Patents by Inventor Marco A. Passini

Marco A. Passini 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: 10821154
    Abstract: The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.
    Type: Grant
    Filed: May 1, 2013
    Date of Patent: November 3, 2020
    Assignee: Genzyme Corporation
    Inventors: Marco A. Passini, Lamya S. Shihabuddin, Catherine R. O'Riordan, Seng H. Cheng
  • Publication number: 20200306387
    Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
    Type: Application
    Filed: March 3, 2020
    Publication date: October 1, 2020
    Inventors: Marco A. PASSINI, James DODGE
  • Patent number: 10765760
    Abstract: Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.
    Type: Grant
    Filed: May 30, 2018
    Date of Patent: September 8, 2020
    Assignee: Sarepta Therapeutics, Inc.
    Inventors: Gunnar J. Hanson, Marco A. Passini, Frederick Joseph Schnell
  • Patent number: 10744210
    Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.
    Type: Grant
    Filed: November 2, 2007
    Date of Patent: August 18, 2020
    Assignee: GENZYME CORPORATION
    Inventors: James Dodge, Lamya Shihabuddin, Marco Passini, Seng H. Cheng, Catherine O'Riordan
  • Publication number: 20200254002
    Abstract: The present disclosure relates to methods of treating Duchenne's Muscular Dystrophy by administering an antisense oligonucleotide that induces exon skipping and a non-steroidal anti-inflammatory compound.
    Type: Application
    Filed: September 28, 2018
    Publication date: August 13, 2020
    Applicants: SAREPTA THERAPEUTICS, INC., CATABASIS PHARMACEUTICALS, INC.
    Inventors: Marco A. PASSINI, Jill C. MILNE, Andrew J. NICHOLS
  • Publication number: 20200248178
    Abstract: The present disclosure relates to methods of treating Duchenne's Muscular Dystrophy by administering an antisense oligonucleotide that induces exon skipping and a non-steroidal anti-inflammatory compound.
    Type: Application
    Filed: September 28, 2018
    Publication date: August 6, 2020
    Inventors: Marco A. PASSINI, Jill C. MILNE, Andrew J. NICHOLS
  • Patent number: 10632213
    Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
    Type: Grant
    Filed: November 2, 2007
    Date of Patent: April 28, 2020
    Assignee: GENZYME CORPORATION
    Inventors: Marco A. Passini, James Dodge
  • Publication number: 20200078465
    Abstract: Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping are described.
    Type: Application
    Filed: December 14, 2017
    Publication date: March 12, 2020
    Inventors: Marco A. PASSINI, Gunnar J. HANSON
  • Publication number: 20200080066
    Abstract: This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).
    Type: Application
    Filed: August 19, 2019
    Publication date: March 12, 2020
    Inventors: Marco A. PASSINI, Robin J. ZIEGLER, James C. DODGE, Lamya SHIHABUDDIN, Seng H. CHENG
  • Publication number: 20190365919
    Abstract: Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.
    Type: Application
    Filed: May 30, 2018
    Publication date: December 5, 2019
    Applicant: Sarepta Therapeutics, Inc.
    Inventors: Gunnar J. Hanson, Marco A. Passini, Frederick Joseph Schnell
  • Publication number: 20190328682
    Abstract: Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.
    Type: Application
    Filed: February 21, 2019
    Publication date: October 31, 2019
    Inventors: James C. DODGE, Marco A. PASSINI, Lamya S. SHIHABUDDIN, Seng H. CHENG
  • Patent number: 10369193
    Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
    Type: Grant
    Filed: May 20, 2016
    Date of Patent: August 6, 2019
    Assignee: Genzyme Corporation
    Inventors: Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng
  • Publication number: 20190083582
    Abstract: Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
    Type: Application
    Filed: August 23, 2018
    Publication date: March 21, 2019
    Inventors: James DODGE, Marco A. PASSINI, Lamya SHIHABUDDIN, Seng H. CHENG
  • Publication number: 20190030058
    Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
    Type: Application
    Filed: February 9, 2018
    Publication date: January 31, 2019
    Applicants: Biogen MA Inc., Cold Spring Harbor Laboratory
    Inventors: C. Frank Bennett, Gene Hung, Frank Rigo, Adrian R. Krainer, Yimin Hua, Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng, Katherine W. Klinger
  • Publication number: 20190015440
    Abstract: The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and methods for treating spinal muscular atrophy and relates to inducing inclusion of exon 7 in SMN2 mRNA.
    Type: Application
    Filed: August 26, 2016
    Publication date: January 17, 2019
    Inventors: Marco A. Passini, Gunnar J. Hanson
  • Publication number: 20180271993
    Abstract: Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
    Type: Application
    Filed: June 6, 2018
    Publication date: September 27, 2018
    Applicant: Sarepta Therapeutics, Inc.
    Inventors: Marco A. PASSINI, Gunnar J. HANSON
  • Patent number: 10080783
    Abstract: Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
    Type: Grant
    Filed: December 3, 2014
    Date of Patent: September 25, 2018
    Assignee: Genzyme Corporation
    Inventors: James Dodge, Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng
  • Publication number: 20180177814
    Abstract: Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.
    Type: Application
    Filed: December 13, 2017
    Publication date: June 28, 2018
    Inventors: Marco A. PASSINI, Gunnar J. HANSON
  • Patent number: 9717750
    Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
    Type: Grant
    Filed: February 9, 2015
    Date of Patent: August 1, 2017
    Assignees: Biogen MA Inc., Cold Spring Harbor Laboratory
    Inventors: C. Frank Bennett, Gene Hung, Frank Rigo, Adrian R. Krainer, Yimin Hua, Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng, Katherine W. Klinger
  • Publication number: 20170152524
    Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
    Type: Application
    Filed: July 1, 2016
    Publication date: June 1, 2017
    Inventors: Marco A. PASSINI, Greg STEWART, James C. DODGE