Patents Assigned to Ionis Pharmaceuticals, Inc.
  • COMPOSITIONS AND METHODS FOR MODULATING GROWTH HORMONE RECEPTOR EXPRESSION
    Publication number: 20200123551
    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: July 24, 2019
    Publication date: April 23, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Sanjay Bhanot, Susan M. Freier, Huynh-Hoa Bui
  • MODULATION OF ANGIOPOIETIN-LIKE 3 EXPRESSION
    Publication number: 20200115711
    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: Application
    Filed: December 20, 2019
    Publication date: April 16, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Rosanne M. Crooke, Mark J. Graham, Richard Lee, Kenneth W. Dobie
  • Cancer treatment
    Patent number: 10619157
    Abstract: In certain embodiments, methods, compounds, and compositions for treating B-cell lymphoma or hepatocellular carcinoma by inhibiting expression of STAT3 mRNA or protein in an animal are provided herein. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate B-cell lymphoma or hepatocellular carcinoma.
    Type: Grant
    Filed: November 27, 2017
    Date of Patent: April 14, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Stanley T. Crooke, Mason Yamashita
  • Method for solution phase detritylation of oligomeric compounds
    Patent number: 10618931
    Abstract: Provided herein are methods for the synthesis of oligomeric compounds wherein removal of the 5?-terminal trityl group is performed at reduced temperature and lower pH relative to standard methods. In certain embodiments, the present methods provide detritylated oligomeric compounds having a reduced percentage of depurination relative to the same detritylated oligomeric compounds prepared using standard methods. In certain embodiments, the present methods provide detritylated oligomeric compounds with increased purity relative to the same detritylated oligomeric compounds prepared using standard methods. In certain embodiments, the present methods provide an increased rate of detritylation compared to standard methods.
    Type: Grant
    Filed: July 27, 2017
    Date of Patent: April 14, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Dana Chreng
  • Modulation of huntingtin expression
    Patent number: 10619158
    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: February 8, 2019
    Date of Patent: April 14, 2020
    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
  • MODULATION OF APOLIPOPROTEIN C-III (APOCIII) EXPRESSION IN LIPODYSTROPHY POPULATIONS
    Publication number: 20200095581
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of ApoCIII mRNA and protein in a patient with Partial Lipodystrophy. Also provided herein are methods, compounds, and compositions for treating, preventing, delaying, or ameliorating Partial Lipodystrophy in a patient. Further provided herein are methods, compounds, and compositions useful to treat, prevent, delay, or ameliorate any one or more of pancreatitis, cardiovascular disease or metabolic disorder, or a symptom thereof, associated with Partial Lipodystrophy in a patient.
    Type: Application
    Filed: July 31, 2019
    Publication date: March 26, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Andres Digenio
  • COMPOSITIONS FOR MODULATING ATAXIN 2 EXPRESSION
    Publication number: 20200087661
    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: April 17, 2019
    Publication date: March 19, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • Compositions and methods for modulation nucleic acids through nonsense mediated decay
    Patent number: 10590412
    Abstract: Disclosed herein are compounds, compositions and methods for modulating the amount or activity of a target nucleic acid. In certain embodiments, the amount or activity of a target nucleic acid is modulated through nonsense mediated decay.
    Type: Grant
    Filed: April 21, 2014
    Date of Patent: March 17, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Amanda Ward, Frank Rigo
  • Compositions and Methods for Modulating MECP2 Expression
    Publication number: 20200078389
    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 8, 2019
    Publication date: March 12, 2020
    Applicants: Ionis Pharmaceuticals, Inc., Baylor College of Medicine
    Inventors: Susan M. Freier, Huda Y. Zoghbi, Ezequiel Sztainberg
  • Methods for monitoring C9ORF72 expression
    Patent number: 10577604
    Abstract: Disclosed herein are methods for monitoring expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Such C9ORF72 specific inhibitors include antisense compounds.
    Type: Grant
    Filed: October 15, 2013
    Date of Patent: March 3, 2020
    Assignees: Ionis Pharmaceuticals, Inc., The Johns Hopkins University
    Inventors: C. Frank Bennett, Susan M. Freier, Jeffrey D. Rothstein, Christopher Donnelly, Rita G. Sattler
  • Modulation of DYRK1B expression
    Patent number: 10577607
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of DYRKIB in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a metabolic disease or disorder in an individual in need.
    Type: Grant
    Filed: March 16, 2017
    Date of Patent: March 3, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Brett P. Monia, Shuling Guo, Susan F. Murray
  • Conjugated antisense compounds and their use
    Patent number: 10570169
    Abstract: Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the gomeric compounds are conjugated to N-Acetylgalactosamine or to N-Acetylgalactosamine analogues.
    Type: Grant
    Filed: May 21, 2015
    Date of Patent: February 25, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Punit P. Seth, Thazha P. Prakash, Michael T. Migawa, Eric E. Swayze
  • MODULATION OF APOLIPOPROTEIN CIII (APOCIII) EXPRESSION
    Publication number: 20200056176
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of ApoCIII mRNA and protein in an animal. Also 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 an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease or metabolic disorder, or a symptom thereof.
    Type: Application
    Filed: May 28, 2019
    Publication date: February 20, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Adam Mullick, Rosanne M. Crooke, Mark J. Graham, Kenneth W. Dobie, Thomas A. Bell, III, Richard Lee
  • COMPOSITIONS AND METHODS FOR MODULATING PKK EXPRESSION
    Publication number: 20200056185
    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: March 25, 2019
    Publication date: February 20, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Susan M. Freier, Huynh-Hoa Bui
  • COMPOUNDS AND METHODS FOR REDUCING ATXN2 EXPRESSION
    Publication number: 20200056179
    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: July 25, 2019
    Publication date: February 20, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Priyam Singh, Frank Rigo, Paymaan Jafar-nejad, Holly Kordasiewicz
  • SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
    Publication number: 20200056187
    Abstract: The present disclosure 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, certain oligomeric compounds selectively reduce the expression of a target nucleic acid transcript relative to a non-target nucleic acid transcript.
    Type: Application
    Filed: March 29, 2019
    Publication date: February 20, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Michael Oestergaard, Punit P. Seth, Eric E. Swayze
  • COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION
    Publication number: 20200048638
    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: March 18, 2019
    Publication date: February 13, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Tamar R. Grossman, Michael L. McCaleb, Andrew T. Watt, Susan M. Freier
  • Modulating apolipoprotein (a) expression
    Patent number: 10557137
    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: November 7, 2016
    Date of Patent: February 11, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Nicholas J. Viney, Richard S. Geary, Yanfeng Wang, Zhengrong Yu
  • MODULATORS OF DIACYGLYCEROL ACYLTRANSFERASE 2 (DGAT2)
    Publication number: 20200040341
    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: Application
    Filed: June 28, 2019
    Publication date: February 6, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Sanjay Bhanot, Susan M. Freier, Eric E. Swayze
  • Compounds and methods for modulation of DUX4
    Patent number: 10538763
    Abstract: The present disclosure pertains generally to double-stranded small interfering RNAs that modulate gene expression for use in research, diagnostics, and/or therapeutics. In certain embodiments, the present disclosure provides double-stranded small interfering RNAs that modulate DUX4 gene expression. In certain embodiments, the present disclosure provides methods of inhibiting DUX4 gene expression by contacting a cell with double-stranded small interfering RNAs.
    Type: Grant
    Filed: January 15, 2016
    Date of Patent: January 21, 2020
    Assignees: Ionis Pharmaceuticals, Inc., Fred Hutchinson Cancer Research Center
    Inventors: Frank Rigo, Stephen J. Tapscott