Patents Assigned to Genethon
  • METHODS FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS
    Publication number: 20180016579
    Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.
    Type: Application
    Filed: August 31, 2017
    Publication date: January 18, 2018
    Applicants: INSERM (Institut National de la Sante et de la Rec herche Medicale), CNRS (Centre National de la Recherche Scientifique ), GENETHON, UNIVERSITE PARIS DESCARTES, ENSCP - Chimie ParisTech - Ecole Nationale Superie ure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
    Inventors: Jean-Michel ROZET, Antoine KICHLER, Isabelle PERRAULT, Josseline KAPLAN, Xavier GERARD, Daniel SCHERMAN, M. Arnold MUNNICH
  • Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
    Patent number: 9862934
    Abstract: The present invention relates to methods for the production of biopharmaceuticals implementing a baculovirus-based system. These methods advantageously allow the production of biopharmaceuticals with a reduced number of or without contaminating baculoviral virions.
    Type: Grant
    Filed: March 27, 2015
    Date of Patent: January 9, 2018
    Assignee: GENETHON
    Inventors: Otto-Wilhelm Merten, Martin Marek, Monique Van Oers
  • 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
  • Methods for the treatment of Leber congenital amaurosis
    Patent number: 9777272
    Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>G CEP290 mRNA.
    Type: Grant
    Filed: September 16, 2016
    Date of Patent: October 3, 2017
    Assignees: INSERM (Institut National de la Sante et de la Recherche Medicale), CNRS (Centre National de la Recherche Scientifique), GENETHON, UNIVERSITE PARIS DESCARTES, ENSCP—Chimie ParisTech—Ecole Nationale Superieure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
    Inventors: Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich
  • METHODS FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS
    Publication number: 20170044533
    Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.
    Type: Application
    Filed: September 16, 2016
    Publication date: February 16, 2017
    Applicants: INSERM (Institut National de la Sante et de la Recherche Medicale), CNRS (Centre National de la Recherche Scientifique), GENETHON, UNIVERSITE PARIS DESCARTES, ENSCP - Chimie ParisTech - Ecole Nationale Superieure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
    Inventors: Jean-Michel ROZET, Antoine KICHLER, Isabelle PERRAULT, Josseline KAPLAN, Xavier GERARD, Daniel SCHERMAN, M. Arnold MUNNICH
  • Methods of increasing efficiency of vector penetration of target tissue
    Patent number: 9546112
    Abstract: Methods for increasing the efficiency of target tissue penetration of an adeno-associated virus (AAV) vector in a patient are provided. In some aspects, the methods involve inhibiting the interaction of the serum protein galectin 3 binding protein (G3BP) with AAV vector. Further provided are methods for reducing tissue distribution of a virus or for neutralizing a virus harbored by an organ destined for transplant, or newly transplanted, by administering a composition comprising G3BP.
    Type: Grant
    Filed: March 22, 2011
    Date of Patent: January 17, 2017
    Assignees: ASSOCIATION INSTITUT DE MYOLOGIE, GÉNÉTHON, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, UNIVERSITÉ PIERRE ET MARIE CURIE (PARIS 6), INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE
    Inventors: Thomas Voit, Luis Garcia, Jérôme Denard, Fedor Svinartchouk
  • Use of epigenome-modifying compounds for the treatment of genetic muscular diseases linked to a protein-conformational disorder
    Patent number: 9526742
    Abstract: A pharmaceutical composition including at least one epigenome-modifying compound, for a use thereof in the treatment of genetic muscular diseases linked to a conformational disorder of at least one protein, said disorder causing the cellular degradation of the protein.
    Type: Grant
    Filed: July 16, 2013
    Date of Patent: December 27, 2016
    Assignee: GENETHON
    Inventor: Isabelle Richard
  • Methods for the treatment of leber congenital amaurosis
    Patent number: 9487782
    Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>G CEP290 mRNA.
    Type: Grant
    Filed: November 18, 2014
    Date of Patent: November 8, 2016
    Assignees: INSERM (Institut National de la Sante et de la Recherche Medicale), CNRS (Centre National de la Recherche Scientifique), GENETHON, UNIVERSITE PARIS DESCARTES, ENSCP—Chimie ParisTech—Ecole Nationale Superieure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
    Inventors: Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich
  • 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
  • METHOD FOR PRODUCING ENVELOPED VIRUSES
    Publication number: 20160230147
    Abstract: The invention relates to a process for producing enveloped viruses in a mildly acid medium. The processes of the invention are useful for producing and recovering at a large scale enveloped viruses under conditions observing good manufacturing practices (or GMP).
    Type: Application
    Filed: September 12, 2014
    Publication date: August 11, 2016
    Applicant: GENETHON
    Inventor: DAVID FENARD
  • METHOD FOR DIAGNOSING MUSCULAR DYSTROPHY
    Publication number: 20150241452
    Abstract: The invention relates to a method for the diagnosis, prognosis and therapeutic monitoring of muscular dystrophy, by detecting titin or one or more fragments of titin in a bodily fluid of a patient.
    Type: Application
    Filed: September 26, 2013
    Publication date: August 27, 2015
    Applicant: GENETHON
    Inventors: Jérémy Rouillon, Fedor Svinartchouk
  • Widespread gene delivery to the retina using systemic administration of AAV vectors
    Patent number: 9114161
    Abstract: The present invention relates to compositions and methods, in particular to methods based on systemic administration of scAAV, for delivering genes to cells of the retina of mammals, and in particular to photoreceptor cells, ganglion cells, glial cells, inner nuclear layer cells or cells of the retinal pigmented epithelium.
    Type: Grant
    Filed: July 30, 2010
    Date of Patent: August 25, 2015
    Assignees: ASSOCIATION INSTITUT DE MYOLOGIE, GENETHON, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6)
    Inventor: Martine Barkats
  • USE OF EPIGENOME-MODIFYING COMPOUNDS FOR THE TREATMENT OF GENETIC MUSCULAR DISEASES LINKED TO A PROTEIN-CONFORMATIONAL DISORDER
    Publication number: 20150141361
    Abstract: A pharmaceutical composition including at least one epigenome-modifying compound, for a use thereof in the treatment of genetic muscular diseases linked to a conformational disorder of at least one protein, said disorder causing the cellular degradation of the protein.
    Type: Application
    Filed: July 16, 2013
    Publication date: May 21, 2015
    Applicant: GENETHON
    Inventor: Isabelle Richard
  • METHODS FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS
    Publication number: 20150111951
    Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.
    Type: Application
    Filed: November 18, 2014
    Publication date: April 23, 2015
    Applicants: INSERM (Institut National de la Sante et de la Recherche Medicale), CNRS (Centre National de la Recherche Scientifique), Genethon, Universite Paris Descartes, ENSCP - Chimie ParisTech - Ecole Nationale Superieure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, Assistance Publique Hopitaux De Paris
    Inventors: Jean-Michel ROZET, Antoine KICHLER, Isabelle PERRAULT, Josseline KAPLAN, Xavier GERARD, Daniel SCHERMAN, M. Arnold MUNNICH
  • Methods for the treatment of Leber congenital amaurosis
    Patent number: 9012425
    Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>G CEP290 mRNA.
    Type: Grant
    Filed: June 8, 2012
    Date of Patent: April 21, 2015
    Assignees: INSERM (Institute National de la Sante et de la Recherche Medicale), CNRS (Centre National de la Recherche Scientifique), Genethon, Universite Paris Descartes, ENSCP—Chimie Paris Tech—Ecole National Superieure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, Assistance Publique—Hopitaux de Paris
    Inventors: Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich
  • Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
    Patent number: 8993317
    Abstract: The present invention relates to methods for the production of biopharmaceuticals implementing a baculovirus-based system. These methods advantageously allow the production of biopharmaceuticals with reduced or no contaminating baculoviral virions.
    Type: Grant
    Filed: August 5, 2010
    Date of Patent: March 31, 2015
    Assignee: Genethon
    Inventors: Otto-Wilhelm Merten, Martin Marek, Monique Van Oers
  • Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
    Patent number: 8957044
    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: February 28, 2014
    Date of Patent: February 17, 2015
    Assignees: Wake Forest University Health Sciences, Genethon, Children's Medical Center Corporation
    Inventors: Martin K. Childers, Alan H. Beggs, Ana Maria Buj Bello
  • BACULOVIRUS EXPRESSION SYSTEMS
    Publication number: 20140356904
    Abstract: The present invention relates to an optimized baculovirus construct useful for the production of virus (-like) particles or viral vectors, in particular viral vectors for gene therapy.
    Type: Application
    Filed: July 27, 2012
    Publication date: December 4, 2014
    Applicant: Genethon
    Inventors: Lionel Galibert, Otto-Wilhelm Merten, Monique Van Oers, Christel Riviere
  • BACULOVIRUS SYSTEM FOR THE EXPRESSION OF A GENE THERAPY VECTOR
    Publication number: 20140349374
    Abstract: The invention concerns a recombinant baculovirus genome useful for the expression of gene therapy vectors by means of a single infection.
    Type: Application
    Filed: July 27, 2012
    Publication date: November 27, 2014
    Applicant: Genethon
    Inventors: Lionel Galibert, Otto-Wilhelm Merten, Aurélien Jacob
  • PEPTIDES WITH VIRAL INFECTION ENHANCING PROPERTIES AND THEIR USE
    Publication number: 20140255349
    Abstract: The invention relates to peptides and functional derivatives thereof and their use for improving transduction efficiency of viruses into target cells.
    Type: Application
    Filed: June 28, 2012
    Publication date: September 11, 2014
    Applicants: GENETHON, Institut National de la Sainte et de la recherche Madicale, Centre National de la Recherche Scientique
    Inventors: David Fenard, Antoine Kichler, Samia Martin