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1. 发明申请

WO2013030243A1 METHOD FOR PREPARING INDUCED PARAXIAL MESODERM PROGENITOR (IPAM) CELLS AND THEIR USE 审中-公开
标题翻译：用于制备诱导型间质细胞原核细胞（IPAM）细胞及其用途的方法
公开(公告)号：WO2013030243A1
公开(公告)日：2013-03-07
申请号：PCT/EP2012/066793
申请日：2012-08-29
申请人： INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , CNRS (CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE) , UNIVERSITE DE STRASBOURG , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES , POURQUIE, Olivier , CHAL, Jérôme
发明人： POURQUIE, Olivier , CHAL, Jérôme
IPC分类号： C12N5/0775
CPC分类号： C12N5/0606 , C12N5/0625 , C12N5/0653 , C12N5/0654 , C12N5/0658 , C12N5/0662 , C12N2500/62 , C12N2501/155 , C12N2501/40 , C12N2501/415 , C12N2501/727 , C12N2506/02
摘要： The present invention relates to an ex vivo method for preparing induced paraxial mesoderm progenitor (iPAM) cells, said method comprising the step of culturing pluripotent cells in an appropriate culture medium comprising an effective amount of an activator of the Wnt signaling pathway and an effective amount of an inhibitor of the Bone Morphogenetic Protein (BMP) signaling pathway.
摘要翻译：本发明涉及一种制备诱导的旁轴中胚层祖细胞（iPAM）细胞的离体方法，所述方法包括在合适的培养基中培养多能细胞的步骤，该培养基包含有效量的Wnt信号传导途径的活化剂和有效量的骨形态发生蛋白（BMP）信号通路的抑制剂。

2. 发明申请

WO2008003864A1 COMBINAISON D'UN INHIBITEUR DE HMG-COA REDUCTASE ET D'UN INHIBITEUR DE FARNESYL-PYROPHOSPHATASE SYNTHASE POUR TRAITER LES MALADIES LIEES A L'ACCUMULATION ET/OU LA PERSISTANCE DE PROTEINES PRENYLEES 审中-公开
标题翻译： HMG-COA还原酶抑制剂和呋喃糖苷酶合成酶抑制剂的组合用于治疗与持续和/或累积蛋白相关的疾病的疾病
公开(公告)号：WO2008003864A1
公开(公告)日：2008-01-10
申请号：PCT/FR2007/001144
申请日：2007-07-05
申请人： UNIVERSITE DE LA MEDITERRANEE AIX-MARSEILLE II , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES (AFM) , ASSISTANCE PUBLIQUE HÔPITAUX DE MARSEILLE , UNIVERSIDAD DE OVIEDO , LEVY, Nicolas , CAU, Pierre , LOPEZ-OTIN, Carlos
发明人： LEVY, Nicolas , CAU, Pierre , LOPEZ-OTIN, Carlos
IPC分类号： A61K31/22 , A61K31/663 , A61P17/00 , A61P43/00
CPC分类号： A61K31/675 , A61K8/4913 , A61K8/498 , A61K8/55 , A61K31/215 , A61K31/22 , A61K31/663 , A61Q19/08 , A61K2300/00
摘要： L'invention a pour objet l'utilisation d'un inhibiteur de rhydroxyméthylglutaryl-coenzyme A (HMG-CoA) réductase et d'un inhibiteur de la farnésyl-pyrophosphate synthase, ou de l'un de leurs sels physiologiquement acceptable, associés dans la préparation d'une composition, particulièrement une composition pharmaceutique, destinée au traitement des situations, pathologiques ou non, humaines ou animales, liées à l'accumulation et/ou la persistance dans les cellules de protéines prénylées, comme au cours de la Progéria (syndrome de Hutchinson-Gilford), de la dermopathie restrictive ou du vieillissement physiologique.
摘要翻译：本发明涉及羟甲基戊二酰辅酶A（HMG-CoA）还原酶抑制剂和法呢基焦磷酸合酶抑制剂或其相关的生理上可接受的盐之一在制备组合物，特别是药物组合物中的用途，用于治疗人或动物，病理或非病理学情况，其与细胞中异戊二烯基化蛋白质的累积和/或持续性有关，例如在前期（Hutchinson-Gilford综合征），限制性皮肤病或生理衰老期间。

3. 发明申请

WO2021123682A1 MODÈLE ORGANOÏDE CARDIAQUE VASCULARISÉ APRES INCORPORATION DE CARDIOMYOCYTES DÉRIVÉS DE CELLULES SOUCHES PLURIPOTENTES INDUITES HUMAINES 审中-公开
公开(公告)号：WO2021123682A1
公开(公告)日：2021-06-24
申请号：PCT/FR2020/052557
申请日：2020-12-18
申请人： CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , UNIVERSITE DE MONTPELLIER , ECOLE NATIONALE SUPERIEURE DE CHIMIE DE MONTPELLIER , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) , UNIVERSITE CLAUDE BERNARD LYON 1 , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES , HOSPICES CIVILS DE LYON , DIAG'NCELL
发明人： NIRDE, Philippe , RICHARD, Sylvain , MOREAU, Adrien , CHEVALIER, Philippe
IPC分类号： C12N5/077 , C12N5/071 , C12N2502/025 , C12N2502/28 , C12N2503/02 , C12N2506/45 , C12N2533/92 , C12N5/0657 , C12N5/0697
摘要： La présente invention a pour objet un modèle organoïde cardiaque vascularisé comprenant des cardiomyocytes dérivés de cellules souches pluripotentes induites humaines (hiPSC-CM) greffés sur une membrane chorio-allantoïde d'œuf fécondé. La présente invention a également pour objet un procédé d'obtention dudit modèle organoïde cardiaque vascularisé. La présente invention a également pour objet un procédé de caractérisation de l'activité d'un composé vis-à-vis du modèle organoïde cardiaque vascularisé. La présente invention a également pour objet un kit comprenant ledit modèle organoïde cardiaque vascularisé.

4. 发明申请

WO2020254632A1 USE OF DISULFIRAM OR ITS DERIVATIVES FOR THE TREATMENT OF MITOCHONDRIAL DISEASES OR DYSFUNCTION 审中-公开
公开(公告)号：WO2020254632A1
公开(公告)日：2020-12-24
申请号：PCT/EP2020/067195
申请日：2020-06-19
申请人： UNIVERSITE D'ANGERS , CENTRE HOSPITALIER UNIVERSITAIRE D'ANGERS , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , UNIVERSITE DE BRETAGNE OCCIDENTALE , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES , UNIVERSITE DE PARIS , UNIVERSITE PARIS-SACLAY , UNIVERSITE DE BORDEAUX
发明人： PROCACCIO, Vincent , RÖTIG, Agnès , DELAHODDE, Agnès , TRIBOUILLARD-TANVIER, Déborah , DUJARDIN, Geneviève , BLONDEL, Marc
IPC分类号： A61K31/145 , A61P3/00
摘要： The present invention relates to the use of disulfiram or one of its derivatives for use in the treatment of a mitochondrial dysfunction or diseases, advantageously of a genetic mitochondrial disease.

5. 发明申请

WO2016055517A1 NOVEL AMINOPYRIDINE COMPOUNDS USEFUL AS INHIBITORS OF PROTEIN PRENYLATION 审中-公开
标题翻译：新型氨基吡啶化合物作为蛋白质预处理的抑制剂有用
公开(公告)号：WO2016055517A1
公开(公告)日：2016-04-14
申请号：PCT/EP2015/073141
申请日：2015-10-07
申请人： INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES , UNIVERSITE D'EVRY VAL D'ESSONNE
发明人： PESCHANSKI, Marc , BLONDEL, Sophie , NISSAN, Xavier
IPC分类号： C07D403/12 , C07D401/14 , C07D401/02 , A61K31/506 , A61P25/00 , A61P3/00 , A61P35/00 , A61P9/00 , A61P31/12
CPC分类号： C07D409/14 , C07D401/02 , C07D401/06 , C07D401/14 , C07D403/12 , C07D405/14
摘要： The present invention relates to 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 formulae (I) and (II), wherein R 1 is 2-pyridyl, 3-pyridyl or 4-pyridyl; R 2 represents formulae (III), (IV) and (V); and wherein R 5 and R 6 represent a hydrogen atom or a (C 1-4 )alkyl group; and R 7 represents an arylcarbonyl group, a heteroarylcarbonyl group, a heteroarylacetyl group, a (C 1 -C 4 )alkoxy-carbonylmethyl group,a formula (VI) group. Also disclosed are novel compounds of formulae (I) and (II).
摘要翻译：本发明涉及用于治疗和/或预防疾病或病症的化合物，其中在式（I）和（II）中需要另外选择蛋白异戊烯基化的蛋白异戊烯基化，其中R1是2-吡啶基，3-吡啶基或4-吡啶基; R2代表式（Ⅲ），（Ⅳ）和（Ⅴ）; 并且其中R 5和R 6表示氢原子或（C 1-4）烷基; 并且R 7表示芳基羰基，杂芳基羰基，杂芳基乙酰基，（C 1 -C 4）烷氧基 - 羰基甲基，式（VI）基团。还公开了式（I）和（II）的新化合物。

6. 发明申请

WO2003087373A3 PRODUCTION OF RECOMBINANT FRAGMENTS OF MUSCLE ACETYLCHOLINE RECEPTOR AND THEIR USE FOR EX VIVO IMMUNOADSORPTION OF ANTI-ACH RECEPTOR ANTIBODIES FROM MYASTHENIC PATIENTS 审中-公开
公开(公告)号：WO2003087373A3
公开(公告)日：2003-10-23
申请号：PCT/GR2003/000014
申请日：2003-04-15
申请人： HELLENIC PASTEUR INSTITUTE , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES , TZARTOS, Socrates , MAMALAKI, Avgi , PSARIDI-LINARDAKI, Loukia , KOSTELIDOU, Kalliopi
发明人： TZARTOS, Socrates , MAMALAKI, Avgi , PSARIDI-LINARDAKI, Loukia , KOSTELIDOU, Kalliopi
IPC分类号： C12N15/12
摘要： A procedure which involves the production of extracellular domains (or larger parts including these domains) of human muscle acetylcholine receptor (AChR) expressed in heterologous or homologous systems and obtained in sufficiently folded state appropriate for binding major fractions of anti-AChR antibodies in myasthenia gravis (MG) patients and of immunoadsorbing these antibodies when said polypeptides are used as permanently immobilized on insoluble matrixes. This procedure allows the ex vivo selective elimination of the antibodies against human muscle acetylcholine receptor (AChR), as a replacement and improvement of the non-specific plasmapheresis or Ig apheresis, for the temporary repeated treatment of the disease MG.

7. 发明申请

WO2016128408A1 RECOMBINANT ADENO-ASSOCIATED VIRUS PARTICLE PURIFICATION COMPRISING AN AFFINITY PURIFICATION STEP 审中-公开
标题翻译：重组腺病毒相关病毒颗粒纯化，包括亲和纯化步骤
公开(公告)号：WO2016128408A1
公开(公告)日：2016-08-18
申请号：PCT/EP2016/052740
申请日：2016-02-09
申请人： INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) , UNIVERSITE DE NANTES , CHU NANTES , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES
发明人： BRUMENT, Nicole
IPC分类号： C12N7/02 , A61K48/00
CPC分类号： C12N7/00 , C12N2750/14151
摘要： The invention describes a method for obtaining purified recombinant Adeno-Associated Virus particles (rAAV), comprising the steps of: a) performing a depth filtration of a starting material previously obtained from cells producing rAAV particles, the said starting material being selected in a group comprising a cell lysate and a culture supernatant, whereby a rAAV-containing clarified composition is provided; b) submitting the rAAV-containing clarified composition to an affinity purification step, whereby a first rAAV enriched composition is provided; c) submitting the first rAAV enriched composition at least once to: cl) a step of anion-exchange chromatography on a chromatographic support wherein elution is performed by using a salt gradient, preferably a linear salt gradient, and wherein the rAAV-containing fraction is collected, whereby a second rAAV enriched composition is provided; or c2) a step of density gradient centrifugation, wherein the rAAV-containing fraction is collected, whereby a second rAAV enriched composition is provided; d) submitting the second rAAV enriched composition to a step of tangential flow filtration, whereby purified recombinant Adeno-Associated Virus particles (rAAV) are provided.
摘要翻译：本发明描述了获得纯化的重组腺相关病毒颗粒（rAAV）的方法，包括以下步骤：a）对产生rAAV颗粒的细胞预先获得的起始材料进行深度过滤，所述起始材料选自组包括细胞裂解物和培养上清液，由此提供含rAAV的澄清组合物; b）将含有rAAV的澄清组合物递送至亲和纯化步骤，由此提供富含第一rAAV的组合物; c）将第一rAAV富集的组合物至少提供一次至：cl）在色谱载体上的阴离子交换层析步骤，其中通过使用盐梯度，优选线性盐梯度进行洗脱，并且其中含rAAV的级分为收集，从而提供第二种富含rAAV的组合物; 或c2）密度梯度离心的步骤，其中收集含rAAV的级分，由此提供第二rAAV富集组合物; d）将第二rAAV富集组合物提交到切向流过滤步骤，由此提供纯化的重组腺相关病毒颗粒（rAAV）。

8. 发明申请

WO2016113357A8 PROTEASOME INHIBITORS FOR TREATING A DISORDER RELATED TO AN ACCUMULATION OF NON-DEGRADED ABNORMAL PROTEIN OR A CANCER 审中-公开
标题翻译：蛋白酶抑制剂用于治疗与非降解的异常蛋白质或癌症累积有关的疾病
公开(公告)号：WO2016113357A8
公开(公告)日：2016-07-21
申请号：PCT/EP2016/050678
申请日：2016-01-14
申请人： UNIVERSITÉ D'AIX-MARSEILLE , ASSISTANCE PUBLIQUE HOPITAUX DE MARSEILLE , INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE - CNRS , PROGELIFE
发明人： LEVY, Nicolas , CAU, Pierre , DE SANDRE-GIOVANNOLI, Annachiara , HARHOURI, Karim , PERRIN, Sophie , NAVARRO, Claire , CHARTIER, Aymeric , SIMONELIG, Martine
IPC分类号： A61K38/06 , A61K8/64 , A61P43/00
摘要： The invention relates to a proteasome inhibitor for use for treating and/or preventing a disorder related to an accumulation of a non-degraded abnormal protein, particularly a premature ageing disorder. The invention also relates to the use of proteasome inhibitors for attenuating physiological ageing. The invention also relates to the use of proteasome inhibitors in the treatment and/or prevention of age-related disorders and their metabolic consequences. The invention also relates to the dermato logical, dermo cosmetic or cosmetic use of a proteasome inhibitor for preventing and/or attenuating skin ageing. The invention also relates to the use of proteasome inhibitors in the treatment of cancer.
摘要翻译：本发明涉及蛋白酶体抑制剂，其用于治疗和/或预防与未降解的异常蛋白质，特别是早衰症的积聚有关的病症。本发明还涉及蛋白酶体抑制剂用于减弱生理老化的用途。本发明还涉及蛋白酶体抑制剂在治疗和/或预防年龄相关疾病及其代谢后果中的用途。本发明还涉及用于预防和/或减轻皮肤老化的蛋白酶体抑制剂的皮肤美容，皮肤美容或美容用途。本发明还涉及蛋白酶体抑制剂在治疗癌症中的用途。

9. 发明申请

WO2014170342A1 METHOD AND MEDIUM FOR AMPLIFYING NEURAL PRECURSOR CELLS 审中-公开
标题翻译：用于放大神经前体细胞的方法和介质
公开(公告)号：WO2014170342A1
公开(公告)日：2014-10-23
申请号：PCT/EP2014/057658
申请日：2014-04-15
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITE D'EVRY VAL D'ESSONE , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES
发明人： PESCHANSKI, Marc , BENCHOUA, Alexandra
IPC分类号： C12N5/0793 , C12N5/0797 , A61K35/30
CPC分类号： C12N5/0623 , A61K35/30 , C12N5/0619 , C12N2500/90 , C12N2501/11 , C12N2501/115 , C12N2501/13 , C12N2501/155 , C12N2501/16 , C12N2501/727 , C12N2506/02 , C12N2533/32 , C12N2533/52 , G01N33/5058
摘要： The present invention relates to a method for amplifying a population of neural precursors comprising the step of culturing neural precursors in the presence of a PKA inhibitor.
摘要翻译：本发明涉及一种用于扩增神经前体群体的方法，包括在PKA抑制剂存在下培养神经前体的步骤。

10. 发明申请

WO2010063848A1 METHOD AND MEDIUM FOR NEURAL DIFFERENTIATION OF PLURIPOTENT CELLS 审中-公开
标题翻译：用于神经细胞分化的方法和介质
公开(公告)号：WO2010063848A1
公开(公告)日：2010-06-10
申请号：PCT/EP2009/066504
申请日：2009-12-07
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES , BENCHOUA, Alexandra , PERRIER, Anselme , AUBRY, Laetitia
发明人： BENCHOUA, Alexandra , PERRIER, Anselme , AUBRY, Laetitia
IPC分类号： C12N5/07
CPC分类号： C12N5/0619 , A61K35/30 , C12N5/0623 , C12N2500/05 , C12N2500/32 , C12N2500/36 , C12N2500/38 , C12N2500/40 , C12N2501/15 , C12N2501/155 , C12N2501/16 , C12N2506/02 , C12N2506/45 , G01N33/5058
摘要： The invention relates to a culture medium comprising an inhibitor of the BMP signaling pathway; and an inhibitor of the TGF/activin/nodal signaling pathway and to a method for obtaining a population of neural precursors using said culture medium.
摘要翻译：本发明涉及包含BMP信号通路抑制剂的培养基; 和TGFβ/激活素/节点信号传导途径的抑制剂以及使用所述培养基获得神经前体群体的方法。

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