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1. 发明申请

WO2015153357A1 COMPOSITIONS AND METHODS FOR IMPROVING CARDIAC FUNCTION 审中-公开
标题翻译：用于改善心脏功能的组合物和方法
公开(公告)号：WO2015153357A1
公开(公告)日：2015-10-08
申请号：PCT/US2015/023066
申请日：2015-03-27
申请人： BEAT BIOTHERAPEUTICS CORP. , UNIVERSITY OF WASHINGTON , MAHAIRAS, Gregory G. , KRANDA, Michael L. , MURRY, Charles E. , REGNIER, Michael , REINECKE, Hans , ODOM, Guy L. , CHAMBERLAIN, Jeffrey S.
发明人： MAHAIRAS, Gregory G. , KRANDA, Michael L. , MURRY, Charles E. , REGNIER, Michael , REINECKE, Hans , ODOM, Guy L. , CHAMBERLAIN, Jeffrey S.
IPC分类号： C12N9/02
CPC分类号： C12N9/0093 , C12Y117/04001 , C12Y117/04002
摘要： The invention described herein relates to compositions, including vectors, comprising a polynucleotide sequence encoding one or both of a ribonucleotide reductase subunit M1 (RRM1) and a ribonucleotide reductase subunit M2 (RRM2), and their use to increase cardiac function, and treat or prevent heart disease and other cardiac conditions.
摘要翻译：本文描述的本发明涉及包含载体的组合物，其包含编码核糖核苷酸还原酶亚基M1（RRM1）和核糖核苷酸还原酶亚基M2（RRM2）之一或两者的多核苷酸序列，以及它们用于增加心脏功能，以及治疗或预防心脏病等心脏病。

2. 发明申请

WO2016115543A2 NOVEL MICRO-DYSTROPHINS AND RELATED METHODS OF USE 审中-公开
标题翻译：新型微晶和相关使用方法
公开(公告)号：WO2016115543A2
公开(公告)日：2016-07-21
申请号：PCT/US2016/013733
申请日：2016-01-15
申请人： UNIVERSITY OF WASHINGTON
发明人： CHAMBERLAIN, Jeffrey S. , RAMOS, Julian , HAUSCHKA, Stephen D.
IPC分类号： A61K48/00 , C07H21/04
CPC分类号： C07K14/4708 , A61K38/00 , A61K48/0058 , A61P21/00 , C07H21/04 , C07K14/4707 , C07K14/4716 , C07K2319/30 , C12N15/85 , C12N2750/14143 , C12N2830/008
摘要： Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.
摘要翻译：提供了包括微型肌营养不良蛋白基因的核苷酸序列。微营养不良蛋白基因可以可操作地连接到调节盒。还提供了治疗具有或有发展肌营养不良症，肌肉减少症，心脏病或恶病质风险的受试者的方法。所述方法可以包括向受试者施用包含微型肌营养不良蛋白基因和递送载体的药物组合物。此外，所述方法可以包括施用具有杜兴肌营养不良或贝克肌营养不良症的受试者的药物组合物。

3. 发明申请

WO2016144968A1 RELAXIN THERAPY FOR DISORDERS OF THE DIAPHRAGM 审中-公开
标题翻译：泄漏治疗方法
公开(公告)号：WO2016144968A1
公开(公告)日：2016-09-15
申请号：PCT/US2016/021389
申请日：2016-03-08
申请人： UNIVERSITY OF WASHINGTON , DMD THERAPEUTICS
发明人： CHAMBERLAIN, Jeffrey S. , BENGTSSON, Niclas , BERENSON, Ronald
IPC分类号： G01N33/53
CPC分类号： A61K38/2221 , A61K45/06
摘要： Methods for treating a subject having diaphragmatic weakness or a condition associated with diaphragmatic weakness are provided. Methods for prophylactically treating a subject at risk of developing diaphragmatic weakness or at risk of developing a condition associated with diaphragmatic weakness are also provided. The methods for treating a subject having, or at risk of developing, diaphragmatic weakness or a condition associated with diaphragmatic weakness may include administering an activator of relaxin/insulin-like family peptide receptor 1 (RXFP1) to the subject. The methods for treating a subject having, or at risk of developing, diaphragmatic weakness or a condition associated with diaphragmatic weakness may also include administering a relaxin, a relaxin analog, a prorelaxin, a prorelaxin analog, or combinations thereof to the subject.
摘要翻译：提供了治疗具有膈肌无力或与膈肌无力相关的病症的受试者的方法。还提供了用于预防性治疗处于发展膈肌无力或有发展与膈肌虚弱有关的病症的风险的受试者的方法。用于治疗患有或具有膈肌虚弱的风险或与膈肌虚弱有关的疾病的受试者的方法可包括向受试者施用松弛素/胰岛素样家族肽受体1（RXFP1）的激活剂。用于治疗具有或具有膈肌无力或与膈肌无力相关的病症的风险的受试者的方法还可以包括向受试者施用松弛素，松弛素类似物，促松弛素类似物或前释放激素类似物或其组合。

4. 发明公开

EP4373949A2 GENERATION OF LARGE PROTEINS BY CO-DELIVERY OF MULTIPLE VECTORS 审中-公开
公开(公告)号：EP4373949A2
公开(公告)日：2024-05-29
申请号：EP22846678.5
申请日：2022-07-22
申请人： University of Washington
发明人： CHAMBERLAIN, Jeffrey S. , TASFAOUT, Hichem
IPC分类号： C12N15/86 , C12N15/861 , C12N9/22 , C07K14/705
CPC分类号： C12N15/86 , C12N2750/1414320130101 , C12N2830/4220130101 , C12N2840/4420130101 , C07K2319/9220130101 , C07K14/4707 , C07K14/4708

5. 发明申请

WO2022216988A2 ARTIFICIAL REGULATORY CASSETTES FOR MUSCLE-SPECIFIC GENE EXPRESSION 审中-公开
公开(公告)号：WO2022216988A2
公开(公告)日：2022-10-13
申请号：PCT/US2022/023915
申请日：2022-04-07
申请人： UNIVERSITY OF WASHINGTON
发明人： CHAMBERLAIN, Jeffrey S. , HAUSCHKA, Stephen D.
IPC分类号： A61P21/00 , C12N15/66 , C12N15/67 , C12N15/85 , C12N15/861 , C12N15/63 , C12N2830/008
摘要： A library of artificial muscle-specific expression cassettes (MSECs) for muscle-specific gene expression is described. Different members of the library can be selected for varied transcription levels in different muscle cell types, for different research or therapeutic purposes. MSECs within the library can be used to develop treatments for muscle-related disorders.

6. 发明公开

EP4319874A2 ARTIFICIAL REGULATORY CASSETTES FOR MUSCLE-SPECIFIC GENE EXPRESSION 审中-公开
公开(公告)号：EP4319874A2
公开(公告)日：2024-02-14
申请号：EP22785484.1
申请日：2022-04-07
申请人： University of Washington
发明人： CHAMBERLAIN, Jeffrey S. , HAUSCHKA, Stephen D.
IPC分类号： A61P21/00 , C12N15/66 , C12N15/67 , C12N15/85 , C12N15/861

7. 发明公开

EP3245291A2 NOVEL MICRO-DYSTROPHINS AND RELATED METHODS OF USE 审中-实审
标题翻译：新型微型营养因子及相关使用方法
公开(公告)号：EP3245291A2
公开(公告)日：2017-11-22
申请号：EP16738022.9
申请日：2016-01-15
申请人： University of Washington
发明人： CHAMBERLAIN, Jeffrey S. , RAMOS, Julian , HAUSCHKA, Stephen D.
IPC分类号： C12N15/12 , C07K14/47 , A61K48/00
CPC分类号： C07K14/4708 , A61K38/00 , A61K48/0058 , A61P21/00 , C07H21/04 , C07K14/4707 , C07K14/4716 , C07K2319/30 , C12N15/85 , C12N2750/14143 , C12N2830/008
摘要： Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

8. 发明申请

WO2023004125A2 GENERATION OF LARGE PROTEINS BY CO-DELIVERY OF MULTIPLE VECTORS 审中-公开
公开(公告)号：WO2023004125A2
公开(公告)日：2023-01-26
申请号：PCT/US2022/038032
申请日：2022-07-22
申请人： UNIVERSITY OF WASHINGTON
发明人： CHAMBERLAIN, Jeffrey S. , TASFAOUT, Hichem
IPC分类号： C12N15/86 , C12N15/861 , C12N9/22 , C07K14/705 , C07K14/47 , C12N2750/14143 , C12N2830/42 , C12N2840/44 , C12P21/02
摘要： Provided herein are methods and compositions for delivering large proteins to a subject in need thereof for treatment of a disease or disorder. In certain embodiments, the methods and compositions described herein are useful in the delivery of large proteins to subjects using a protein expression system comprising at first and second AAV vector for the treatment of muscular or neuromuscular disease or disorders.

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