Patents Assigned to Ionis Pharmaceuticals, Inc.
  • 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
  • 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
  • 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
  • Publication number: 20220184112
    Abstract: The present disclosure provides compositions for enhanced intestinal absorption of oligomeric compounds.
    Type: Application
    Filed: November 9, 2021
    Publication date: June 16, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Justin Drew Searcy, Zhengrong Yu, Julie C. Ma, Yanfeng Wang, Kaustubh H. Kulkarni
  • Publication number: 20220186222
    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: February 15, 2022
    Publication date: June 16, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Brooke A. Anderson, Xue-hai Liang, William John Drury, III, Michael Oestergaard, Michael T. Migawa, Punit P. Seth
  • Publication number: 20220177893
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of KCNT1 RNA in a cell or subject, and in certain instances reducing the amount of KCNT1 protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurological condition. Such symptoms and hallmarks include seizures, encephalopathy, and behavioral abnormalities. Non-limiting examples of neurological conditions that benefit from these compounds, methods, and pharmaceutical compositions are epilepsy of infancy with migrating focal seizures (EIMFS), autosomal dominant nocturnal frontal lobe epilepsy (ADNFLE), West syndrome, and Ohtahara syndrome.
    Type: Application
    Filed: March 13, 2020
    Publication date: June 9, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Huynh-Hoa Bui, Susan M. Freier
  • Publication number: 20220170023
    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: Application
    Filed: July 2, 2021
    Publication date: June 2, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Huynh-Hoa Bui
  • Publication number: 20220162603
    Abstract: The present invention provides compositions comprising at least one oligomeric compound comprising an alternating motif and further include a region that is complementary to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.
    Type: Application
    Filed: June 28, 2021
    Publication date: May 26, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Charles Allerson, Balkrishen Bhat, Anne B. Eldrup, Muthiah Manoharan, Richard H. Griffey, Brenda F. Baker, Eric E. Swayze
  • Publication number: 20220162615
    Abstract: Provided herein are methods for decreasing Ataxin-2 mRNA expression. Such methods are useful to ameliorate symptoms of Ataxin-2 associated diseases. Such Ataxin-2 associated diseases include amyotrophic lateral sclerosis (ALS). Such symptoms include loss of motor function, reduced CMAP amplitude, denervation, and loss of motor neurons.
    Type: Application
    Filed: May 18, 2021
    Publication date: May 26, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Frank Rigo
  • Patent number: 11339393
    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: Grant
    Filed: January 15, 2019
    Date of Patent: May 24, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Frank Rigo
  • Publication number: 20220153775
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of AGT RNA in a cell or subject, and in certain instances reducing the amount of AGT in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a cardiovascular disease. Such compound and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a RAAS pathway-related disease or disorder. Such symptoms and hallmarks include hypertension, chronic kidney disease, stroke, myocardial infarction, heart failure, valvular heart disease, aneurysms of the blood vessels, peripheral artery disease, and organ damage. Such cardiovascular diseases include hypertension, resistant hypertension, Marfan syndrome, and heart failure.
    Type: Application
    Filed: November 18, 2021
    Publication date: May 19, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Adam Mullick, Susan M. Freier
  • Patent number: 11332746
    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 5 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: June 27, 2019
    Date of Patent: May 17, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • Patent number: 11312962
    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: Grant
    Filed: June 28, 2019
    Date of Patent: April 26, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Sanjay Bhanot, Susan M. Freier, Eric E. Swayze
  • Patent number: 11312964
    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: Grant
    Filed: August 4, 2020
    Date of Patent: April 26, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Sanjay Bhanot, Susan M. Freier, Huynh-Hoa Bui
  • Publication number: 20220112503
    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: December 20, 2019
    Publication date: April 14, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Huynh-Hoa Bui, Susan M. Freier, Hien Thuy Zhao, Priyam Singh
  • Patent number: 11299736
    Abstract: Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
    Type: Grant
    Filed: October 14, 2020
    Date of Patent: April 12, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
  • Patent number: 11299737
    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: Grant
    Filed: June 24, 2021
    Date of Patent: April 12, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Frank Rigo
  • Patent number: 11293025
    Abstract: Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.
    Type: Grant
    Filed: November 26, 2019
    Date of Patent: April 5, 2022
    Assignees: Ionis Pharmaceuticals, Inc., Academisch Ziekenhuis Leiden
    Inventors: Frank Rigo, Thazha P. Prakash, Wilhelmina Maria Clasina Van Roon-Mom, Lodewijk Julius Anton Toonen
  • Patent number: 11279932
    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: Grant
    Filed: February 27, 2020
    Date of Patent: March 22, 2022
    Assignee: IONIS PHARMACEUTICALS, INC.
    Inventors: Susan M. Freier, Youngsoo Kim, Robert MacLeod
  • Publication number: 20220081689
    Abstract: Provided herein are methods, compounds, and compositions for modulation of dystrophin pre-mRNA in an animal. Such methods, compounds, and compositions are useful, for example, to treat, prevent, or ameliorate one or more symptoms of Duchenne Muscular Dystrophy disease.
    Type: Application
    Filed: November 18, 2020
    Publication date: March 17, 2022
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Frank Rigo, Thazha P. Prakash, Punit P. Seth