公开(公告)号：US11099193B2

公开(公告)日：2021-08-24

申请号：US15110868

申请日：2015-01-09

Applicant: GENETHON

Inventor： Jeremy Rouillon ,  Fedor Svinartchouk ,  Jerome Poupiot ,  Isabelle Richard

IPC: G01N31/00 ,  G01N33/53 ,  G01N33/68

Abstract: The invention relates to a method for prognosing, diagnosing, determining the risk, and monitoring the evolution of a muscular dystrophy. It also relates to a method for evaluating the efficacy of a treatment of a muscular dystrophy in a subject in need thereof.

公开(公告)号：US20210246469A1

公开(公告)日：2021-08-12

申请号：US17224367

申请日：2021-04-07

Applicant: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  GENETHON ,  SORBONNE UNIVERSITÉ ,  UNIVERSITÉ DE PARIS ,  SPARK THERAPEUTICS, INC.

Inventor： Sébastien LACROIX-DESMAZES ,  Federico MINGOZZI ,  Jordan DIMITROV ,  Christian LEBORGNE ,  Sean ARMOUR

IPC: C12N15/86 ,  A61K38/48

Abstract: Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering a protease that cleaves peptide bonds present in immunoglobulins or by administering a glycosidase that cleaves carbohydrate residues present on immunoglobulins, or other similar enzymatic cleavage of immunoglobulins in vivo. Also disclosed are methods for utilizing IdeS and other immunoglobulin G-degrading enzyme polypeptides for gene therapy treatment of a disease in a patient in need thereof.

公开(公告)号：US20210107948A1

公开(公告)日：2021-04-15

申请号：US16981086

申请日：2019-04-04

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 ,  Frederico MINGOZZI ,  Giuseppe RONZITTI ,  Patrice VIDAL

IPC: C07K14/005 ,  C12N7/00 ,  C12N15/86

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：US20210095291A1

公开(公告)日：2021-04-01

申请号：US16604121

申请日：2018-04-10

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

Inventor： Ana Maria BUJ BELLO

IPC: C12N15/113 ,  A61K31/712 ,  A61P25/02 ,  A61K38/46

Abstract: The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).

公开(公告)号：US20200370069A1

公开(公告)日：2020-11-26

申请号：US16629339

申请日：2018-07-06

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

Inventor： ANA BUJ BELLO ,  MARTINA MARINELLO ,  SAMIA MARTIN

IPC: C12N15/86 ,  A61P25/00 ,  C07K14/47 ,  A61K38/17 ,  A61K48/00

Abstract: The present invention relates to a recombinant adeno-associated virus (rAAV) vector comprising a serotype 9 or rh10 AAV capsid, for use in a method for the treatment of spinal muscular atrophy (SMA).

公开(公告)号：US10786546B2

公开(公告)日：2020-09-29

申请号：US16185248

申请日：2018-11-09

Applicant: GENETHON ,  ROYAL HOLLOWAY AND BEDFORD NEW COLLEGE

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

IPC: A61K38/17 ,  A61K48/00 ,  C12N15/86 ,  C12N7/00

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：US10471132B2

公开(公告)日：2019-11-12

申请号：US15303834

申请日：2015-04-27

Applicant: GENETHON ,  INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY

Inventor： Federico Mingozzi ,  Giuseppe Ronzitti ,  Fanny Collaud ,  Andrés Muro ,  Giulia Bortolussi

IPC: A61K38/45 ,  C12N15/86 ,  A61K48/00 ,  C12N9/10 ,  C12N7/00

Abstract: The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.

公开(公告)号：US20190153474A1

公开(公告)日：2019-05-23

申请号：US16214214

申请日：2018-12-10

Applicant: GENETHON ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE

Inventor： Martine Barkats

IPC: C12N15/86 ,  A61K48/00

Abstract: The present invention relates to compositions and methods for the delivery of therapeutic proteins to the CNS using recombinant AAV vectors. More specifically, the invention relates to compositions and methods for delivering proteins into the cerebrospinal fluid of mammalian subjects through peripheral administration of AAV vectors. The invention may be used to treat various disorders of the central nervous system, including degenerative diseases and motor neuron diseases.

公开(公告)号：US20180362980A1

公开(公告)日：2018-12-20

申请号：US16060396

申请日：2016-12-09

Applicant: GENETHON

Inventor： France PIETRI-ROUXEL ,  Virginie FRANCOIS

IPC: C12N15/113 ,  A61K48/00 ,  A61P21/00 ,  C12N15/62

Abstract: The invention relates to a recombinant adeno-associated viral vector (rAAV) comprising a sequence encoding an antisense oligonucleotide (AON) directed against a segment of at least 33 bases from the +30 to +69 region of exon 53 of the pre-messenger RNA (pre-mRNA) of dystrophin, advantageously of human origin, and to the use thereof as a drug, in particular for the treatment of Duchenne muscular dystrophy (DMD).

公开(公告)号：US10125352B2

公开(公告)日：2018-11-13

申请号：US15022049

申请日：2014-09-12

Applicant: GENETHON

Inventor： David Fenard

IPC: A61K39/00 ,  C12N7/00 ,  C07K14/005 ,  A61K39/12 ,  A01N63/00 ,  C12N15/86

Abstract: The invention relates to a process for producing enveloped viruses in a mildly acid medium. The processes of the invention are useful for producing and recovering at a large scale enveloped viruses under conditions observing good manufacturing practice (GMP).