Application
    Filed: May 18, 2020
    Publication date: August 4, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Adam Mullick, Yichun Kuo, Ting Yuan Yeh, Thazha P. Prakash
  • MODULATORS OF MALAT1 EXPRESSION
    Publication number: 20220243205
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting MALAT1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with MALAT1.
    Type: Application
    Filed: February 8, 2022
    Publication date: August 4, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. FREIER, Youngsoo KIM, Robert MACLEOD
  • COMPOSITIONS AND METHODS FOR MODULATING TAU EXPRESSION
    Publication number: 20220243200
    Abstract: Disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Also disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Such compounds and methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration Tau antisense oligonucleotides include Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy, Chronic Traumatic Encephalopathy, Epilepsy, and Dravet's Syndrome.
    Type: Application
    Filed: September 22, 2021
    Publication date: August 4, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Frank Rigo
  • MODULATORS OF COMPLEMENT FACTOR B
    Publication number: 20220243209
    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: September 2, 2021
    Publication date: August 4, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Tamar R. Grossman, Michael L. McCaleb, Andrew T. Watt, Susan M. Freier
  • COMPOUNDS AND METHODS FOR REDUCING FUS EXPRESSION
    Publication number: 20220243203
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of FUS RNA in a cell or subject, and in certain instances reducing the amount of FUS protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative condition. Such symptoms and hallmarks include muscle weakness and fatigue, protein aggregates in the central nervous system, and speech difficulties and behavioral abnormalities. Non-limiting examples of neurodegenerative conditions that benefit from these compounds, methods, and pharmaceutical compositions are amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD).
    Type: Application
    Filed: May 28, 2020
    Publication date: August 4, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Huynh-Hoa Bui
  • Method of conjugating oligomeric compounds
    Patent number: 11400161
    Abstract: Provided herein are solid phase methods for the synthesis of conjugated oligomeric compounds and intermediates used in such methods. In particular, the solid phase methods provide for addition of a phosphoramidite functionalized conjugate group to a solid support bound oligomeric compound. The methods also provide an increase in overall yield and a cost benefit over existing methods.
    Type: Grant
    Filed: October 6, 2017
    Date of Patent: August 2, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Isaiah E. Cedillo, Recaldo Carty
  • Compositions and Methods for Modulating MECP2 Expression
    Publication number: 20220226361
    Abstract: Disclosed herein are compounds and methods for decreasing MECP2 mRNA and protein expression. Such compounds and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders and syndromes. Such MECP2 associated disorders include MECP2 duplication syndrome.
    Type: Application
    Filed: August 23, 2021
    Publication date: July 21, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • MODULATION OF APOLIPOPROTEIN C-III (APOCIII) EXPRESSION IN LIPOPROTEIN LIPASE DEFICIENT (LPLD) POPULATIONS
    Publication number: 20220213472
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of ApoCIII mRNA and protein in a patient with Fredrickson Type I dyslipidemia, FCS, LPLD. Also provided herein are methods, compounds, and compositions for treating, preventing, delaying, or ameliorating Fredrickson Type I dyslipidemia, FCS, LPLD, in a patient. Further provided herein are methods, compounds, and compositions for increasing HDL levels and/or improving the ratio of TG to HDL and reducing plasma lipids and plasma glucose in a patient with Fredrickson Type I dyslipidemia, FCS, LPLD. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of pancreatitis, cardiovascular disease or metabolic disorder, or a symptom thereof.
    Type: Application
    Filed: July 9, 2021
    Publication date: July 7, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Veronica J. Alexander, Nicholas J. Viney, Joseph L. Witztum
  • Modulation of factor 11 expression
    Patent number: 11376273
    Abstract: Disclosed herein are antisense compounds and methods for decreasing Factor 11 and treating or preventing thromboembolic complications in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to Factor 11 include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, and stroke. Antisense compounds targeting Factor 11 can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.
    Type: Grant
    Filed: July 29, 2020
    Date of Patent: July 5, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Chenguang Zhao, Brett P. Monia, Hong Zhang, Jeffrey R. Crosby, Andrew M. Siwkowski
  • COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
    Publication number: 20220195431
    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: February 21, 2020
    Publication date: June 23, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Hien Thuy Zhao, Holly Kordasiewicz, Ruben E. Valas
  • Compositions for Modulating Ataxin 2 Expression
    Publication number: 20220195430
    Abstract: Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotropic sclerosis (ALS), and parkinsonism.
    Type: Application
    Filed: August 4, 2021
    Publication date: June 23, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • Modulators of EZH2 expression
    Patent number: 11365416
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting EZH2 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with EZH2.
    Type: Grant
    Filed: April 11, 2019
    Date of Patent: June 21, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Minji Jo, Youngsoo Kim, Robert MacLeod, Susan M. Freier