Patents Assigned to Ionis Pharmaceuticals, Inc.
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • Patent number: 10751419
    Abstract: Provided herein are improved methods for the synthesis of reactive conjugate clusters and intermediates used in such methods. In particular, improvements are provided that enhance the synthesis of reactive conjugate clusters by reducing the number of synthetic steps required. The reactive conjugate clusters prepared using the improved methods don't include any transacylation impurities that are formed using existing methods. The improved methods also provide an increase in overall yield and a cost benefit over existing methods.
    Type: Grant
    Filed: September 7, 2018
    Date of Patent: August 25, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Michael T. Migawa, Jinghua Yu, W. Brad Wan, Sayten P. Patel, Guillermo Vasquez, Garth A. Kinberger, Thazha P. Prakash, Punit P. Seth, Eric E. Swayze
  • Patent number: 10745756
    Abstract: Described herein are compounds useful for the treatment and investigation of diseases, methods for the prediction of in vivo toxicity of compounds useful for the treatment and investigation of diseases, and methods of discovering and identifying compounds useful for the treatment and investigation of diseases that have reduced in vivo toxicity.
    Type: Grant
    Filed: July 25, 2017
    Date of Patent: August 18, 2020
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Sebastien Burel