公开(公告)号：US20180318447A1

公开(公告)日：2018-11-08

申请号：US15772902

申请日：2016-11-30

Applicant: GENETHON ,  INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  UNIVERSITE D'EVRY VAL D'ESSONNE

Inventor： Saliha MAJDOUL ,  David FENARD

IPC: A61K48/00 ,  C07K14/47 ,  C12N15/87

CPC classification number: A61K48/0016 ,  C07K14/47 ,  C07K14/82 ,  C07K2319/033 ,  C07K2319/10 ,  C12N15/86 ,  C12N15/87 ,  C12N2740/15043 ,  C12N2750/14143

Abstract: The present invention relates to peptides and compositions for use in improving transduction efficiency of viruses into target cells.

公开(公告)号：US20170112905A1

公开(公告)日：2017-04-27

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: A61K38/46 ,  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：US20170095540A1

公开(公告)日：2017-04-06

申请号：US15220139

申请日：2016-07-26

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 ,  A61K48/00

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：US20150252335A1

公开(公告)日：2015-09-10

申请号：US14670459

申请日：2015-03-27

Applicant: Genethon

Inventor： OTTO-WILHELM MERTEN ,  MARTIN MAREK ,  MONIQUE VAN OERS

IPC: C12N7/00 ,  C12N15/86

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.

Abstract translation: 本发明涉及生产实施基于杆状病毒的系统的生物药物的方法。 这些方法有利地允许生产具有减少数量的或不含有污染性杆状病毒病毒粒子的生物药物。

公开(公告)号：US20150241452A1

公开(公告)日：2015-08-27

申请号：US14431030

申请日：2013-09-26

Applicant: GENETHON

Inventor： Jérémy Rouillon ,  Fedor Svinartchouk

IPC: G01N33/68 ,  H01J49/16 ,  G01N27/447

CPC classification number: G01N33/6893 ,  G01N27/447 ,  G01N33/6887 ,  G01N2333/4712 ,  G01N2800/10 ,  G01N2800/2878 ,  G01N2800/52 ,  H01J49/164

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.

Abstract translation: 本发明涉及一种诊断，预后和治疗性监测肌营养不良症的方法，通过检测患者体液中的滴度或一种或多种滴度碎片。

公开(公告)号：US20150141361A1

公开(公告)日：2015-05-21

申请号：US14407211

申请日：2013-07-16

Applicant: GENETHON

Inventor： Isabelle Richard

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

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: 一种药物组合物，其包含至少一种表观遗传调节蛋白修饰化合物，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。

公开(公告)号：US20250041448A1

公开(公告)日：2025-02-06

申请号：US17603355

申请日：2020-04-20

Applicant: Genethon ,  Institut National de la Sante et de la Recherche Medicale ,  Universite d'Evry Val d'Essonne

Inventor： Giuseppe RONZITTI ,  Louisa JAUZE ,  Séverine CHARLES ,  Federico MINGOZZI

IPC: A61K48/00 ,  A61K38/18 ,  A61P7/00 ,  C12N15/86

Abstract: The invention relates to a nucleic acid construct for gene therapy of FGF-23 related hypophosphatemic diseases, in particular gene therapy directed to muscle, liver or hematopoietic tissue, more particularly liver tissue. The invention relates also to a vector comprising the nucleic acid construct, and their use for the treatment of FGF-23 related hypophosphatemic diseases, in particular XLH, by gene therapy.

公开(公告)号：US12098355B2

公开(公告)日：2024-09-24

申请号：US16627013

申请日：2018-06-29

Applicant: UNIVERSITE DE PARIS ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) ,  GENETHON

Inventor： Florence Gazeau ,  Amanda Karine Andriola Silva ,  Otto-Wilhelm Merten ,  Claire Wilhelm ,  Max Piffoux

IPC: C12M3/06 ,  C12M1/00 ,  C12M1/12

CPC classification number: C12M27/14 ,  C12M25/16 ,  C12M29/04

Abstract: A fluidic system for producing extracellular vesicles from producer cells, including at least one container, a liquid medium contained by the container and producer cells, characterised in that it also includes microcarriers suspended in the liquid medium, the majority of producer cells being adherent to the surface of the microcarriers, and a liquid medium agitator, the agitator and the dimensions of the container being adapted to control a turbulent flow of the liquid medium in the container.

公开(公告)号：US11819478B2

公开(公告)日：2023-11-21

申请号：US15988728

申请日：2018-05-24

Applicant: GENETHON ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE

Inventor： Ana Maria Buj Bello ,  Isabelle Richard

IPC: A61K48/00 ,  C12N9/16 ,  C12N9/64 ,  A61K38/46 ,  A61K38/48 ,  C12N15/86 ,  C07K14/47

CPC classification number: A61K48/0066 ,  A61K38/465 ,  A61K38/4873 ,  A61K48/0058 ,  A61K48/0075 ,  C12N9/16 ,  C12N9/6472 ,  C12N15/86 ,  C07K14/4707 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2830/008 ,  C12Y301/03048 ,  C12Y304/22054

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.

公开(公告)号：US11767538B2

公开(公告)日：2023-09-26

申请号：US16536503

申请日：2019-08-09

Applicant: GENETHON ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE

Inventor： Martine Barkats

IPC: C12N15/86 ,  A61K48/00

CPC classification number: C12N15/86 ,  A61K48/00 ,  C12N2750/14143 ,  C12N2830/008

Abstract: The present invention relates to compositions and methods, in particular to methods based on systemic injection of rAAV, for delivering genes to cells of the central nervous system in mammals, such as brain neurons or glial cells, and in particular to motor neurons or glial cells of the spinal cord The invention also relates to methods of treating motor neuron disorders in mammals by expression of therapeutic genes. The invention stems from the unexpected discovery that peripheral injection of AAV vectors leads to a bypass of the blood brain barrier and a massive infection of motor neurons. The invention may be used in any mammal, including human subjects.