Patents by Inventor Ana Maria Buj Bello

Ana Maria Buj Bello 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).

  • Selective gene therapy expression system
    Patent number: 11819478
    Abstract: The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
    Type: Grant
    Filed: May 24, 2018
    Date of Patent: November 21, 2023
    Assignees: GENETHON, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
    Inventors: Ana Maria Buj Bello, Isabelle Richard
  • Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
    Patent number: 11564977
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Grant
    Filed: December 28, 2017
    Date of Patent: January 31, 2023
    Assignees: Wake Forest University Health Sciences, Genethon, Children's Medical Center Corporation
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
    Patent number: 11499154
    Abstract: The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).
    Type: Grant
    Filed: April 10, 2018
    Date of Patent: November 15, 2022
    Assignees: GENETHON, UNIVERSITE D'EVRY-VAL-D'ESSONNE, INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
    Inventor: Ana Maria Buj Bello
  • Compositions and methods for the treatment of myotonic dystrophy
    Patent number: 11427824
    Abstract: The present invention relates to compositions and methods for the treatment of myotonic dystrophy.
    Type: Grant
    Filed: October 27, 2017
    Date of Patent: August 30, 2022
    Assignees: GENETHON, INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
    Inventors: Ana Maria Buj Bello, Mirella Lo Scrudato
  • Compositions and methods for the treatment of nucleotide repeat expansion disorders
    Patent number: 11104897
    Abstract: The present invention relates to compositions and methods for the treatment of nucleotide repeat expansion disorders such as myotonic dystrophy.
    Type: Grant
    Filed: April 27, 2016
    Date of Patent: August 31, 2021
    Assignees: GENETHON, INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), UNIVERSITE D'EVRY VAL D'ESSONNE
    Inventors: Ana Maria Buj Bello, Mirella Lo Scrudato
  • SYSTEMIC GENE REPLACEMENT THERAPY FOR TREATMENT OF X-LINKED MYOTUBULAR MYOPATHY (XLMTM)
    Publication number: 20210162020
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Application
    Filed: July 22, 2020
    Publication date: June 3, 2021
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • ANTISENSE TARGETING DYNAMIN 2 AND USE FOR THE TREATMENT OF CENTRONUCLEAR MYOPATHIES AND NEUROPATHIES
    Publication number: 20210095291
    Abstract: The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).
    Type: Application
    Filed: April 10, 2018
    Publication date: April 1, 2021
    Inventor: Ana Maria BUJ BELLO
  • Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
    Patent number: 10736945
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Grant
    Filed: December 28, 2017
    Date of Patent: August 11, 2020
    Assignees: Wake Forest University Health Sciences, Genethon, Children's Medical Center Corporation
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • COMPOSITIONS AND METHODS FOR THE TREATMENT OF MYOTONIC DYSTROPHY
    Publication number: 20200140867
    Abstract: The present invention relates to compositions and methods for the treatment of myotonic dystrophy.
    Type: Application
    Filed: October 27, 2017
    Publication date: May 7, 2020
    Inventors: ANA MARIA BUJ BELLO, MIRELLA LO SCRUDATO
  • COMPOSITIONS AND METHODS FOR THE TREATMENT OF MYOTONIC DYSTROPHY
    Publication number: 20190256868
    Abstract: The present invention relates to compositions and methods for the treatment of myotonic dystrophy.
    Type: Application
    Filed: October 27, 2017
    Publication date: August 22, 2019
    Inventors: ANA MARIA BUJ BELLO, MIRELLA LO SCRUDATO
  • SYSTEMIC GENE REPLACEMENT THERAPY FOR TREATMENT OF X-LINKED MYOTUBULAR MYOPATHY (XLMTM)
    Publication number: 20180369343
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Application
    Filed: December 28, 2017
    Publication date: December 27, 2018
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • SELECTIVE GENE THERAPY EXPRESSION SYSTEM
    Publication number: 20180256752
    Abstract: The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
    Type: Application
    Filed: May 24, 2018
    Publication date: September 13, 2018
    Inventors: Ana Maria Buj Bello, Isabelle Richard
  • Selective gene therapy expression system
    Patent number: 9981049
    Abstract: The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
    Type: Grant
    Filed: April 10, 2014
    Date of Patent: May 29, 2018
    Assignees: GENETHON, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
    Inventors: Ana Maria Buj Bello, Isabelle Richard
  • COMPOSITIONS AND METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS
    Publication number: 20180100144
    Abstract: The present invention relates to compositions and methods for the treatment of nucleotide repeat expansion disorders such as myotonic dystrophy.
    Type: Application
    Filed: April 27, 2016
    Publication date: April 12, 2018
    Inventors: Ana Maria BUJ BELLO, Mirella LO SCRUDATO
  • Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
    Patent number: 9895426
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Grant
    Filed: December 20, 2016
    Date of Patent: February 20, 2018
    Assignees: Wake Forest University Health Sciences, Genethon, Children's Medical Center Corporation
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
    Patent number: 9839677
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Grant
    Filed: July 26, 2016
    Date of Patent: December 12, 2017
    Assignees: Wake Forest University Health Sciences, Children's Medical Center Corporation, Genethon
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • SYSTEMIC GENE REPLACEMENT THERAPY FOR TREATMENT OF X-LINKED MYOTUBULAR MYOPATHY (XLMTM)
    Publication number: 20170112905
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Application
    Filed: December 20, 2016
    Publication date: April 27, 2017
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • SYSTEMIC GENE REPLACEMENT THERAPY FOR TREATMENT OF X-LINKED MYOTUBULAR MYOPATHY (XLMTM)
    Publication number: 20170095540
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Application
    Filed: July 26, 2016
    Publication date: April 6, 2017
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • METHOD OF TREATING PERIPHERAL NEUROPATHIES AND MOTOR NEURON DISEASES
    Publication number: 20170071972
    Abstract: A composition comprising a molecule for use in the delivery of the molecule to the peripheral nervous system (PNS) and/or to the central nervous system (CNS), wherein the composition is administered by regional infusion.
    Type: Application
    Filed: April 20, 2015
    Publication date: March 16, 2017
    Inventors: Ana Maria Buj Bello, Martin K. Childers
  • Systemic gene replacement therapy for treatment of X-linked MyoTubular Myopathy (XLMTM)
    Patent number: 9415120
    Abstract: The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
    Type: Grant
    Filed: January 13, 2015
    Date of Patent: August 16, 2016
    Assignees: Wake Forest University Health Sciences, Genethon, Children's Medical Center Corporation
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello