Patents Assigned to Ionis Pharmaceuticals, Inc.
  • Selective Reduction of Allelic Variants
    Publication number: 20200377946
    Abstract: Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).
    Type: Application
    Filed: January 14, 2020
    Publication date: December 3, 2020
    Applicants: Ionis Pharmaceuticals, Inc., The University of British Columbia
    Inventors: C. Frank Bennett, Michael Hayden, Susan M. Freier, Sarah Greenlee, Jeffrey Carroll, Simon Warby, Eric E. Swayze
  • Modulation of SMN expression
    Patent number: 10851371
    Abstract: Certain embodiments are directed to methods and compounds for inhibiting SMN-NAT, the natural antisense transcript of SMN. Such methods and compounds are useful for increasing expression of SMN in cells and animals.
    Type: Grant
    Filed: April 11, 2016
    Date of Patent: December 1, 2020
    Assignees: Ionis Pharmaceuticals, Inc., The Johns Hopkins University
    Inventors: Frank Rigo, C. Frank Bennett, Constantin Van Outryve D'Ydewalle, Charlotte J. Sumner
  • Conjugated antisense compounds and their use
    Patent number: 10844379
    Abstract: Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
    Type: Grant
    Filed: December 26, 2018
    Date of Patent: November 24, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
  • MODULATING APOLIPOPROTEIN (A) EXPRESSION
    Publication number: 20200362340
    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.
    Type: Application
    Filed: December 30, 2019
    Publication date: November 19, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Nicholas J. Viney, Richard S. Geary, Yanfeng Wang, Zhengrong Yu
  • Modulation of Dystrophia Myotonica-Protein Kinase (DMPK) Expression
    Publication number: 20200362353
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
    Type: Application
    Filed: December 20, 2019
    Publication date: November 19, 2020
    Applicants: Ionis Pharmaceuticals, Inc., University of Rochester
    Inventors: C. Frank Bennett, Susan M. Freier, Robert A. MacLeod, Sanjay K. Pandey, Charles A. Thornton, Thurman Wheeler, Seng H. Cheng, Andrew Leger, Bruce M. Wentworth
  • Modulation of huntingtin expression
    Patent number: 10837016
    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 26, 2020
    Date of Patent: November 17, 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
  • Compounds and methods for the modulation of proteins
    Patent number: 10822369
    Abstract: In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the uORF inhibitor is an antisense compound.
    Type: Grant
    Filed: November 16, 2015
    Date of Patent: November 3, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Stanley T. Crooke, Xue-hai Liang, Wen Shen
  • Compositions for modulating C9ORF72 expression
    Patent number: 10815483
    Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal. Such methods are useful to treat, prevent, ameliorate, or slow progression of neurodegenerative diseases in an individual in need thereof.
    Type: Grant
    Filed: October 17, 2018
    Date of Patent: October 27, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Frank Rigo
  • Modulation of alpha synuclein expression
    Patent number: 10815480
    Abstract: Disclosed herein are antisense compounds and methods for decreasing alpha-synuclein mRNA and protein expression. Also disclosed herein are methods for treating, preventing, and ameliorating neurodegenerative diseases in an individual in need thereof.
    Type: Grant
    Filed: April 18, 2017
    Date of Patent: October 27, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • Compositions for modulating expression of C9ORF72 antisense transcript
    Patent number: 10793855
    Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 antisense transcript in an animal with C9ORF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 antisense transcript specific inhibitors include antisense compounds.
    Type: Grant
    Filed: January 6, 2016
    Date of Patent: October 6, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Frank Rigo
  • Compositions and methods for modulating growth hormone receptor expression
    Patent number: 10793862
    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: July 24, 2019
    Date of Patent: October 6, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Sanjay Bhanot, Susan M. Freier, Huynh-Hoa Bui
  • COMBINATIONS FOR THE MODULATION OF SMN EXPRESSION
    Publication number: 20200308580
    Abstract: Certain embodiments are directed to methods and compounds for modulating expression of SMN. In certain embodiments at least two compounds are used: a first compound for inhibiting SMN-NAT and increasing expression of SMN, and a second compound for modulating the splicing of SMN. Such methods and compounds are useful for increasing expression exon 7 containing SMN mRNA in cells and animals.
    Type: Application
    Filed: June 16, 2017
    Publication date: October 1, 2020
    Applicants: Ionis Pharmaceuticals, Inc., The Johns Hopkins University
    Inventors: Frank Rigo, C. Frank Bennett, Constantin Van Outryve D'Ydewalle, Charlotte J. Sumner
  • Modulation of Factor 11 expression
    Patent number: 10772906
    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: February 12, 2019
    Date of Patent: September 15, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • Modulators of PNPLA3 expression
    Patent number: 10774333
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting PNPLA3 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PNPLA3.
    Type: Grant
    Filed: September 18, 2019
    Date of Patent: September 15, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Huynh-Hoa Bui
  • ANTISENSE COMPOUNDS
    Publication number: 20200276221
    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: October 21, 2019
    Publication date: September 3, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Eric E. Swayze
  • MODULATORS OF MALAT1 EXPRESSION
    Publication number: 20200277604
    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 27, 2020
    Publication date: September 3, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Youngsoo Kim, Robert MacLeod
  • 2'-METHOXY SUBSTITUTED OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN GENE MODULATIONS
    Publication number: 20200276222
    Abstract: Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification. In some embodiments the modification is a 2?-OCH3 substituent group on a sugar moiety. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleotide of the oligomer has a modified sugar and/or backbone modification.
    Type: Application
    Filed: October 8, 2019
    Publication date: September 3, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventor: Stanley T. Crooke
  • METHODS AND COMPOSITIONS FOR MODULATING APOLIPOPROTEIN (A) EXPRESSION
    Publication number: 20200276224
    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: October 9, 2019
    Publication date: September 3, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Rosanne M. Crooke, Mark J. Graham, Susan M. Freier, Marc Lim, Andrew Dibble
  • Single-Stranded RNAI Oligonucleotides Targeting APOC-III
    Publication number: 20200270609
    Abstract: The present disclosure pertains generally to chemically-modified oligonucleotides for use in research, diagnostics, and/or therapeutics. In certain embodiments, the present disclosure describes compounds and methods for the modulation of a target nucleic acid. In certain embodiments, the present disclosure describes compounds and methods for the modulation of Apoliprotein C-III expression.
    Type: Application
    Filed: March 9, 2020
    Publication date: August 27, 2020
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Walter F. Lima, Garth A. Kinberger, Heather Murray, Eric E. Swayze, Stanley T. Crooke
  • Compounds and methods for detecting oligonucleotides
    Patent number: 10752940
    Abstract: The present invention provides methods of detecting and/or quantitating a target oligonucleotide in a biological sample.
    Type: Grant
    Filed: November 10, 2014
    Date of Patent: August 25, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Sarah Greenlee, Zhengrong Yu, Thazha P. Prakash, John E. Matson