Patents Assigned to Université d'Evry val d'Essonne
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Publication number: 20210198636Abstract: The invention relates to a pharmaceutical composition for targeting drug delivery including gene delivery to lung tissue, comprising at least a therapeutic drug or gene associated to a syncytin protein, and its use in the prevention and/or treatment of lung diseases, in particular in gene therapy of said diseases using lentiviral vector particles or lentivirus-like particles pseudotyped with syncytin protein.Type: ApplicationFiled: October 19, 2018Publication date: July 1, 2021Applicants: Genethon, Institut National de la Santé et de la Recherche Médicale, Université d'Evry val d'EssonneInventors: Anne Galy, Youna Coquin, Maxime Ferrand
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Publication number: 20210107948Abstract: The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.Type: ApplicationFiled: April 4, 2019Publication date: April 15, 2021Applicants: Genethon, INSERM (Institut National de la Santé et de la Recherche Médicale), Universite d'Evry Val d'Essonne, Sorbonne Universite, Association Institut de MyologieInventors: Isabelle RICHARD, Evelyne GICQUEL, Frederico MINGOZZI, Giuseppe RONZITTI, Patrice VIDAL
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Publication number: 20200261589Abstract: The invention relates to a pharmaceutical composition for targeting drug delivery including gene delivery to regenerating muscle tissue, comprising at least a therapeutic drug or gene, associated to a syncytin protein, and its use in the prevention and/or treatment of muscle injuries or diseases, in particular in gene therapy of said diseases using lentiviral vector particles or lentivirus-like particles pseudotyped with syncytin protein.Type: ApplicationFiled: October 19, 2018Publication date: August 20, 2020Applicants: Genethon, Institut National de la Santé et de la Recherche Médicale, Université d'Evry val d'EssonneInventors: Anne Galy, Maxime Ferrand
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Patent number: 10654838Abstract: A compound for use in the treatment and/or prevention of diseases or disorders wherein an inhibition of protein prenylation is required, which is alternatively chosen among wherein R1 is 2-pyridyl, 3-pyridyl or 4-pyridyl; R2 represents: and wherein R5 and R6 represent a hydrogen atom or a (C1-4)alkyl group; and R7 represents an arylcarbonyl group, a heteroarylcarbonyl group, a heteroarylacetyl group, a (C1-C4)alkoxy-carbonylmethyl group, a group. Also disclosed are novel compounds of formulae (I) and (II).Type: GrantFiled: October 7, 2015Date of Patent: May 19, 2020Assignees: INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM), ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES, UNIVERSITE D'EVRY VAL D'ESSONNEInventors: Marc Peschanski, Sophie Blondel, Xavier Nissan
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Publication number: 20180016579Abstract: 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: ApplicationFiled: August 31, 2017Publication date: January 18, 2018Applicants: 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 PARISInventors: Jean-Michel ROZET, Antoine KICHLER, Isabelle PERRAULT, Josseline KAPLAN, Xavier GERARD, Daniel SCHERMAN, M. Arnold MUNNICH
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Publication number: 20170305892Abstract: A compound for use in the treatment and/or prevention of diseases or disorders wherein an inhibition of protein prenylation is required, which is alternatively chosen among wherein R1 is 2-pyridyl, 3-pyridyl or 4-pyridyl; R2 represents: and wherein R5 and R6 represent a hydrogen atom or a (C1-4)alkyl group; and R7 represents an arylcarbonyl group, a heteroarylcarbonyl group, a heteroarylacetyl group, a (C1-C4)alkoxy-carbonylmethyl group, a group. Also disclosed are novel compounds of formulae (I) and (II).Type: ApplicationFiled: October 7, 2015Publication date: October 26, 2017Applicants: INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM), ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES, UNIVERSITE D'EVRY VAL D'ESSONNEInventors: Marc PESCHANSKI, Sophie BLONDEL, Xavier NISSAN
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Patent number: 9777272Abstract: 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: GrantFiled: September 16, 2016Date of Patent: October 3, 2017Assignees: 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 PARISInventors: Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich
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Publication number: 20170044533Abstract: 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: ApplicationFiled: September 16, 2016Publication date: February 16, 2017Applicants: 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 PARISInventors: Jean-Michel ROZET, Antoine KICHLER, Isabelle PERRAULT, Josseline KAPLAN, Xavier GERARD, Daniel SCHERMAN, M. Arnold MUNNICH
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Patent number: 9487782Abstract: 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: GrantFiled: November 18, 2014Date of Patent: November 8, 2016Assignees: 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 PARISInventors: Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich
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Publication number: 20150111951Abstract: 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: ApplicationFiled: November 18, 2014Publication date: April 23, 2015Applicants: 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 ParisInventors: Jean-Michel ROZET, Antoine KICHLER, Isabelle PERRAULT, Josseline KAPLAN, Xavier GERARD, Daniel SCHERMAN, M. Arnold MUNNICH
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Patent number: 9012425Abstract: 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: GrantFiled: June 8, 2012Date of Patent: April 21, 2015Assignees: 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 ParisInventors: Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich
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Patent number: 8658150Abstract: The invention relates to a static copolymer of linear polyethylenimine that comprises two monomer units of the following formulas I and II, in which R1 is a hydrogen atom or a C1-C6 alkyl radical, R2 is a hydrogen atom, a C1-C6 alkyl, aryl or aralkyl group, wherein the alkyl group is a C1-C6 one, n is a number between 1 and 99% of the total monomers, m is a number between 1 and 99% of the total monomers, and advantageously m+n=100%. The invention also relates to complexes of this copolymer with a nucleic acid, and to the use thereof in in vivo, in vitro or ex vivo gene transfection.Type: GrantFiled: March 4, 2009Date of Patent: February 25, 2014Assignees: Centre National de la Recherche Scientifique, Universite d'Orleans, Universite d'Evry val d'EssonneInventors: Hervé Cheradame, Mathieu Sassatelli, Philippe Guegan, Patrick Midoux, Chantal Pichon
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Publication number: 20130171172Abstract: A composition comprising a calpain-3 inhibitor for treating muscular dystrophies and cardiomyopathies, in particular tibial muscular dystrophy (TMD).Type: ApplicationFiled: June 30, 2011Publication date: July 4, 2013Applicants: Genethon, Universite D'Evry Val D'EssonneInventors: Isabelle Richard, Karine Charton
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Publication number: 20040009505Abstract: The invention is derived from the identification of mutations in the GJB6 gene, responsible for Clouston's syndrome. The symptomatology of said syndrome suggests that the GJB6 coding for connexin 30 (Cx-30), is most probably involved also in other types of alopecia with genetic susceptibility, in particular non-pathological. Therefore, the invention concerns the GJB6 gene sequence bearing at least one of the mutations 31 (G>A) and 263 (C>T), responsible for Clouston's syndrome, and the use of constructs comprising the GJB6 gene, both for preparing pharmaceutical compositions for treating Clouston's syndrome and/or certain disorders of the body hair system, and for screening molecules likely to have a beneficial effect in the treatment of alopecia. The invention also concerns methods for diagnosing Clouston's syndrome.Type: ApplicationFiled: March 27, 2003Publication date: January 15, 2004Applicant: Genethon III and Universite D'Evry Val D'EssonneInventors: Gilles Waksman, Jerome Lamartine