Patents by Inventor Ryszard Kole

Ryszard Kole has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • Oligonucleotide analogues targeting human LMNA
    Patent number: 11802283
    Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Grant
    Filed: September 17, 2020
    Date of Patent: October 31, 2023
    Assignees: Sarepta Therapeutics, Inc., The United States of America, as rep. by the Secretary, Dept. of Health and Human Services, The Progeria Research Foundation
    Inventors: Michael R. Erdos, Francis S. Collins, Kan Cao, Ryszard Kole, Richard Keith Bestwick, Leslie B. Gordon
  • Compound and method for treating myotonic dystrophy
    Patent number: 11236329
    Abstract: An antisense compound for use in treating myotonic dystrophy DM1 or DM2, a method of enhancing antisense targeting to heart and quadricep muscles, and a method for treating DM1 or DM2 in a mammalian subject are disclosed. The oligonucleotide has 8-30 bases, with at least 8 contiguous bases being complementary to the polyCUG or polyCCUG repeats in the 3?UTR region of dystrophia myotonica protein kinase (DMPK) mRNA in DM1 or DM2, respectively. Conjugated to the oligonucleotide is a cell-penetrating peptide having the sequence (RXRR(B/X)R)2XB, where R is arginine; B is ?-alanine; and each X is —C(O)—(CH2)n—NH—, where n is 4-6. The antisense compound is effective to selectively block the sequestration of muscleblind-like 1 protein (MBNL1) and/or CUGBP, in heart and quadricep muscle in a myotonic dystrophy animal model.
    Type: Grant
    Filed: April 24, 2014
    Date of Patent: February 1, 2022
    Assignee: Sarepta Therapeutics, Inc.
    Inventors: Hong M. Moulton, Ryszard Kole
  • COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY
    Publication number: 20220002733
    Abstract: Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3?UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
    Type: Application
    Filed: February 5, 2021
    Publication date: January 6, 2022
    Inventors: Gunnar J. Hanson, Ryszard Kole
  • Oligonucleotide analogues targeting human LMNA
    Patent number: 11071749
    Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Grant
    Filed: April 17, 2019
    Date of Patent: July 27, 2021
    Assignee: SAREPTA THERAPEUTICS, INC.
    Inventors: Ryszard Kole, Richard Keith Bestwick
  • MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
    Publication number: 20210180060
    Abstract: Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.
    Type: Application
    Filed: June 12, 2020
    Publication date: June 17, 2021
    Applicant: Sarepta Therapeutics, Inc.
    Inventors: Peter SAZANI, Ryszard Kole
  • Compound and method for treating myotonic dystrophy
    Patent number: 10927378
    Abstract: Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3?UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
    Type: Grant
    Filed: October 18, 2018
    Date of Patent: February 23, 2021
    Assignee: Sarepta Therapeutics, Inc.
    Inventors: Ryszard Kole, Gunnar J. Hanson
  • OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA
    Publication number: 20210010001
    Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Application
    Filed: September 17, 2020
    Publication date: January 14, 2021
    Applicants: Sarepta Therapeutics, Inc., The Progeria Research Foundation, THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES, University of Maryland, The Progeria Research Foundation
    Inventors: Michael R. Erdos, Francis S. Collins, Kan Cao, Ryszard Kole, Richard Keith Bestwick, Leslie B. Gordon
  • Oligonucleotide analogues targeting human LMNA
    Patent number: 10822608
    Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Grant
    Filed: April 28, 2017
    Date of Patent: November 3, 2020
    Assignees: Sarepta Therapeutics, Inc., THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES, University of Maryland, The Progeria Research Foundation
    Inventors: Michael R. Erdos, Francis S. Collins, Kan Cao, Ryszard Kole, Richard Keith Bestwick, Leslie B. Gordon
  • Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
    Publication number: 20200283776
    Abstract: Methods and compositions are disclosed for controlling expression of TNF receptors (TNFR1 and TNFR2) and of other receptors in the TNFR superfamily using compounds that modulate splicing of pre-mRNA encoding these receptors. More specifically these compounds cause the removal of the transmembrane domains of these receptors and produce soluble forms of the receptor which act as an antagonist to reduce TNF-? activity or activity of the relevant ligand. Reducing TNF-? activity provides a method of treating or ameliorating inflammatory diseases or conditions associated with TNF-? activity. Similarly, diseases associated with other ligands can be treated in like manner. In particular, the compounds of the invention are splice-splice switching oligomers (SSOs) which are small molecules that are stable in vivo, hybridize to the RNA in a sequence specific manner and, in conjunction with their target, are not degraded by RNAse H.
    Type: Application
    Filed: October 22, 2019
    Publication date: September 10, 2020
    Inventors: Peter L. Sazani, Ryszard Kole, Henrik Orum
  • OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA
    Publication number: 20200147119
    Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Application
    Filed: April 17, 2019
    Publication date: May 14, 2020
    Inventors: Ryszard KOLE, Richard Keith BESTWICK
  • MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
    Publication number: 20190284556
    Abstract: Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.
    Type: Application
    Filed: September 28, 2018
    Publication date: September 19, 2019
    Applicant: Sarepta Therapeutics, Inc.
    Inventors: Peter Sazani, Ryszard Kole
  • Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA
    Patent number: 10398721
    Abstract: Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Grant
    Filed: October 6, 2017
    Date of Patent: September 3, 2019
    Assignees: SAREPTA THERAPEUTICS, INC., THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES, UNIVERSITY OF MARYLAND, PROGERIA RESEARCH FOUNDATION, INC.
    Inventors: Ryszard Kole, Francis S. Collins, Michael R. Erdos, Kan Cao, Leslie B. Gordon
  • COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY
    Publication number: 20190264210
    Abstract: Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3?UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
    Type: Application
    Filed: October 18, 2018
    Publication date: August 29, 2019
    Inventors: Ryszard Kole, Gunnar J. Hanson
  • MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
    Publication number: 20190127738
    Abstract: Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.
    Type: Application
    Filed: January 9, 2019
    Publication date: May 2, 2019
    Applicant: Sarepta Therapeutics, Inc.
    Inventors: Peter SAZANI, Ryszard KOLE
  • OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA
    Publication number: 20190127735
    Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Application
    Filed: April 28, 2017
    Publication date: May 2, 2019
    Applicants: Sarepta Therapeutics, Inc., THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES, UNIVERSITY OF MARYLAND
    Inventors: Michael R. Erdos, Francis S. Collins, Kan Cao, Ryszard Kole, Richard Keith Bestwick, Leslie B. Gordon
  • Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
    Publication number: 20190017052
    Abstract: Methods and compositions are disclosed for controlling expression of TNF receptors (TNFR1 and TNFR2) and of other receptors in the TNFR superfamily using compounds that modulate splicing of pre-mRNA encoding these receptors. More specifically these compounds cause the removal of the transmembrane domains of these receptors and produce soluble forms of the receptor which act as an antagonist to reduce TNF-? activity or activity of the relevant ligand. Reducing TNF-? activity provides a method of treating or ameliorating inflammatory diseases or conditions associated with TNF-? activity. Similarly, diseases associated with other ligands can be treated in like manner. In particular, the compounds of the invention are splice-splice switching oligomers (SSOs) which are small molecules that are stable in vivo, hybridize to the RNA in a sequence specific manner and, in conjunction with their target, are not degraded by RNAse H.
    Type: Application
    Filed: July 19, 2018
    Publication date: January 17, 2019
    Inventors: Peter L. Sazani, Ryszard Kole, Henrik Orum
  • Compound and method for treating myotonic dystrophy
    Patent number: 10106796
    Abstract: Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3?UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
    Type: Grant
    Filed: August 20, 2014
    Date of Patent: October 23, 2018
    Assignee: SAREPTA THERAPEUTICS, INC.
    Inventors: Ryszard Kole, Gunnar J. Hanson
  • METHODS FOR TREATING PROGEROID LAMINOPATHIES USING OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA
    Publication number: 20180271893
    Abstract: Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Application
    Filed: October 6, 2017
    Publication date: September 27, 2018
    Inventors: RYSZARD KOLE, Francis S. Collins, Michael R. Erdos, Kan Cao
  • Oligonucleotide analogues targeting human LMNA
    Patent number: 10076536
    Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Grant
    Filed: May 15, 2017
    Date of Patent: September 18, 2018
    Assignee: SAREPTA THERAPEUTICS, INC.
    Inventors: Ryszard Kole, Richard Keith Bestwick
  • OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA
    Publication number: 20180092938
    Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
    Type: Application
    Filed: May 15, 2017
    Publication date: April 5, 2018
    Inventors: Ryszard KOLE, Richard Keith BESTWICK