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公开(公告)号：US12049500B2
公开(公告)日：2024-07-30
申请号：US18263430
申请日：2022-03-31
申请人： EMERGENCE THERAPEUTICS GMBH , UNIVERSITE D'AIX-MARSEILLE , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , INSTITUT JEAN PAOLI & IRENE CALMETTES , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE—CNRS
发明人： Jack Elands , Florence L'Hospice , Xavier Préville , Daniel Olive , Marc Lopez
CPC分类号： C07K16/2803 , A61K47/68037 , A61K47/6849 , A61P35/00 , C07K2317/21 , C07K2317/24 , C07K2317/31
摘要： The present invention relates to antibodies having specificity to Nectin-4 and uses thereof.
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公开(公告)号：US11939577B2
公开(公告)日：2024-03-26
申请号：US17279206
申请日：2019-09-24
申请人： CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
发明人： Liliane Massade , Charbel Massaad , Susan Boutary , Giorgia Maria Laura Urbinati , Patrick Couvreur , Didier Desmaële
IPC分类号： C12N15/113 , A61K9/51 , A61K45/06 , A61K47/69
CPC分类号： C12N15/113 , A61K9/5123 , A61K45/06 , A61K47/6929 , C12N2310/11 , C12N2310/14 , C12N2310/351 , C12N2320/32
摘要： The present invention relates to antisense RNAs targeting PMP22 and able to inhibit from 40% to 60% the expression of PMP22 in the cells and a pharmaceutical composition comprising thereof. The antisense RNAs are preferably siRNA and are preferably provided in the form of nanoparticles. The present invention also relates to the use of these antisense RNAs targeting PMP22 for the treatment of Charcot-Marie-Tooth 1A disease.
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3.发明公开公开(公告)号：US20240096495A1
公开(公告)日：2024-03-21
申请号：US18510726
申请日：2023-11-16
IPC分类号： G16H50/20 , C12Q1/6886 , G16H20/10 , G16H50/50
CPC分类号： G16H50/20 , C12Q1/6886 , G16H20/10 , G16H50/50 , C12Q2600/106 , C12Q2600/112 , C12Q2600/118 , C12Q2600/158
摘要： The present invention provides a powerful tool to identify personalized therapeutic strategies. In particular, the invention provides methods for determining therapeutically targetable dominant signaling pathways in a cancer sample from a subject affected with a solid cancer, determining a treatment protocol for the subject, selecting a subject for a therapy, determining whether the subject is susceptible to benefit from a therapy, predicting clinical outcome of the subject, treating the subject and/or predicting the sensitivity of a solid cancer to a therapy.
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公开(公告)号：US11926829B2
公开(公告)日：2024-03-12
申请号：US17166377
申请日：2021-02-03
申请人： ASSOCIATION ISNTITUT DE MYOLOGIE , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , SORBONNE UNIVERSITE , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS)
发明人： Marc Bitoun , Delphine Trochet , Bernard Prudhon
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2320/34 , C12Y306/05005
摘要： The invention relates to an allele specific siRNA able to silence the expression of only one allele of a heterozygous DNM2 gene, for treating diseases caused by heterozygous mutation and/or overexpression of Dynamin 2.
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公开(公告)号：US20240034791A1
公开(公告)日：2024-02-01
申请号：US18263430
申请日：2022-03-31
申请人： EMERGENCE THERAPEUTICS AG , UNIVERSITE D’AIX-MARSEILLE , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , INSTITUT JEAN PAOLI & IRENE CALMETTES , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE – CNRS –
发明人： Jack ELANDS , Florence LHOSPPICE , Xavier PRÉVILLE , Daniel OLIVE , Marc LOPEZ
CPC分类号： C07K16/2803 , A61K47/68037 , A61K47/6849 , A61P35/00 , C07K2317/21 , C07K2317/24 , C07K2317/31
摘要： The present invention relates to antibodies having specificity to Nectin-4 and uses thereof.
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6.发明授权公开(公告)号：US11857532B2
公开(公告)日：2024-01-02
申请号：US17418981
申请日：2019-12-27
申请人： INSERM (Institut National de la Santé et de la Recherche Médicale) , Université de Paris , Assistance Publique—Hôpitaux de Paris (APHP) , Fondation Imagine
发明人： Anne Agnès Rotig , Arnold Munnich , Floriane Petit
IPC分类号： A01N43/04 , A61K31/70 , A61K31/357 , A61K31/194 , A61K31/7076
CPC分类号： A61K31/357 , A61K31/194 , A61K31/7076
摘要： Friedreich ataxia (FRDA) is caused by a GAA repeat expansion in FXN gene that encodes a mitochondrial protein, frataxin, involved in iron sulfur complex (ISC) assembly. Frataxin deficiency results in abnormal ISC containing proteins, namely respiratory chain complex I-III and aconitases and accumulation of iron in brain and heart of patients. Here, the inventors show that FRDA fibroblasts are unable to limit iron uptake inducing a massive cytosolic iron accumulation and to a lesser extent in mitochondria. The inventors also observed increased transferrin receptor (TfR1) steady state levels and membrane TfR1 accumulation that they ascribed to impaired post-translational modification by palmitoylation as well as delayed transferrin recycling. Finally, the inventors showed that artesunate, dichloroacetate and Coenzyme-A improved TfR1 palmitoylation and thus represent candidate molecules for the treatment of patients with Friedreich ataxia. Thus the present invention relates to methods of treating Friedreich ataxia (FRDA) as well as to methods of predicting whether a patient suffering from FRDA will achieve a therapeutic response.
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公开(公告)号：US20230416196A1
公开(公告)日：2023-12-28
申请号：US18037331
申请日：2021-11-18
申请人： INSTITUT CURIE , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , PARIS SCIENCES ET LETTRES
发明人： RAPHAËL RODRIGUEZ , ANTOINE VERSINI , TATIANA CAÑEQUE , SEBASTIAN MÜLLER , STÉPHANIE SOLIER , LEEROY BARON
IPC分类号： C07C279/26 , A61P35/00
CPC分类号： C07C279/265 , A61P35/00
摘要： The present invention relates to compounds comprising two biguanidyl radicals that can be useful as anti-inflammatory agent, and also to new compounds comprising two biguanidyl radicals and their use as a drug, in particular for treating a cancer, a metabolic disease, a secondary mitochondrial disorder due to copper overload including Indian childhood cirrhosis, Wilson's disease and Idiopathic infantile copper toxicosis or due to iron overload, an infection by a virus such as a coronavirus or an influenza virus, a neurodegenerative disease or disorder and aging.
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公开(公告)号：US20230322952A1
公开(公告)日：2023-10-12
申请号：US17791549
申请日：2021-01-08
申请人： BIOMUNEX PHARMACEUTICALS , INSTITUT CURIE , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
发明人： OLIVIER LANTZ , SEBASTIAN AMIGORENA , MICHAEL SAITAKIS , MAUDE GUILLOT-DELOST , EUGENE ZHUKOVSKY , PIERRE-EMMANUEL GERARD , MUSTAPHA FAROUDI
CPC分类号： C07K16/468 , A61P35/00 , C07K2317/31 , C07K2317/55 , C07K2317/522 , C07K2317/565
摘要： The invention provides a multispecific molecule capable of simultaneous binding to a Mucosal Associated Invariant T (MAIT) cell and a tumor cell, which multispecific molecule comprises at least one domain that specifically binds a Vα7.2 T cell receptor (TCR) and at least one domain that specifically binds a tumor associated antigen (TAA).
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公开(公告)号：US20230270885A2
公开(公告)日：2023-08-31
申请号：US17753768
申请日：2020-09-18
申请人： GENETHON , UNIVERSITE D’EVRY-VAL-D’ESSONNE , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
发明人： Evelyne GICQUEL , Isabelle RICHARD
CPC分类号： A61K48/0058 , A61K48/0008 , A61K9/0019 , A61P21/00
摘要： The present invention concerns an expression system for systemic administration comprising a sequence encoding a FKRP protein, and:
a promoter sequence allowing the expression at a therapeutically acceptable level of FKRP in the skeletal muscles and a target sequence of an miRNA expressed in the heart; or
a promoter sequence allowing the expression at a therapeutically acceptable level of FKRP in the skeletal muscles and presenting a promoter activity at a toxically acceptable level in the heart;
and its use for the treatment of various diseases linked to FKRP deficiencies.-
10.发明公开公开(公告)号：US20230256117A1
公开(公告)日：2023-08-17
申请号：US18001648
申请日：2021-06-18
申请人： GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D’EVRY-VAL-D’ESSONNE
发明人： Isabelle RICHARD , Jerome POUPIOT
IPC分类号： A61K48/00
CPC分类号： A61K48/0058
摘要： The present invention concerns an expression system for systemic administration comprising a sequence encoding gamma-sarcoglycan (SGCG) placed under the control of a promoter allowing an adequate expression of SGCG in the skeletal muscles and in the heart, and its use for the treatment of Limb-Girdle Muscular Dystrophy type C.
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