Patents by Inventor Brian K. Kaspar

Brian K. Kaspar 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).

  • Patent number: 10842886
    Abstract: The disclosure relates to methods for treating subjects with musculoskeletal diseases or with muscle wasting not associated with a musculoskeletal disease by gene transfer with recombinant adeno-associated viruses (rAAV) encoding myostatin inhibitors such as follistatin-344. The rAAV are administered prior to development of diffuse muscle fibrosis in a subject, or the rAAV administration avoids regions of muscle fibrosis in a subject.
    Type: Grant
    Filed: October 9, 2015
    Date of Patent: November 24, 2020
    Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
    Inventors: Jerry R. Mendell, Brian K. Kaspar, Samiah Al-Zaidy
  • Patent number: 10793861
    Abstract: The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.
    Type: Grant
    Filed: July 20, 2018
    Date of Patent: October 6, 2020
    Assignees: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL, LUDWIG INSTITUTE FOR CANCER RESEARCH
    Inventors: Brian K. Kaspar, Kevin Foust, Don W. Cleveland
  • Publication number: 20200297872
    Abstract: The present invention relates to Adeno-associated virus 9 methods and materials useful for systemically delivering polynucleotides across the blood brain barrier. Accordingly, the present invention also relates to methods and materials useful for systemically delivering polynucleotides to the central and peripheral nervous systems. The present invention also relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as spinal muscle atrophy and amyotrophic lateral sclerosis as well as Pompe disease and lysosomal storage disorders. Use of the methods and materials is also indicated, for example, for treatment of Rett syndrome.
    Type: Application
    Filed: June 2, 2020
    Publication date: September 24, 2020
    Inventors: Brian K. Kaspar, Kevin Foust
  • Publication number: 20200179467
    Abstract: Methods and materials for intrathecal delivery of recombinant Adeno-associated virus 9 (rAAV9) encoding Methyl-CpG binding protein 2 (MECP2) are provided. Use of the methods and materials is contemplated, for example, for the treatment of Rett syndrome.
    Type: Application
    Filed: November 17, 2017
    Publication date: June 11, 2020
    Inventors: Brian K. Kaspar, Kevin Foust
  • Publication number: 20190336618
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAAV9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Application
    Filed: July 3, 2019
    Publication date: November 7, 2019
    Inventor: Brian K. Kaspar
  • Publication number: 20190269798
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAAV9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Application
    Filed: May 8, 2019
    Publication date: September 5, 2019
    Inventors: Brian K. Kaspar, Arthur Burghes, Paul Porensky
  • Publication number: 20190262474
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAAV9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Application
    Filed: May 8, 2019
    Publication date: August 29, 2019
    Inventor: Brian K. Kaspar
  • Publication number: 20190151477
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-os-molar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Application
    Filed: June 4, 2018
    Publication date: May 23, 2019
    Inventors: Brian K. Kaspar, Arthur Burghes, Paul Porensky
  • Publication number: 20190144868
    Abstract: The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.
    Type: Application
    Filed: July 20, 2018
    Publication date: May 16, 2019
    Inventors: Brian K. Kaspar, Kevin Foust, Don W. Cleveland
  • Publication number: 20190134226
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Application
    Filed: January 14, 2019
    Publication date: May 9, 2019
    Inventors: Brian K. Kaspar, Arthur Burghes, Paul Porensky
  • Publication number: 20190134225
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Application
    Filed: January 14, 2019
    Publication date: May 9, 2019
    Inventors: Brian K. Kaspar, Arthur Burghes, Paul Porensky
  • Publication number: 20190099503
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Application
    Filed: September 12, 2018
    Publication date: April 4, 2019
    Inventors: Brian K. Kaspar, Arthur Burghes, Paul Porensky
  • Publication number: 20190030189
    Abstract: The present invention relates to Adeno-associated virus 9 methods and materials useful for systemically delivering polynucleotides across the blood brain barrier. Accordingly, the present invention also relates to methods and materials useful for systemically delivering polynucleotides to the central and peripheral nervous systems. The present invention also relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as spinal muscle atrophy and amyotrophic lateral sclerosis as well as Pompe disease and lysosomal storage disorders. Use of the methods and materials is also indicated, for example, for treatment of Rett syndrome.
    Type: Application
    Filed: October 15, 2018
    Publication date: January 31, 2019
    Inventors: Brian K. Kaspar, Kevin Foust
  • Publication number: 20180282684
    Abstract: The present invention is directed to methods and materials for producing recombinant viruses. In particular, methods and materials are provided for producing recombinant viruses in eukaryotic microalgae such as Chlamydomonas reinhardtii. Recombinant adeno-associated viruses are examples of recombinant viruses produced according to the invention.
    Type: Application
    Filed: November 5, 2015
    Publication date: October 4, 2018
    Inventor: Brian K. Kaspar
  • Publication number: 20180036431
    Abstract: The present invention relates to Adeno-associated virus 9 methods and materials useful for systemically delivering polynucleotides across the blood brain barrier. Accordingly, the present invention also relates to methods and materials useful for systemically delivering polynucleotides to the central and peripheral nervous systems. The present invention also relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as spinal muscle atrophy and amyotrophic lateral sclerosis as well as Pompe disease and lysosomal storage disorders. Use of the methods and materials is also indicated, for example, for treatment of Rett syndrome.
    Type: Application
    Filed: September 27, 2017
    Publication date: February 8, 2018
    Inventors: Brian K. Kaspar, Kevin Foust
  • Publication number: 20170246322
    Abstract: The disclosure relates to methods for treating subjects with musculoskeletal diseases or with muscle wasting not associated with a musculoskeletal disease by gene transfer with recombinant adeno-associated viruses (rAAV) encoding myostatin inhibitors such as follistatin-344. The rAAV are administered prior to development of diffuse muscle fibrosis in a subject, or the rAAV administration avoids regions of muscle fibrosis in a subject.
    Type: Application
    Filed: October 9, 2015
    Publication date: August 31, 2017
    Inventors: Jerry R. MENDELL, Brian K. KASPAR, Samiah AL-ZAIDY
  • Patent number: 9725719
    Abstract: The invention relates to pharmaceutical compositions, kits, methods, and uses for the treatment of amyotrophic lateral sclerosis. In particular, the invention relates to compositions, kits, methods, and uses for the treatment of amyotrophic lateral sclerosis by inhibiting NF-?B in microglia or macrophages and by inhibiting motor neuron death. The invention further relates to compositions, kits, methods, and uses for the treatment of amyotrophic lateral sclerosis by inhibiting NF-?B in microglia in combination with inhibiting SOD-1 in astrocytes. The invention also relates to a method for inhibiting the expression or the activity of NF-?B in microglia or macrophages to inhibit motor neuron death, alone or in combination with inhibiting SOD-1 expression in astrocytes.
    Type: Grant
    Filed: November 4, 2014
    Date of Patent: August 8, 2017
    Assignee: THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
    Inventors: Brian K. Kaspar, Ashley E. Frakes
  • Publication number: 20170216458
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Application
    Filed: April 14, 2017
    Publication date: August 3, 2017
    Inventors: Brian K. Kaspar, Arthur Burghes, Paul Porensky
  • Publication number: 20170151348
    Abstract: The present invention relates to methods for inhibitiing myostatin, a regulator of muscle mass, for muscle enhancement (including inducing hypertrophy and/or hyperplasia) as well as improving muscle function (including decreasing atrophy and/or increasing endurance, force and/or strength). Some of the methods involve delivering genes to cells using gene delivery or other delivery techniques known in the art in order to inhibit myostatin. Examples of genes to be delivered are genes encoding proteins such as Follistatin, Follistatin-related gene-1 (FLRG-1), growth differentiation factor associated protein-1 (GASP-1) and myostatin precursor propeptide. The genes can be delivered using, for example, a recombinant Adeno-associated virus (rAAV), lentivirus or equine-associated virus capable of infecting the cells. Following introduction, the genes are expressed in the cell body of the infected cell and the encoded proteins are secreted systemically.
    Type: Application
    Filed: July 13, 2016
    Publication date: June 1, 2017
    Inventors: Brian K. Kaspar, Jerry R. Mendell
  • Publication number: 20160289676
    Abstract: The invention relates to pharmaceutical compositions, kits, methods, and uses for the treatment of amyotrophic lateral sclerosis. In particular, the invention relates to compositions, kits, methods, and uses for the treatment of amyotrophic lateral sclerosis by inhibiting NF-?B in microglia or macrophages and by inhibiting motor neuron death. The invention further relates to compositions, kits, methods, and uses for the treatment of amyotrophic lateral sclerosis by inhibiting NF-?B in microglia in combination with inhibiting SOD-1 in astrocytes. The invention also relates to a method for inhibiting the expression or the activity of NF-?B in microglia or macrophages to inhibit motor neuron death, alone or in combination with inhibiting SOD-1 expression in astrocytes.
    Type: Application
    Filed: November 4, 2014
    Publication date: October 6, 2016
    Inventors: Brian K. KASPAR, Ashley E. FRAKES