Patents Assigned to Ionis Pharmaceuticals, Inc.
  • Modulators of APOL1 expression
    Patent number: 10927377
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting APOL1 expression, which may be useful for treating, preventing, or ameliorating a disease associated with APOL1.
    Type: Grant
    Filed: May 21, 2019
    Date of Patent: February 23, 2021
    Assignee: IONIS PHARMACEUTICALS, INC.
    Inventor: Susan M. Freier
  • MODULATORS OF IRF4 EXPRESSION
    Publication number: 20210038631
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting IRF4 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with IRF4.
    Type: Application
    Filed: March 1, 2019
    Publication date: February 11, 2021
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Tianyuan Zhou, Youngsoo Kim, Robert MacLeod, Huynh-Hoa Bui, Susan M. Freier
  • COMPOUNDS AND METHODS FOR MODULATING GFAP
    Publication number: 20210038629
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GFAP RNA in a cell or subject, and in certain instances reducing the amount of GFAP in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include motor delays, cognitive delays, paroxysmal deterioration, seizures, vomiting, swallowing difficulties, ataxic gait, palatal myoclonus, autonomic dysfunction, and presence of intra-astrocytic inclusions called Rosenthal fibers. Such leukodystrophies include Alexander Disease.
    Type: Application
    Filed: July 24, 2020
    Publication date: February 11, 2021
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Berit Elissa Powers, Huynh-Hoa Bui, Susan M. Freier, Frank Rigo
  • COMPOUNDS AND METHODS FOR THE MODULATION OF AMYLOID-BETA PRECURSOR PROTEIN
    Publication number: 20210040480
    Abstract: Certain embodiments disclosed herein are directed to compounds and methods for modulating APP expression. In certain embodiments, modulating the splicing of amyloid precursor protein (APP) reduces amyloid ? (A?) production.
    Type: Application
    Filed: March 1, 2019
    Publication date: February 11, 2021
    Applicants: Ionis Pharmaceuticals, Inc., Rosalind Franklin University of Medicine and Science
    Inventors: Frank Rigo, Michelle Hastings
  • Compounds and methods for modulating angiotensinogen expression
    Patent number: 10912792
    Abstract: Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating AGT and modulating a RAAS pathway related disease, disorder and/or condition in an individual in need thereof. A RAAS pathway related disease, disorder and/or condition in an individual such as hypertension can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to AGT.
    Type: Grant
    Filed: October 7, 2016
    Date of Patent: February 9, 2021
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Adam Mullick, Mark J. Graham, Punit P. Seth, Susan M. Freier
  • Methods for reducing LRRK2 expression
    Patent number: 10907160
    Abstract: Provided herein are methods for decreasing LRRK2 mRNA expression. Such methods are useful to ameliorate LRRK2 associated diseases. Such LRRK2 associated diseases include Parkinson's Disease, including non-LRRK2 mediated Parkinson's Disease.
    Type: Grant
    Filed: January 5, 2017
    Date of Patent: February 2, 2021
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Hien Thuy Zhao, Holly Kordasiewicz
  • COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION
    Publication number: 20210024923
    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: January 28, 2021
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
  • MODIFIED COMPOUNDS AND USES THEREOF
    Publication number: 20210017513
    Abstract: The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.
    Type: Application
    Filed: February 12, 2019
    Publication date: January 21, 2021
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Punit P. Seth, Michael Oestergaard, Michael T. Migawa, Xue-hai Liang, Wen Shen, Stanley T. Crooke, Eric E. Swayze
  • METHODS AND COMPOSITIONS FOR MODULATING APOLIPOPROTEIN (A) EXPRESSION
    Publication number: 20210002643
    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: August 3, 2020
    Publication date: January 7, 2021
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Rosanne M. Crooke, Mark J. Graham, Susan M. Freier, Marc Lim, Andrew Dibble
  • MODULATION OF HSD17B13 EXPRESSION
    Publication number: 20210000906
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of HSD17B13 in a cell or individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a liver disease, metabolic disease, or cardiovascular disease or disorder, including but not limited to NASH, in an individual.
    Type: Application
    Filed: March 21, 2019
    Publication date: January 7, 2021
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan F. Murray, Jiangwei Zhang, Huynh-Hoa Bui
  • Selective Antisense Compounds and Uses Thereof
    Publication number: 20210002636
    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. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.
    Type: Application
    Filed: February 18, 2020
    Publication date: January 7, 2021
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Michael Oestergaard, Punit P. Seth, Eric E. Swayze
  • Compositions and methods for modulating apolipoprotein (a) expression
    Patent number: 10883104
    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: February 7, 2018
    Date of Patent: January 5, 2021
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Mark J. Graham
  • METHODS OF MODULATING ANTISENSE ACTIVITY
    Publication number: 20200407717
    Abstract: Disclosed herein are methods for increasing antisense activity by modulating EGFR. In certain embodiments, a compound comprising an antisense oligonucleotide is co-administered with an EGFR modulator.
    Type: Application
    Filed: February 13, 2019
    Publication date: December 31, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Shiyu Wang, Alexey Revenko, Xue-hai Liang, Stanley T. Crooke
  • Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
    Publication number: 20200392493
    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: January 21, 2020
    Publication date: December 17, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Eric E. Swayze
  • COMPOUNDS AND METHODS FOR REDUCING SNCA EXPRESSION
    Publication number: 20200392494
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein 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 of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia with Lewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.
    Type: Application
    Filed: November 9, 2018
    Publication date: December 17, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Holly Kordasiewicz, Priyam Singh, Susan M. Freier, Tracy A. Cole
  • CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE
    Publication number: 20200392509
    Abstract: The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, wherein the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound. In certain embodiments, the compounds disclosed herein are useful for modulating the expression of extra-hepatic target nucleic acids.
    Type: Application
    Filed: December 14, 2018
    Publication date: December 17, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Michael Oestergaard, Punit P. Seth, Frank Rigo, Chrissa A. Dwyer
  • Modulators of DNM2 expression
    Patent number: 10865414
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting DNM2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DNM2.
    Type: Grant
    Filed: January 15, 2019
    Date of Patent: December 15, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • Oligomeric compounds comprising ?-?-constrained nucleic acid
    Patent number: 10865413
    Abstract: The present disclosure provides oligomeric compounds comprising at least one ?-?-constrained nucleic acid as provided herein. More particularly, the ?-?-constrained nucleic acid provided herein comprise an optionally modified nucleoside with a phosphorus containing constrained internucleoside linkage such as for example a cyclic phosphate internucleoside linkage. The ?-?-constrained nucleic acid provided herein are expected to be useful for enhancing one or more properties of oligomeric compounds they are incorporated into such as for example nuclease resistance. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.
    Type: Grant
    Filed: January 4, 2019
    Date of Patent: December 15, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Michael Oestergaard, Eric E. Swayze, Punit P. Seth
  • 5' MODIFIED NUCLEOSIDES AND OLIGOMERIC COMPOUNDS PREPARED THEREFROM
    Publication number: 20200385722
    Abstract: The present invention provides 5? modified nucleosides and oligomeric compounds prepared therefrom. More particularly, the present invention provides modified nucleosides having at least one 5?-substituent and an optional 2? substituent, oligomeric compounds comprising at least one of these modified nucleosides and methods of using the oligomeric compounds. In some embodiments, the oligomeric compounds provided herein are expected to hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.
    Type: Application
    Filed: April 30, 2020
    Publication date: December 10, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
  • Methods for treating hypercholesterolemia
    Patent number: 10858657
    Abstract: Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.
    Type: Grant
    Filed: August 7, 2018
    Date of Patent: December 8, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Rosanne M. Crooke, Mark J. Graham, Kristina M. Lemonidis, Diane Tribble, Sanjay Bhanot, Andrew T. Watt