公开(公告)号：US20230190965A1

公开(公告)日：2023-06-22

申请号：US18049296

申请日：2022-10-25

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

CPC classification number: A61K48/0066 ,  A61K48/0033 ,  A61P3/08 ,  C12N7/00 ,  C12N9/16 ,  C12Y301/03001 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2750/14145

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

公开(公告)号：US20230173102A1

公开(公告)日：2023-06-08

申请号：US17921727

申请日：2021-04-28

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

Inventor： Ana Buj Bello ,  Edith Renaud-Gabardos ,  Dirk Grimm ,  Jonas Weinmann

IPC: A61K48/00 ,  A61P21/00

CPC classification number: A61K48/0058 ,  A61K48/0075 ,  A61P21/00

Abstract: The invention relates to the use of a recombinant porcine adeno-associated virus (AAV) vector comprising a peptide-modified porcine AAV serotype 1 (AAVpol) capsid in gene therapy of muscle and/or central nervous system (CNS) disorders, in particular neuromuscular diseases such as genetic neuromuscular diseases.

公开(公告)号：US11564977B2

公开(公告)日：2023-01-31

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

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：US11541131B2

公开(公告)日：2023-01-03

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 ,  C07H21/04 ,  C12N15/00 ,  C12N15/63 ,  C12N7/00 ,  A61P3/08 ,  C12N9/16

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

公开(公告)号：US20220235371A1

公开(公告)日：2022-07-28

申请号：US17464727

申请日：2021-09-02

Applicant: GENETHON

Inventor： NICOLAS MARCEAU ,  MEHDI GASMI

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

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：US11371061B2

公开(公告)日：2022-06-28

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: C12N15/87 ,  C12N15/86 ,  C07K14/82 ,  A61K48/00 ,  C07K14/47

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：US20210198636A1

公开(公告)日：2021-07-01

申请号：US16757590

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 ,  Youna Coquin ,  Maxime Ferrand

IPC: C12N7/04 ,  A61P11/00 ,  C12N15/113 ,  A61K48/00

Abstract: 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.

公开(公告)号：US20200010852A1

公开(公告)日：2020-01-09

申请号：US16536503

申请日：2019-08-09

Applicant: GENETHON ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE

Inventor： Martine BARKATS

IPC: C12N15/86

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.

公开(公告)号：US20190390184A1

公开(公告)日：2019-12-26

申请号：US16332373

申请日：2017-09-12

Applicant: GENETHON ,  SORBONNE UNIVERSITE ,  DUKE UNIVERSITY

Inventor： FEDERICO MINGOZZI ,  GIUSEPPE RONZITTI ,  DWIGHT D. KOEBERL ,  SANG-OH HAN

IPC: C12N9/26 ,  C12N15/86 ,  A61K38/47

Abstract: The present invention relates to variants of acid-alpha glucosidase and uses thereof. Said variants are sequence-optimized and/or are linked to a heterogenous signal peptide.

公开(公告)号：US20190256868A1

公开(公告)日：2019-08-22

申请号：US16345742

申请日：2017-10-27

Applicant: GENETHON ,  INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)

Inventor： ANA MARIA BUJ BELLO ,  MIRELLA LO SCRUDATO

IPC: C12N15/90 ,  A61P21/00 ,  C12N15/113 ,  C12N9/22 ,  C12N15/86 ,  A61K9/00 ,  A61K35/12

Abstract: The present invention relates to compositions and methods for the treatment of myotonic dystrophy.