公开(公告)号：US20210292724A1

公开(公告)日：2021-09-23

申请号：US17265528

申请日：2019-08-06

Applicant: GENETHON ,  INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE ,  SORBONNE UNIVERSITÉ ,  UNIVERSITE D'EVRY VAL D'ESSONNE ,  ASSOCIATION INSTITUT DE MYOLOGIE

Inventor： GIUSEPPE RONZITTI ,  PATRICE VIDAL ,  FEDERICO MINGOZZI

IPC: C12N9/44 ,  C12N15/86 ,  C12N9/10

Abstract: The present invention relates to a mini-GDE for the treatment of glycogen storage disease III.

公开(公告)号：US11104897B2

公开(公告)日：2021-08-31

申请号：US15569203

申请日：2016-04-27

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

Inventor： Ana Maria Buj Bello ,  Mirella Lo Scrudato

IPC: C12N15/113 ,  A61K48/00 ,  C12N15/11 ,  C12N5/10 ,  C12N15/10

Abstract: The present invention relates to compositions and methods for the treatment of nucleotide repeat expansion disorders such as myotonic dystrophy.

公开(公告)号：US20210228738A1

公开(公告)日：2021-07-29

申请号：US17050911

申请日：2017-07-17

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: A61K48/00 ,  C12N9/24 ,  C12N15/86 ,  G01N33/53 ,  C12N15/113

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：US20210162020A1

公开(公告)日：2021-06-03

申请号：US16935437

申请日：2020-07-22

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 ,  C12N9/16

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：US20200291402A1

公开(公告)日：2020-09-17

申请号：US16807919

申请日：2020-03-03

Applicant: INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) ,  GENETHON ,  UNIVERSITE PARIS DESCARTES ,  ENSCP - CHIMIE PARIS TECH - ECOLE NATIONALE SUPERIEURE DE CHIME DE PARIS ,  ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS

Inventor： Jean-Michel Rozet ,  Antoine Kichler ,  Isabelle Perrault ,  Josseline Kaplan ,  Xavier Gerard ,  Daniel Scherman ,  M. Arnold Munnich

IPC: C12N15/113 ,  A61K48/00

Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c. 2991+1655 A>G CEP290 mRNA.

公开(公告)号：US10767192B2

公开(公告)日：2020-09-08

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

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：US10752898B2

公开(公告)日：2020-08-25

申请号：US16060396

申请日：2016-12-09

Applicant: GENETHON

Inventor： France Pietri-Rouxel ,  Virginie Francois

IPC: C12N15/113 ,  A61P21/00 ,  A61K48/00 ,  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：US20200069790A1

公开(公告)日：2020-03-05

申请号：US16490181

申请日：2018-03-01

Applicant: GENETHON ,  INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) ,  SORBONNE UNIVERSITÉ ,  ASSOCIATION INSTITUT DE MYOLOGIE

Inventor： BÉRANGÉRE BERTIN ,  CAROLE MASURIER ,  OTTO-WILHELM MERTEN ,  FEDERICO MINGOZZI

IPC: A61K39/235 ,  A61M1/36 ,  C07K16/08 ,  C07K16/06 ,  C12N7/00

Abstract: The present invention relates to a method for removing undesired anti-AAV antibodies from a blood-derived composition.

公开(公告)号：US20190390224A1

公开(公告)日：2019-12-26

申请号：US16332376

申请日：2017-09-12

Applicant: GENETHON ,  SORBONNE UNIVERSITE

Inventor： FEDERICO MINGOZZI ,  GIUSEPPE RONZITTI ,  PASQUALINA COLELLA ,  FRANCESCO PUZZO

IPC: C12N15/86 ,  A61K35/34 ,  A61K35/407

Abstract: The present invention relates to variants of acid-alpha glucosidase and uses thereof.

公开(公告)号：US09981049B2

公开(公告)日：2018-05-29

申请号：US14782396

申请日：2014-04-10

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 ,  C07K14/4707 ,  C12N9/16 ,  C12N9/6472 ,  C12N15/86 ,  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/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.