公开(公告)号：EP4324845A2

公开(公告)日：2024-02-21

申请号：EP23201456.3

申请日：2014-01-31

申请人： Institut National de la Santé et de la Recherche Médicale (INSERM) ,  Centre National de la Recherche Scientifique (CNRS) ,  Université de Strasbourg ,  Cornell University ,  Université Paris-Saclay ,  APHP (Assistance Publique - Hôpitaux de Paris)

发明人： PUCCIO, Hélène Monique ,  AUBOURG, Patrick ,  CRYSTAL, Ronald G ,  BOUGNERES, Pierre

IPC分类号： C07K14/47

摘要： The present invention relates a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure.
More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.

公开(公告)号：EP4324845A3

公开(公告)日：2024-04-17

申请号：EP23201456.3

申请日：2014-01-31

申请人： Institut National de la Santé et de la Recherche Médicale (INSERM) ,  Centre National de la Recherche Scientifique (CNRS) ,  Université de Strasbourg ,  Cornell University ,  Université Paris-Saclay ,  APHP (Assistance Publique - Hôpitaux de Paris)

发明人： PUCCIO, Hélène Monique ,  AUBOURG, Patrick ,  CRYSTAL, Ronald G ,  BOUGNERES, Pierre

IPC分类号： A61K31/7088 ,  A61K38/44 ,  C07K14/47 ,  A61K48/00 ,  A61P9/00

CPC分类号： A61K48/005 ,  C07K14/47 ,  C12N2750/1403220130101 ,  C12N2750/1407120130101 ,  C12N2750/1414320130101 ,  A61K31/7088 ,  A61K38/44 ,  C12Y116/03001 ,  A61P9/00

摘要： The present invention relates a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure.
More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.

公开(公告)号：EP2950824A1

公开(公告)日：2015-12-09

申请号：EP14702565.4

申请日：2014-01-31

申请人： INSERM - Institut National de la Santé et de la Recherche Médicale ,  Centre National de la Recherche Scientifique (C.N.R.S.) ,  Université de Strasbourg ,  Cornell University ,  Université Paris Sud-Paris XI ,  APHP (Assistance Publique - Hôpitaux de Paris)

发明人： PUCCIO, Hélène Monique ,  AUBOURG, Patrick ,  CRYSTAL, Ronald G. ,  BOUGNERES, Pierre

IPC分类号： A61K48/00 ,  C07K14/47

CPC分类号： C12N15/86 ,  A61K9/0019 ,  A61K31/7088 ,  A61K38/44 ,  A61K48/00 ,  A61K48/0016 ,  A61K48/005 ,  C07K14/47 ,  C12N7/00 ,  C12N2750/14032 ,  C12N2750/14071 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12Y116/03001

摘要： A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).

摘要翻译： 提供了一种用于在需要其的受试者的预防或治疗由于能量衰竭心肌病的方法。 该方法包括施用给所述受试者治疗有效量的包含编码基因也可逆转能量衰竭的核酸序列的载体的。 示例性心肌病与做所有这一切是弗里德赖希共济失调和示例性的核酸序列相关的包括核酸确实编码frataxin（FXN）。