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1. 发明授权

US10626382B2 Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency 有权
公开(公告)号：US10626382B2
公开(公告)日：2020-04-21
申请号：US15861427
申请日：2018-01-03
申请人： The Trustees of the University of Pennsylvania
发明人： Lili Wang , James M. Wilson
IPC分类号： C12N9/10 , A61K48/00 , C12N15/86
CPC分类号： C12N9/1018 , A61K48/0058 , C12N15/86 , C12Y201/03003 , C12N2710/10343 , C12N2740/15043 , C12N2750/14032 , C12N2750/14033 , C12N2750/14043 , C12N2750/14071 , C12N2750/14143 , C12N2800/22
摘要： Non-viral delivery systems comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.

2. 发明授权

US09890365B2 Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency 有权
公开(公告)号：US09890365B2
公开(公告)日：2018-02-13
申请号：US15124620
申请日：2015-03-09
申请人： The Trustees of the University of Pennsylvania
发明人： Lili Wang , James M. Wilson
IPC分类号： A61K48/00 , C12N9/10 , C12N15/86
CPC分类号： C12N9/1018 , A61K48/0058 , C12N15/86 , C12N2710/10343 , C12N2740/15043 , C12N2750/14032 , C12N2750/14033 , C12N2750/14043 , C12N2750/14071 , C12N2750/14143 , C12N2800/22 , C12Y201/03003
摘要： Non-viral delivery systems comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.

3. 发明申请

US20170051259A1 COMPOSITIONS USEFUL IN TREATMENT OF ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY 审中-公开
标题翻译：用于治疗异丙肾上腺素（OTC）缺乏症的组合物
公开(公告)号：US20170051259A1
公开(公告)日：2017-02-23
申请号：US15122853
申请日：2015-03-09
申请人： The Trustees of the University of Pennsylvania
发明人： Lili Wang , James M WILSON
IPC分类号： C12N9/10 , A61K48/00 , C12N15/86
CPC分类号： C12N9/1018 , A61K48/0058 , C12N15/86 , C12N2710/10343 , C12N2740/15043 , C12N2750/14032 , C12N2750/14033 , C12N2750/14043 , C12N2750/14071 , C12N2750/14143 , C12N2800/22 , C12Y201/03003
摘要： Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis cirrhosis in OTCD patients by administering hOTC.
摘要翻译：提供包含工程化hOTC DNA和RNA序列的病毒载体，其在递送至有需要的受试者时可用于治疗高氨血症，鸟氨酸转氨甲酰酶转氨基甲酰酶缺乏症和与之相关的症状。还提供了通过施用hOTC在OTCD患者中使用hOTC治疗肝纤维化肝硬化的方法。

4. 发明申请

US20160024526A1 METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE 审中-公开
标题翻译：用于治疗和预防由于能量失效引起的心脏病的方法和药物组合物
公开(公告)号：US20160024526A1
公开(公告)日：2016-01-28
申请号：US14764065
申请日：2014-01-31
申请人： APHP (ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS) , CORNELL UNIVERSITY , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , UNIVERSITE DE STRASBOURG , UNIVERSITÉ PARIS-SUD XI
发明人： Helene Monique PUCCIO , Patrick AUBORG , Ronald G. CRYSTAL , Pierre BOUGNERES
IPC分类号： C12N15/86 , A61K9/00
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： The present invention relates to 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.
摘要翻译：本发明涉及一种用于预防或治疗由于有需要的受试者的能量衰竭引起的心肌病的方法，其包括向所述受试者施用治疗有效量的载体，所述载体包含可恢复能量衰竭的基因的核酸序列。更具体地说，本发明涉及用于预防或治疗与有需要的受试者相关的弗里德赖希共济失调的心肌病的方法，包括向所述受试者施用治疗有效量的载体，所述载体包含编码核酸的frataxin（FXN）的载体。

5. 发明公开

US20230416700A1 COMPOSITIONS USEFUL IN TREATMENT OF ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY 审中-公开
公开(公告)号：US20230416700A1
公开(公告)日：2023-12-28
申请号：US18339457
申请日：2023-06-22
申请人： The Trustees of the University of Pennsylvania
发明人： Lili Wang , James M. Wilson
IPC分类号： C12N9/10 , C12N15/86 , A61K48/00
CPC分类号： C12N9/1018 , C12Y201/03003 , C12N15/86 , A61K48/0058 , C12N2750/14032 , C12N2750/14071 , C12N2750/14033 , C12N2710/10343 , C12N2740/15043 , C12N2750/14143 , C12N2800/22 , C12N2750/14043
摘要： Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.

6. 发明申请

US20180334687A1 METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE 审中-公开
公开(公告)号：US20180334687A1
公开(公告)日：2018-11-22
申请号：US15916907
申请日：2018-03-09
申请人： INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , UNIVERSITE DE STRASBOURG , Cornell University , UNIVERSITÉ PARIS-SUD XI , Assistance Publique-Hopitaux de Paris (APHP)
发明人： Helene Monique PUCCIO , Patrick AUBORG , Ronald G. CRYSTAL , Pierre BOUGNERES
IPC分类号： C12N15/86 , C12N7/00 , A61K31/7088 , A61K38/44 , A61K48/00 , C07K14/47 , A61K9/00
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： The present invention relates to 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.

7. 发明申请

US20140234261A1 Parvovirus Cancer Therapy and Combination with Chemotherapy 审中-公开
标题翻译：细小病毒癌症治疗与化疗联合
公开(公告)号：US20140234261A1
公开(公告)日：2014-08-21
申请号：US13788802
申请日：2013-03-07
申请人： Deutsches Krebsforschungszentrum Stiftung Des Oeffentlichen Rechts , Ruprecht-Karls-Universitaet Heidelberg
发明人： Jean ROMMELAERE , Zahari Raykov , Joerg Schlehofer , Irina Kiprijanova , Tanja Heimberger , Assia Angelova , Karsten Geletneky , Marc Aprahamian
IPC分类号： A61K35/76 , A61K31/495 , A61K31/7068
CPC分类号： A61K31/495 , A61K31/4188 , A61K31/7068 , A61K35/768 , C12N2750/14032 , A61K2300/00
摘要： Described is a pharmaceutical composition containing (a) a parvovirus and (b) a chemotherapeutic agent, preferably as separate entities. The parvovirus might be based on parvovirus H1, LuIII, Mouse minute virus (MMV), Mouse parvovirus (MPV), Rat minute virus (RMV), Rat parvovirus (RPV), Rat virus (RV), vectors based on the foregoing viral species, and/or cells capable of actively producing the foregoing viral species. The pharmaceutical composition is beneficial for the treatment of a tumor. Tumors for which a parvovirus or the adjunction of the invention has particular utility include glioma, medulloblastoma, meningioma and pancreatic cancer. Preferred chemotherapeutic agents are gemcitabine and Temozolodine.
摘要翻译：描述了包含（a）细小病毒和（b）化学治疗剂的药物组合物，优选作为分开的实体。细小病毒可能基于细小病毒H1，LuIII，小鼠微病毒（MMV），小鼠细小病毒（MPV），大鼠微小病毒（RMV），大鼠细小病毒（RPV），大鼠病毒（RV），基于上述病毒物种的载体，和/或能够主动产生上述病毒物种的细胞。该药物组合物有利于治疗肿瘤。细小病毒或本发明的辅助物具有特殊用途的肿瘤包括神经胶质瘤，成神经管细胞瘤，脑膜瘤和胰腺癌。优选的化学治疗剂是吉西他滨和替莫唑胺。

8. 发明授权

US08414883B2 Parvovirus cancer therapy and combination with chemotherapy 有权
标题翻译：细小病毒癌症治疗与化疗相结合
公开(公告)号：US08414883B2
公开(公告)日：2013-04-09
申请号：US12810457
申请日：2008-12-23
申请人： Jean Rommelaere , Zahari Raykov , Joerg Schlehofer , Irina Kiprijanova , Tanja Heimberger , Assia Angelova , Karsten Geletneky , Marc Aprahamian
发明人： Jean Rommelaere , Zahari Raykov , Joerg Schlehofer , Irina Kiprijanova , Tanja Heimberger , Assia Angelova , Karsten Geletneky , Marc Aprahamian
IPC分类号： A61K48/00 , A61K35/76 , A61P1/18
CPC分类号： A61K31/495 , A61K31/4188 , A61K31/7068 , A61K35/768 , C12N2750/14032 , A61K2300/00
摘要： Described is a pharmaceutical composition containing (a) a parvovirus and (b) a chemotherapeutic agent, preferably as separate entities. The parvovirus might be based on parvovirus H1, LuIII, Mouse minute virus (MMV), Mouse parvovirus (MPV), Rat minute virus (RMV), Rat parvovirus (RPV), Rat virus (RV), vectors based on the foregoing viral species, and/or cells capable of actively producing the foregoing viral species. The pharmaceutical composition is beneficial for the treatment of a tumor. Tumors for which a parvovirus or the adjunction of the invention has particular utility include glioma, medulloblastoma, meningioma and pancreatic cancer. Preferred chemotherapeutic agents are gemcitabine and Temozolodine.
摘要翻译：描述了包含（a）细小病毒和（b）化学治疗剂的药物组合物，优选作为分开的实体。细小病毒可能基于细小病毒H1，LuIII，小鼠微病毒（MMV），小鼠细小病毒（MPV），大鼠微小病毒（RMV），大鼠细小病毒（RPV），大鼠病毒（RV），基于上述病毒物种的载体，和/或能够主动产生上述病毒物种的细胞。该药物组合物有利于治疗肿瘤。细小病毒或本发明的辅助物具有特殊用途的肿瘤包括神经胶质瘤，成神经管细胞瘤，脑膜瘤和胰腺癌。优选的化学治疗剂是吉西他滨和替莫唑胺。

9. 发明申请

US20040248826A1 Treatment of cancer by in vivo gene-transfer induced TIMP-3 expression 审中-公开
标题翻译：通过体内基因转移诱导的TIMP-3表达来治疗癌症
公开(公告)号：US20040248826A1
公开(公告)日：2004-12-09
申请号：US10452878
申请日：2003-06-03
发明人： Alberto Auricchio , Markus Hildinger
IPC分类号： A61K048/00
CPC分类号： A61K38/57 , A61K48/00 , C12N2750/14032 , C12N2750/14143
摘要： Cancer is a major cause of death in developed countries. Yet, there is still a high degree of unmet need in the prevention, treatment and/or cure of cancer. The present invention relates to methods for treating cancer, preventing cancer and/or inhibiting the growth of cancer (cells) by administering to a mammalian subject a gene transfer vector in vivo comprising a nucleic acid composition whose expression directly or indirectly leads to the expression and/or secretion of Tissue inhibitor of metalloproteinase-3 (TIMP-3). Upon successful transduction, expression and/or secretion of TIMP-3nulleither locally (in the vicinity or within the cancer cells) or systemicallynullwill inhibit cancer growth. Also provided are pharmaceutical kits containing the gene transfer vector in a suitable pharmaceutical suspension for administration.
摘要翻译：癌症是发达国家的主要死因。然而，在预防，治疗和/或治疗癌症方面仍然存在高度的未满足的需求。本发明涉及通过向哺乳动物受试者施用包含其直接或间接导致表达的核酸组合物的体内基因转移载体来治疗癌症，预防癌症和/或抑制癌症生长的方法（细胞），以及 /或金属蛋白酶-3组织抑制剂（TIMP-3）的分泌。成功转导后，TIMP-3在局部（在癌细胞附近）或全身性地表达和/或分泌会抑制癌细胞的生长。还提供了含有用于给药的合适的药物悬浮液中的基因转移载体的药物试剂盒。

10. 发明申请

US20180135030A1 COMPOSITIONS USEFUL IN TREATMENT OF ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY 审中-公开
公开(公告)号：US20180135030A1
公开(公告)日：2018-05-17
申请号：US15861427
申请日：2018-01-03
申请人： The Trustees of the University of Pennsylvania
发明人： Lili Wang , James M. Wilson
IPC分类号： C12N9/10 , A61K48/00 , C12N15/86
CPC分类号： C12N9/1018 , A61K48/0058 , C12N15/86 , C12N2710/10343 , C12N2740/15043 , C12N2750/14032 , C12N2750/14033 , C12N2750/14043 , C12N2750/14071 , C12N2750/14143 , C12N2800/22 , C12Y201/03003
摘要： Non-viral delivery systems comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.

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