Patents by Inventor Louise Rodino-Klapac

Louise Rodino-Klapac 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).

  • Systemic delivery of adeno-associated virus vector expressing gamma-sarcoglycan and the treatment of muscular dystrophy
    Patent number: 11925675
    Abstract: Described herein are methods of treating muscular dystrophy comprising administering to a subject a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCG vector, methods of expressing gamma-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV scAAVrh74.MHCK7.hSGCG for usage in such methods, the doses and the ranges thereof for such methods, and methods of generating and producing the rAAV scAAVrh74.MHCK7.hSGCG.
    Type: Grant
    Filed: September 7, 2021
    Date of Patent: March 12, 2024
    Assignee: Sarepta Therapeutics, Inc.
    Inventors: Louise Rodino-Klapac, Eric Pozsgai
  • Methods of treating muscular dystrophy
    Patent number: 11820972
    Abstract: The invention provides for AAV vectors expressing the ANO5 gene and antioxidant therapy as methods of inducing muscle regeneration and a method of treating muscular dystrophy.
    Type: Grant
    Filed: November 11, 2016
    Date of Patent: November 21, 2023
    Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
    Inventor: Louise Rodino-Klapac
  • Adeno-Associated Virus Vector Delivery of Muscle Specific Micro-Dystrophin to Treat Muscular Dystrophy
    Publication number: 20230302157
    Abstract: The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
    Type: Application
    Filed: December 27, 2022
    Publication date: September 28, 2023
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell
  • Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
    Publication number: 20230279431
    Abstract: Described herein are methods of treating muscular dystrophy comprising administering a self complementary recombinant AAV (rAAV) scAAVrh74.tMCK.hSGCA vector, methods of expressing alpha-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.
    Type: Application
    Filed: November 30, 2022
    Publication date: September 7, 2023
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell
  • GENE THERAPY WITH DYSFERLIN DUAL VECTORS
    Publication number: 20230279065
    Abstract: Recombinant polynucleotides encoding fragments of a human dysferlin protein are described herein. In addition, plasmids, viral vectors, dual vector systems, cells, and compositions comprising such recombinant polynucleotides are further described. Such recombinant polynucleotides, plasmids, viral vectors, dual vector systems, cells, and compositions may be used to treat dysfer-linopathies.
    Type: Application
    Filed: May 12, 2021
    Publication date: September 7, 2023
    Inventor: Louise Rodino-Klapac
  • Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
    Patent number: 11723986
    Abstract: The invention provides for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
    Type: Grant
    Filed: April 14, 2017
    Date of Patent: August 15, 2023
    Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell
  • ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND THE TREATMENT OF MUSCULAR DYSTROPHY
    Publication number: 20230241252
    Abstract: Described herein are methods of treating muscular dystrophy comprising administering a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.
    Type: Application
    Filed: February 26, 2020
    Publication date: August 3, 2023
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell
  • MATERIALS AND METHODS FOR TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINOPATHIES
    Publication number: 20230233651
    Abstract: The present application provides materials and methods for treating a patient with a titin-based myopathy, particularly a titin-based cardiomyopathy, and/or other titinopathy. In addition, the present application provides materials and methods for editing the titin gene in a cell by genome editing.
    Type: Application
    Filed: February 1, 2023
    Publication date: July 27, 2023
    Inventors: Louise Rodino-Klapac, Rachael Potter
  • ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
    Publication number: 20230173101
    Abstract: The invention provide for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
    Type: Application
    Filed: June 10, 2022
    Publication date: June 8, 2023
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell
  • Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
    Publication number: 20230139985
    Abstract: Described herein are methods of treating muscular dystrophy comprising administering a self complementary recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.
    Type: Application
    Filed: October 14, 2022
    Publication date: May 4, 2023
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell
  • Repairing a mutant human titin gene using CRISPR technology
    Patent number: 11617783
    Abstract: The present application provides materials and methods for treating a patient with a titin-based myopathy, particularly a titin-based cardiomyopathy, and/or other titinopathy. In addition, the present application provides materials and methods for editing the titin gene in a cell by genome editing.
    Type: Grant
    Filed: November 15, 2016
    Date of Patent: April 4, 2023
    Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
    Inventors: Louise Rodino-Klapac, Rachael Potter
  • COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING THE DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
    Publication number: 20230040544
    Abstract: Disclosed herein are methods of repairing or restoring a sarcoglycan complex or DAPC, stabilizing DAPC, restoring DAPC function, or increasing or enhancing expression of one or more components of a sarcoglycan complex or DAPC in a subject suffering from a muscular dystrophy.
    Type: Application
    Filed: December 15, 2020
    Publication date: February 9, 2023
    Inventors: Eric Pozsgai, Louise Rodino-Klapac
  • Adeno-Associated Virus Vector Delivery of Microrna-29 to Treat Muscular Dystrophy
    Publication number: 20230001015
    Abstract: The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
    Type: Application
    Filed: April 12, 2022
    Publication date: January 5, 2023
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell, Kristin N. Heller
  • Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
    Patent number: 11534501
    Abstract: The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
    Type: Grant
    Filed: March 16, 2018
    Date of Patent: December 27, 2022
    Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell
  • Recombinant Adeno-Associated Virus Delivery of Alpha-Sarcoglycan Polynucleotides
    Publication number: 20220370639
    Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of an alpha-sarcoglycan gene. The invention provides rAAV products and methods of using the rAAV in the treatment of limb girdle muscular dystrophies such as LGMD2D.
    Type: Application
    Filed: May 17, 2022
    Publication date: November 24, 2022
    Inventors: Jerry R. Mendell, Louis Chicoine, Louise Rodino-Klapac, Kelly Reed Clark, Thomas J. Preston
  • Adeno-Associated Virus Vector Delivery of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
    Publication number: 20220364117
    Abstract: The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
    Type: Application
    Filed: June 3, 2022
    Publication date: November 17, 2022
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell
  • Adeno-associated virus antibodies, fragments thereof and encoding polynucleotides
    Patent number: 11472866
    Abstract: The present disclosure relates to an isolated anti-AAV (adeno-associated virus) antibody or an antigen-binding fragment thereof capable of specifically binding an epitope of AAVrh74 capsid protein and uses thereof.
    Type: Grant
    Filed: September 17, 2021
    Date of Patent: October 18, 2022
    Assignee: Sarepta Therapeutics, Inc.
    Inventors: Sohrab Khan, Danielle Griffin, Louise Rodino-Klapac
  • Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
    Patent number: 11446396
    Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of an alpha-sarcoglycan gene. The invention provides rAAV products and methods of using the rAAV in the treatment of limb girdle muscular dystrophies such as LGMD2D.
    Type: Grant
    Filed: September 19, 2018
    Date of Patent: September 20, 2022
    Assignee: NATIONWIDE CHILDREN'S HOSPITAL, INC.
    Inventors: Jerry R. Mendell, Louis Chicoine, Louise Rodino-Klapac, Kelly Reed Clark, Thomas J. Preston
  • ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF ALPHA-SARCOGLYCAN AND THE TREATMENT OF MUSCULAR DYSTROPHY
    Publication number: 20220290180
    Abstract: Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.hSCGA using a systemic route of administration and at a dose of about 1.0×1012 vg/kg to about 5.0×1015 vg/kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject.
    Type: Application
    Filed: August 21, 2020
    Publication date: September 15, 2022
    Inventors: Louise Rodino-Klapac, Danielle Griffin, Jerry R. Mendell
  • ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND MICRORNA-29 AND THE TREATMENT OF MUSCULAR DYSTROPHY
    Publication number: 20220251156
    Abstract: Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising ?-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vector(s) expressing ?-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
    Type: Application
    Filed: January 20, 2022
    Publication date: August 11, 2022
    Inventors: Louise Rodino-Klapac, Jerry R. Mendell