公开(公告)号：US20230203537A1

公开(公告)日：2023-06-29

申请号：US17929816

申请日：2022-09-06

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

Inventor： Anne GALY

IPC: C12N15/86 ,  A61K35/76 ,  C07K14/005 ,  C12N5/0781 ,  A61K35/17

CPC classification number: C12N15/86 ,  A61K35/76 ,  C07K14/005 ,  C12N5/0635 ,  A61K35/17 ,  A61K48/00

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：US20230117384A1

公开(公告)日：2023-04-20

申请号：US17750705

申请日：2022-05-23

Applicant: GENETHON ,  INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) ,  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：US11596698B2

公开(公告)日：2023-03-07

申请号：US16628650

申请日：2018-07-06

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 ,  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：US20230033268A1

公开(公告)日：2023-02-02

申请号：US17770641

申请日：2020-10-22

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

Inventor： PASQUALINA COLELLA ,  FRANCESCO PUZZO ,  FEDERICO MINGOZZI

IPC: C07K7/08 ,  C12N15/86 ,  A61P3/00

Abstract: The present invention relates to a nucleic acid molecule encoding a chimeric polypeptide comprising a peptide of interest fused to one or more heterologous moieties, wherein at least one of the heterologous moieties is a ligand of the Sortilin receptor. The invention also relates to a chimeric polypeptide encoded by said nucleic acid molecule and uses thereof.

公开(公告)号：US20230013736A1

公开(公告)日：2023-01-19

申请号：US17905290

申请日：2021-03-02

Applicant: ADHARA ,  CENTRE D'ETUDE DES CELLULES SOUCHES (CECS) ,  UNIVERSITE D'EVRY VAL D'ESSONNE

Inventor： Christine BALDESHI ,  Sophie DOMINGUES ,  Christelle LAURENSOU ,  Marielle BOUSCHBACHER ,  Sophie CLEMENT ,  Guillaume OLIVE

IPC: A61L27/60 ,  A61L27/22 ,  A61L27/38

Abstract: The invention relates to a process for obtaining a skin substitute, comprising the following steps: a) mixing fibroblasts, endothelial cells and hydrogel of exclusively biological origin; b) incubating the mixture obtained in step a) for a sufficient time and under suitable conditions to obtain a pre-vascularized dermis; c) adding keratinocytes to the pre-vascularized dermis of step b) to obtain a skin substitute; wherein said fibroblasts, endothelial cells and keratinocytes were obtained from pluripotent stem cells.

公开(公告)号：US11339406B2

公开(公告)日：2022-05-24

申请号：US16332379

申请日：2017-09-12

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

Inventor： Federico Mingozzi ,  Giuseppe Ronzitti

IPC: C12N9/24 ,  C12N15/90 ,  C12N15/87 ,  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：US20210346519A1

公开(公告)日：2021-11-11

申请号：US16968196

申请日：2019-02-07

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

Inventor： FEDERICO MINGOZZI ,  PASQUALINA COLELLA

IPC: A61K48/00 ,  C12N15/86

Abstract: The present invention relates to hybrid transcription regulatory elements to drive gene expression, in particular hybrid promoters, designed by the fusion of at least two transcription regulatory elements with different tissue selectivity, such as two promoters driving expression in different tissues in a tissue-selective manner.

公开(公告)号：US20210059999A1

公开(公告)日：2021-03-04

申请号：US16960503

申请日：2019-01-10

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

Inventor： Isabelle RICHARD ,  Dorianna SANDONA

IPC: A61K31/47 ,  A61K31/427 ,  A61P21/00

Abstract: Sarcoglycanopathies are autosomal recessive diseases caused by mutations in the one of the genes coding for any sarcoglycans (SG). The inventors previously showed that the application of small molecules developed to rescue ΔF508-CFTR trafficking, and known as CFTR correctors, improved the maturation of several α-sarcoglycan mutants that were consequently rescued at the plasma membrane (WO 014086687). Now, the inventors show that some specific CFTR correctors provide additive and even synergic effect when administered in combination.

公开(公告)号：US20190390225A1

公开(公告)日：2019-12-26

申请号：US16332379

申请日：2017-09-12

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

Inventor： FEDERICO MINGOZZI ,  GIUSEPPE RONZITTI

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：US20170305892A1

公开(公告)日：2017-10-26

申请号：US15518127

申请日：2015-10-07

Applicant: INSTITUT NATIONAL DE LA SANTE ET DE LA RECERCHERCHE MEDICALE (INSERM) ,  ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES ,  UNIVERSITE D'EVRY VAL D'ESSONNE

Inventor： Marc PESCHANSKI ,  Sophie BLONDEL ,  Xavier NISSAN

IPC: C07D409/14 ,  C07D403/12 ,  C07D401/14 ,  C07D405/14 ,  C07D401/06

CPC classification number: C07D409/14 ,  C07D401/02 ,  C07D401/06 ,  C07D401/14 ,  C07D403/12 ,  C07D405/14

Abstract: A compound for use in the treatment and/or prevention of diseases or disorders wherein an inhibition of protein prenylation is required, which is alternatively chosen among wherein R1 is 2-pyridyl, 3-pyridyl or 4-pyridyl; R2 represents: and wherein R5 and R6 represent a hydrogen atom or a (C1-4)alkyl group; and R7 represents an arylcarbonyl group, a heteroarylcarbonyl group, a heteroarylacetyl group, a (C1-C4)alkoxy-carbonylmethyl group, a group. Also disclosed are novel compounds of formulae (I) and (II).