Patents Assigned to Ionis Pharmaceuticals, Inc.
  • COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
    Publication number: 20240093201
    Abstract: Provided herein are compositions and methods for non-parenteral delivery of conjugated oligomeric compounds. In certain embodiments, compositions and methods are provided for oral delivery of conjugated oligomeric compounds. In certain embodiments, the oligomeric compounds are conjugated to one or more N-acetylgalactosamines or N-acetylgalactosamine analogues.
    Type: Application
    Filed: December 2, 2022
    Publication date: March 21, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Stanley T. Crooke
  • PURIFICATION METHODS FOR OLIGOMERIC COMPOUNDS
    Publication number: 20240092822
    Abstract: The present disclosure provides a process of separating designated oligomeric compounds from sample solutions comprising at least one contaminant In certain embodiments, the designated oligomeric compounds comprise a modified oligonucleotide and a conjugate group comprising at least one carbohydrate cluster. In certain embodiments, the present disclosure provides HPLC conditions that increase the separation of a designated oligomeric compound from at least one contaminant compared to standard HPLC conditions.
    Type: Application
    Filed: March 2, 2022
    Publication date: March 21, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Andrew A. Rodriguez, Christopher Michael Gabriel
  • COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
    Publication number: 20240082291
    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: Application
    Filed: February 13, 2023
    Publication date: March 14, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Holly Kordasiewicz
  • Compounds and methods for reducing ATXN2 expression
    Patent number: 11926825
    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: Grant
    Filed: April 23, 2021
    Date of Patent: March 12, 2024
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Frank Rigo
  • Modulators of Complement Factor B
    Patent number: 11926830
    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: Grant
    Filed: September 2, 2021
    Date of Patent: March 12, 2024
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Tamar R. Grossman, Michael L McCaleb, Andrew T. Watt, Susan M. Freier
  • METHODS AND COMPOSITIONS FOR MODULATING APOLIPOPROTEIN (A) EXPRESSION
    Publication number: 20240067964
    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: Application
    Filed: February 28, 2023
    Publication date: February 29, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Rosanne M. Crooke, Mark J. Graham, Susan M. Freier, Marc Lim, Andrew Dibble
  • COMPOUNDS AND METHODS FOR MODULATING ATXN1
    Publication number: 20240067962
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN1 RNA in a cell or subject, and in certain instances reducing the amount of ATXN1 in a cell or subject. 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 gait and limb ataxia, cognitive impairments, difficulty with speaking and swallowing, atrophy of the cerebellum and brainstem in magnetic resonance imaging (MRI), neurochemical abnormalities in the cerebellum and brainstem detected via magnetic resonance spectroscopy (MRS), and death within 10-15 years of symptom onset. Such neurodegenerative diseases include Spinocerebellar ataxia type 1.
    Type: Application
    Filed: October 19, 2022
    Publication date: February 29, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • ANTISENSE COMPOUNDS
    Publication number: 20240058370
    Abstract: Provided herein are gapmer oligomeric compounds for reduction of target RNA in vivo comprising different nucleotide modifications within one or both wing regions. Also provided are methods of using such oligomeric compounds, including use in animals. In certain embodiments, such compound have desirable potency and toxicity characteristics.
    Type: Application
    Filed: January 5, 2023
    Publication date: February 22, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Eric E. Swayze
  • COMPOUNDS AND METHODS FOR MODULATING TMPRSS6 EXPRESSION
    Publication number: 20240026364
    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: July 25, 2023
    Publication date: January 25, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Shuling Guo, Mariam Aghajan, Eric E. Swayze
  • Compounds and methods for reducing LRRK2 expression
    Patent number: 11873495
    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: Grant
    Filed: April 4, 2022
    Date of Patent: January 16, 2024
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Tracy A. Cole, Susan M. Freier
  • COMPOUNDS FOR MODULATING CHMP7
    Publication number: 20240002852
    Abstract: Provided are compounds and pharmaceutical compositions for reducing the amount or activity of Charged Multivesicular Body Protein 7 (CHMP7) RNA in a cell or subject, and in certain instances reducing the amount of CHMP7 protein in a cell or subject. Such compounds and pharmaceutical compositions are useful to ameliorate diseases or conditions associated with aberrant activation of Endosomal Sorting Complexes Required for Transport-III proteins.
    Type: Application
    Filed: October 1, 2021
    Publication date: January 4, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Huynh-Hoa Bui
  • LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
    Publication number: 20240002851
    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: Application
    Filed: January 25, 2023
    Publication date: January 4, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Brooke A. Anderson, Xue-Hai Liang, William John Drury, III, Michael Oestergaard, Michael T. Migawa, Punit P. Seth
  • Antisense oligonucleotide compositions
    Patent number: 11859180
    Abstract: The present disclosure provides compositions comprising an antisense oligonucleotide and one or more excipients that modulates viscosity, turbidity or both viscosity and turbidity. In certain embodiments, compositions comprising an antisense oligonucleotide and one or more excipients having low viscosity are provided. In certain embodiments, compositions comprising an antisense oligonucleotide and one or more excipients having low turbidity are provided. In certain embodiments, pharmaceutical compositions comprising an antisense oligonucleotide and one or more excipients having low viscosity and turbidity are provided.
    Type: Grant
    Filed: June 9, 2020
    Date of Patent: January 2, 2024
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Andrew Dibble, Lloyd Tillman, Bret Coldren, Marc Lim
  • Compositions and methods for modulating apolipoprotein (a) expression
    Patent number: 11851655
    Abstract: Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)]. In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
    Type: Grant
    Filed: October 1, 2020
    Date of Patent: December 26, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Mark J. Graham
  • Method of treating fatty liver disease
    Patent number: 11840689
    Abstract: The present disclosure provides methods of treating or preventing fatty liver disease and/or lowering cholesterol and LDL cholesterol levels in a subject. The present disclosure further provides methods of lowering expression of Pleckstrin and Sec7 Domain Containing 3 (PSD3) in a subject.
    Type: Grant
    Filed: September 21, 2021
    Date of Patent: December 12, 2023
    Assignees: ASTRAZENECA AB, IONIS PHARMACEUTICALS, INC.
    Inventors: Daniel Linden, Richard Lee, Stefano Romeo, Huynh-Hoa Bui
  • Modulation of prekallikrein (PKK) expression
    Patent number: 11840686
    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: July 2, 2021
    Date of Patent: December 12, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Huynh-Hoa Bui
  • Oligomeric compounds for reducing DMPK expression
    Patent number: 11833221
    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: Grant
    Filed: August 31, 2022
    Date of Patent: December 5, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Frank Rigo, Chrissa A. Dwyer
  • Compounds and methods for increasing STMN2 expression
    Patent number: 11833168
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include ataxia, neuropathy, synaptic dysfunction, deficits in cognition, and decreased longevity. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and dementia with Lewy bodies (DLB).
    Type: Grant
    Filed: June 14, 2019
    Date of Patent: December 5, 2023
    Assignees: Ionis Pharmaceuticals, Inc., Ludwig Institute For Cancer Research
    Inventors: Huynh-Hoa Bui, Don W. Cleveland, Ze'ev Melamed
  • Compositions for modulating Ataxin 2 expression
    Patent number: 11834660
    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: Grant
    Filed: August 4, 2021
    Date of Patent: December 5, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Gene Hung, C. Frank Bennett
  • COMPOUNDS AND METHODS FOR MODULATING PMP22
    Publication number: 20230374519
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PMP22 RNA in a cell or animal, and in certain instances reducing the amount of PMP22 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 demyelination, progressive axonal damage and/or loss, weakness and wasting of foot and lower leg muscles, foot deformities, and weakness and atrophy in the hands. Such neurodegenerative diseases include Charcot-Marie-Tooth disease.
    Type: Application
    Filed: June 18, 2021
    Publication date: November 23, 2023
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Eric E. Swayze, Holly Kordasiewicz, Punit P. Seth, Hien Thuy Zhao, Michael T. Migawa, Ruben E. Valas, Thazha P. Prakash