公开(公告)号：US11298348B2

公开(公告)日：2022-04-12

申请号：US16960503

申请日：2019-01-10

Applicant: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) ,  UNIVERSITE D'EVRY-VAL-D'ESSONNE ,  GENETHON ,  UNIVERSITA 'DEGLI STUDI DI PADOVA

Inventor： Isabelle Richard ,  Dorianna Sandona

IPC: A61K31/47 ,  A61P21/00 ,  A61K31/427

Abstract: Sarcoglycanopathies are autosomal recessive diseases caused by mutations in the one of the genes coding for any sarcoglycans (SG). The inventors previously showed that the application of small molecules developed to rescue ΔF508-CFTR trafficking, and known as CFTR correctors, improved the maturation of several α-sarcoglycan mutants that were consequently rescued at the plasma membrane (WO 014086687). Now, the inventors show that some specific CFTR correctors provide additive and even synergic effect when administered in combination.

公开(公告)号：US20200289673A1

公开(公告)日：2020-09-17

申请号：US16491623

申请日：2018-03-09

Applicant: GENETHON ,  UNIVERSITE D'EVRY VAL D'ESSONNE ,  INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE ,  SORBONNE UNIVERSITÉ

Inventor： FEDERICO MINGOZZI ,  GIUSEPPE RONZITTI ,  PATRICE VIDAL

IPC: A61K48/00 ,  A61P3/08 ,  C12N7/00 ,  C12N9/16

Abstract: The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.

公开(公告)号：US20200261589A1

公开(公告)日：2020-08-20

申请号：US16757591

申请日：2018-10-19

Applicant: Genethon ,  Institut National de la Santé et de la Recherche Médicale ,  Université d'Evry val d'Essonne

Inventor： Anne Galy ,  Maxime Ferrand

IPC: A61K47/64 ,  A61K9/127 ,  A61K45/06 ,  A61K9/00 ,  A61K35/76 ,  A61K47/69 ,  A61P21/00

Abstract: 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.

公开(公告)号：US20190211360A1

公开(公告)日：2019-07-11

申请号：US16356005

申请日：2019-03-18

Applicant: GENETHON

Inventor： NICOLAS MARCEAU ,  MEHDI GASMI

IPC: C12N15/86 ,  C12N7/00 ,  C12N7/02

CPC classification number: C12N15/86 ,  C12N7/00 ,  C12N7/02 ,  C12N2740/10051 ,  C12N2740/15011 ,  C12N2740/16051

Abstract: The present invention relates to the industrialization of the production of recombinant lentiviral vectors in order to manufacture sufficient materials for therapeutic applications such as gene therapy and/or DNA vaccination, for use in clinical trials and/or commercial use.

公开(公告)号：US20180369343A1

公开(公告)日：2018-12-27

申请号：US15856653

申请日：2017-12-28

Applicant: Wake Forest University Health Sciences ,  Genethon ,  Children's Medical Center Corporation

Inventor： Martin K. Childers ,  Alan H. Beggs ,  Ana Maria Buj Bello

IPC: A61K38/46 ,  C12N9/16 ,  A61K48/00 ,  C12N7/00

CPC classification number: A61K38/465 ,  A61K48/005 ,  A61K48/0058 ,  C12N7/00 ,  C12N9/16 ,  C12N2750/14143 ,  C12Y301/03 ,  C12Y301/03064

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.

公开(公告)号：US20180305717A1

公开(公告)日：2018-10-25

申请号：US16029870

申请日：2018-07-09

Applicant: GENETHON

Inventor： LIONEL GALIBERT ,  OTTO-WILHELM MERTEN ,  AURELIEN JACOB

IPC: C12N15/86 ,  C12N7/00

CPC classification number: C12N15/86 ,  C12N7/00 ,  C12N2710/14044 ,  C12N2750/14043 ,  C12N2750/14141 ,  C12N2750/14151 ,  C12N2800/50 ,  C12N2830/20 ,  C12N2999/007

Abstract: The invention relates to a recombinant baculoviral genome useful for the production of viral vectors for gene therapy, allowing said production from a single infection

公开(公告)号：US09895426B2

公开(公告)日：2018-02-20

申请号：US15384976

申请日：2016-12-20

Applicant: Wake Forest University Health Sciences ,  Genethon ,  Children's Medical Center Corporation

Inventor： Martin K. Childers ,  Alan H. Beggs ,  Ana Maria Buj Bello

IPC: A61K48/00 ,  A61K38/46 ,  C12N7/00 ,  C07H21/00 ,  C07H21/04 ,  C12N15/00 ,  C12N15/85 ,  C12P21/06

CPC classification number: A61K38/465 ,  A61K48/005 ,  A61K48/0058 ,  C12N7/00 ,  C12N9/16 ,  C12N2750/14143 ,  C12Y301/03 ,  C12Y301/03064

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.

公开(公告)号：US09862934B2

公开(公告)日：2018-01-09

申请号：US14670459

申请日：2015-03-27

Applicant: GENETHON

Inventor： Otto-Wilhelm Merten ,  Martin Marek ,  Monique Van Oers

IPC: C12N15/866 ,  C12N15/86 ,  C12N7/00

CPC classification number: C12N7/00 ,  C12N15/86 ,  C12N2710/14143 ,  C12N2710/14152 ,  C12N2750/14143 ,  C12N2800/50

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.

公开(公告)号：US20170157213A1

公开(公告)日：2017-06-08

申请号：US15321416

申请日：2015-04-24

Applicant: GENETHON ,  ROYAL HOLLOWAY AND BEDFORD NEW COLLEGE

Inventor： George Dickson ,  Thomas Voit ,  Philippe Moullier ,  Caroline Le Guiner

IPC: A61K38/17 ,  C12N7/00 ,  A61K48/00

CPC classification number: A61K38/1719 ,  A61K48/005 ,  A61K48/0058 ,  A61K48/0075 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2840/007

Abstract: A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein:—the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin;—the composition is systemically administered.

公开(公告)号：US09526742B2

公开(公告)日：2016-12-27

申请号：US14407211

申请日：2013-07-16

Applicant: GENETHON

Inventor： Isabelle Richard

IPC: A61K31/706 ,  A61K31/167 ,  G01N33/50 ,  A61K31/7068

CPC classification number: A61K31/706 ,  A61K31/167 ,  A61K31/7068 ,  G01N33/5061 ,  G01N2440/10 ,  G01N2440/12 ,  G01N2500/10 ,  G01N2800/2878

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.

Abstract translation: 一种包含至少一种表观遗传调节蛋白修饰化合物的药物组合物，用于治疗与至少一种蛋白质的构象紊乱相关的遗传性肌肉疾病，所述疾病导致蛋白质的细胞降解。