公开(公告)号：US20240317814A1

公开(公告)日：2024-09-26

申请号：US18735636

申请日：2024-06-06

Applicant: Genethon ,  INSERM (Institut National de la Santé et de la Recherche Médicale) ,  Universite d'Evry Val d'Essonne ,  Sorbonne Universite ,  Association Institut de Myologie

Inventor： Isabelle Richard ,  Evelyne Gicquel ,  Federico Mingozzi

IPC: C07K14/005 ,  A61K48/00 ,  A61P25/28 ,  C07K14/075 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

CPC classification number: C07K14/005 ,  A61P25/28 ,  C07K14/075 ,  C12N7/00 ,  C12N9/22 ,  C12N15/111 ,  C12N15/86 ,  A61K48/00 ,  C12N2310/20 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2750/14171

Abstract: 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.

公开(公告)号：US11993781B2

公开(公告)日：2024-05-28

申请号：US16095098

申请日：2017-04-21

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

Inventor： Anne Galy

IPC: C12N15/63 ,  A61K35/17 ,  A61K35/76 ,  C07H21/04 ,  C07K14/005 ,  C12N5/0781 ,  C12N15/85 ,  C12N15/86 ,  A61K48/00

CPC classification number: C12N15/86 ,  A61K35/17 ,  A61K35/76 ,  C07K14/005 ,  C12N5/0635 ,  A61K48/00 ,  C12N2740/10022 ,  C12N2740/16022 ,  C12N2740/16043 ,  C12N2740/16052

Abstract: The present invention relates to a method for obtaining stable pseudotyped lentiviral particles including a heterologous gene of interest, comprising the following steps: a) transfecting at least one plasmid in appropriate cell lines, wherein said at least one plasmid comprises the gene of interest, the rev. gag and pol genes, and a sequence coding for an ERV syncytin, wherein the rev, gag and pol genes are retroviral genes; b) incubating the transfected cells obtained in a), so that they produce the stable pseudotyped lentiviral particles in the supernatant; and c) harvesting and concentrating the stable lentiviral particles obtained in b). The present invention also relates to a method to transduce immune cells using lentiviral vectors pseudotyped with an ERV syncytin glycoprotein. The method can be performed on non-stimulated blood cells or on cells stimulated briefly with IL7, and the cells can be expanded. The stable pseudotyped lentiviral particles obtained are particularly useful in gene therapy.

公开(公告)号：US20230321277A1

公开(公告)日：2023-10-12

申请号：US18177446

申请日：2023-03-02

Applicant: GENETHON ,  UNIVERSITE D'EVRY-VAL-D'ESSONNE ,  Inserm (Institut National De La Sante Et De La Recherche Medicale)

Inventor： Isabelle RICHARD ,  Evelyne GICQUEL-ZOUIDA ,  William LOSTAL

IPC: A61K48/00 ,  A61P21/00 ,  C07K14/47 ,  C12N15/86

CPC classification number: A61K48/005 ,  A61P21/00 ,  C07K14/4707 ,  C12N15/86 ,  A61K38/00

Abstract: The present invention concerns synthetic polynucleotides encoding a human fukutin-related protein (FKRP) wherein the synthetic polynucleotides contain at least a mutation avoiding supplementary transcript(s) generated from frameshift start codon(s). The synthetic polynucleotides are useful, especially for treating a pathology linked to a FKRP deficiency or induced by a defect in α-dystroglycan (α-DG) glycosylation, such as LGMD2I.

公开(公告)号：US20230277540A1

公开(公告)日：2023-09-07

申请号：US18009034

申请日：2021-06-09

Applicant: Genethon ,  Universite d'Evry Val d'Essonne ,  INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE)

Inventor： Isabelle Richard ,  Ariane Biquand

IPC: A61K31/519 ,  A61K31/4439 ,  A61K31/7088 ,  A61P9/00

CPC classification number: A61K31/519 ,  A61K31/4439 ,  A61K31/7088 ,  A61P9/00

Abstract: The invention relates to the treatment of dilated cardiomyopathies, in particular genetic dilated cardiomyopathies, using non-expressible inhibitors of the WNT pathway or TGF-β pathway, alone or in combination.

公开(公告)号：US20230220391A1

公开(公告)日：2023-07-13

申请号：US18009018

申请日：2021-06-09

Applicant: Genethon ,  Universite d'Evry Val d'Essonne ,  INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE)

Inventor： Isabelle Richard ,  Ariane Biquand

IPC: C12N15/113 ,  A61P9/04 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61P9/04 ,  C12N15/86 ,  C12N2310/531 ,  C12N2310/14 ,  C12N2320/32 ,  C12N2750/14143 ,  C12N2750/14171

Abstract: The present disclosure relates to the treatment of dilated cardiomyopathies, in particular to 5 the use of an inhibitor of CILP-1.

公开(公告)号：US20230203534A1

公开(公告)日：2023-06-29

申请号：US17926709

申请日：2021-05-21

Applicant: GENETHON ,  INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) ,  UNIVERSITE D'EVRY VAL D'ESSONNE ,  UNIVERSITE CLAUDE BERNARD LYON 1

Inventor： Louisa JAUZE ,  Fabienne RAJAS ,  Giuseppe RONZITTI

IPC: C12N15/86 ,  C07K14/81 ,  C12N9/16

CPC classification number: C12N15/86 ,  C07K14/8125 ,  C12N9/16 ,  C12Y301/03009 ,  C12N2750/14143 ,  C12N2750/14171

Abstract: The invention relates to an adeno-associated virus (AAV) vector comprising a nucleic acid construct for the expression of a glucose-6-phosphatase-a (G6Pase-a) in a cell, the construct comprising a nucleic acid sequence encoding the G6Pase-a, wherein the nucleic acid sequence encoding the G6Pase-a is operably linked to a human alpha-1 antitrypsin (hAAT) promoter, a cell transformed with the vector of the invention, a composition comprising the vector or the cell of the invention, and the use thereof.

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：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：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.