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1. 发明公开

EP2800580A1 MEANS AND METHODS FOR TREATING OR DIAGNOSING IDH1 R132H MUTANT-POSITIVE CANCERS 有权
标题翻译： PEPTIDEFÜRDIE BEHANDLUNG UND DIORNOS VON IDH1 R132H POSITIVEN KREBSARTEN
公开(公告)号：EP2800580A1
公开(公告)日：2014-11-12
申请号：EP13700266.3
申请日：2013-01-03
申请人： Deutsches Krebsforschungszentrum , Ruprecht-Karls-Universität Heidelberg
发明人： PLATTEN, Michael , SCHUMACHER, Theresa , WICK, Wolfgang
IPC分类号： A61K38/43 , G01N33/00 , A61P35/00
CPC分类号： G01N33/57496 , A61K38/43 , A61K39/0011 , C12N9/0006 , C12Y101/01042 , G01N33/57407 , G01N33/57426 , G01N2333/904
摘要： The present invention concerns the field of tumor therapeutics and diagnostics. Specifically, it relates to a peptide comprising at least 8 amino acids in length which are present as contiguous amino acid sequence in the human Isocitratdehydrogenase Type 1 (IDH1), wherein said peptide has at least one amino acid exchange from R to H at a position corresponding to position 132, for use in preventing and/or treating cancer. Further contemplated is a medicament comprising the said peptide. Furthermore, the invention relates to a method for diagnosing cancer characterized by having a mutation in the genome of at least some cancer cells which results in the expression of a mutant IDH1 having the R132H mutation comprising the steps of contacting a blood sample of a subject suspected to suffer from such a cancer with a peptide comprising at least 10 amino acids in length which are present as contiguous amino acid sequence in the IDH1,wherein said peptide has at least one amino acid exchange from R to H at a position corresponding to position 132 for a time and under conditions which allow for specific binding of a component of the immune system to the peptide, and determining whether, or not, binding of the said component of the immune system to the peptide occurred, wherein the cancer is diagnosed if the occurrence of binding has been determined. Provided by the invention is also a kit and a device for carrying out said method.
摘要翻译：本发明涉及肿瘤治疗和诊断领域。具体地说，本发明涉及包含长度为至少8个氨基酸的肽，其在人类异柠檬酸脱氢酶1型（IDH1）中作为连续氨基酸序列存在，其中所述肽在位置上具有至少一个R至H的氨基酸交换对应于位置132，用于预防和/或治疗癌症。进一步考虑的是包含所述肽的药物。此外，本发明涉及一种用于诊断癌症的方法，其特征在于在至少一些癌细胞的基因组中具有突变，其导致具有R132H突变的突变体IDH1的表达，包括以下步骤：使被怀疑的受试者的血液样品患有长度为至少10个氨基酸的肽的这种癌症，其在IDH1中作为连续氨基酸序列存在，其中所述肽在对应于位置132的位置具有至少一个从R至H的氨基酸交换在一段时间和在允许免疫系统的组分与肽的特异性结合的条件下，以及确定是否发生免疫系统的所述组分与肽的结合，其中如果确定了结合的发生。本发明还提供一种用于执行所述方法的套件和装置。

2. 发明公开

EP3501532A2 THERAPEUTIC COMPOSITIONS INCLUDING FRATAXIN, LACTOFERRIN, AND MITOCHONDRIAL ENERGY GENERATING ENZYMES, AND USES THEREOF 审中-公开
公开(公告)号：EP3501532A2
公开(公告)日：2019-06-26
申请号：EP18210159.2
申请日：2015-05-27
申请人： Stealth BioTherapeutics Corp
发明人： WILSON, D. Travis
IPC分类号： A61K38/07 , A61K31/353
CPC分类号： A61K38/10 , A61K38/05 , A61K38/06 , A61K38/07 , A61K38/08 , A61K38/13 , A61K38/1709 , A61K38/40 , A61K38/44 , A61K38/443 , A61K38/45 , A61K38/46 , A61K38/51 , A61K38/53 , A61K47/645 , C07K5/06078 , C07K5/06095 , C07K5/06156 , C07K5/0812 , C07K5/0815 , C07K5/0817 , C07K5/0821 , C07K5/1016 , C07K5/1021 , C12Y101/01037 , C12Y101/01042 , C12Y102/04002 , C12Y103/05001 , C12Y106/05003 , C12Y109/03001 , C12Y110/02002 , C12Y203/03001 , C12Y306/03014 , C12Y402/01002 , C12Y402/01003 , C12Y602/01004 , C12Y604/01001
摘要： Disclosed herein are methods and compositions for the treatment and/or prevention of diseases or conditions comprising administration of a therapeutic biological molecule, and/or naturally or artificially occurring derivatives, analogues, or pharmaceutically acceptable salts thereof, alone or in combination with one or more active agents ( e.g., an aromatic-cationic peptide). The present technology provides compositions related to aromatic-cationic peptides linked to a therapeutic biological molecule and uses of the same. In some embodiments, the aromatic-cationic peptide comprises 2',6'-dimethyl-Tyr-D-Arg-Phe-Lys-NH 2 , Phe-D-Arg-Phe-Lys-NH 2 , or D-Arg-2',6'-Dmt-Lys-Phe-NH 2 .

3. 发明公开

EP2406389A4 METHODS AND COMPOSITIONS FOR CELL-PROLIFERATION-RELATED DISORDERS 有权转让
标题翻译：方法和组合物细胞增殖相关疾病
公开(公告)号：EP2406389A4
公开(公告)日：2013-01-16
申请号：EP10751525
申请日：2010-03-12
申请人： AGIOS PHARMACEUTICALS INC
发明人： DANG LENNY , FANTIN VALERIA , GROSS STEFAN , JANG HYUN GYUNG , JIN SHENGFANG , SALITURO FRANCESCO G , SAUNDERS JEFFREY O , SU SHINSAN , YEN KATHARINE
IPC分类号： G01N33/574 , A61P35/00 , C12Q1/32
CPC分类号： A61B5/055 , A61K31/41 , A61K31/426 , A61K45/06 , C12N15/1137 , C12N2310/14 , C12Q1/32 , C12Q1/6886 , C12Y101/01042 , G01N33/574 , G06F19/328 , Y02A90/24 , A61K2300/00
摘要： Methods of treating and evaluating subjects having neoactive mutants are described herein.
摘要翻译：治疗和评估其新活性突变体中描述的对象的方法。

4. 发明公开

EP2846821A4 HYDRODYNAMIC METHODS FOR DELIVERING FLUIDS TO KIDNEY TISSUES AND RELATED MATERIALS AND METHODS 有权
标题翻译：水动力过程液体肾组织及相关材料和方法的分布
公开(公告)号：EP2846821A4
公开(公告)日：2016-03-16
申请号：EP13784903
申请日：2013-05-03
申请人： UNIV INDIANA RES & TECH CORP
发明人： BACALLAO ROBERT , ATKINSON SIMON , RHODES GEORGE , CORRIDON PETER
IPC分类号： A61K38/18 , C12N15/00
CPC分类号： A61K48/0075 , A61K9/0019 , A61K38/443 , A61K38/45 , A61K48/0058 , C12N7/00 , C12N2710/10041 , C12Y101/01042 , C12Y208/02
摘要： The present invention provides methods and materials useful delivering liquids, including liquids comprising nucleic acid molecules into cells. In particular, the present invention provides methods for delivering saline solution, exogenous compositions, and isolated vectors to kidney cells, using the renal vein as a guide and under hydrodynamic pressure. The delivery methods and materials herein are useful to research, prognose, ameliorate symptoms of kidney injury, and treat kidney pathologies.
摘要翻译：本发明提供的方法和材料有用输送液体，液体包括包含核酸分子进入细胞。特别地，本发明提供了用于递送盐水溶液，外源的组合物，和隔离的向量肾细胞，使用肾静脉作为引导和下流体动压力的方法。交付方法和材料的研究，预测有用，改善肾损伤的症状，治疗肾脏疾病。

5. 发明公开

EP2846821A1 HYDRODYNAMIC METHODS FOR DELIVERING FLUIDS TO KIDNEY TISSUES AND RELATED MATERIALS AND METHODS 有权
标题翻译：水动力过程液体肾组织及相关材料和方法的分布
公开(公告)号：EP2846821A1
公开(公告)日：2015-03-18
申请号：EP13784903.0
申请日：2013-05-03
申请人： Indiana University Research&Technology Corporation
发明人： BACALLAO, Robert , ATKINSON, Simon , RHODES, George , CORRIDON, Peter
IPC分类号： A61K38/18 , C12N15/00
CPC分类号： A61K48/0075 , A61K9/0019 , A61K38/443 , A61K38/45 , A61K48/0058 , C12N7/00 , C12N2710/10041 , C12Y101/01042 , C12Y208/02
摘要： The present invention provides methods and materials useful delivering liquids, including liquids comprising nucleic acid molecules into cells. In particular, the present invention provides methods for delivering saline solution, exogenous compositions, and isolated vectors to kidney cells, using the renal vein as a guide and under hydrodynamic pressure. The delivery methods and materials herein are useful to research, prognose, ameliorate symptoms of kidney injury, and treat kidney pathologies.

6. 发明公开

EP2406389A1 METHODS AND COMPOSITIONS FOR CELL-PROLIFERATION-RELATED DISORDERS 有权转让
标题翻译：细胞增殖相关疾病的方法和组合物
公开(公告)号：EP2406389A1
公开(公告)日：2012-01-18
申请号：EP10751525.6
申请日：2010-03-12
申请人： Agios Pharmaceuticals, Inc.
发明人： DANG, Lenny , FANTIN, Valeria , GROSS, Stefan , JANG, Hyun Gyung , JIN, Shengfang , SALITURO, Francesco G. , SAUNDERS, Jeffrey, O. , SU, Shinsan , YEN, Katharine
IPC分类号： C12Q1/32 , A61P35/00
CPC分类号： A61B5/055 , A61K31/41 , A61K31/426 , A61K45/06 , C12N15/1137 , C12N2310/14 , C12Q1/32 , C12Q1/6886 , C12Y101/01042 , G01N33/574 , G06F19/00 , G06F19/328 , Y02A90/24 , Y02A90/26 , A61K2300/00
摘要： Methods of treating and evaluating subjects having neoactive mutants are described herein.
摘要翻译：本文描述了治疗和评估具有新活性突变体的受试者的方法。

7. 发明公开

EP3365686A1 CYSTEINE REACTIVE PROBES AND USES THEREOF 审中-公开
公开(公告)号：EP3365686A1
公开(公告)日：2018-08-29
申请号：EP16858391.2
申请日：2016-10-21
申请人： The Scripps Research Institute
发明人： CRAVATT, Benjamin F. , BACKUS, Keriann M. , CORREIA, Bruno E. , BLEWETT, Megan M. , TEIJARO, John R.
IPC分类号： G01N33/68 , G01N33/536 , G01N33/532
CPC分类号： G01N33/6845 , C12N9/0006 , C12N9/1007 , C12N9/6472 , C12Y101/01042 , C12Y201/01023 , C12Y304/22061 , C12Y304/22063 , G01N33/5005 , G01N33/5008 , G01N33/6842
摘要： Disclosed herein are methods, compositions, probes, polypeptides, assays, and kits for identifying a cysteine containing protein as a binding target for a small molecule fragment. Also disclosed herein are methods, compositions, and probes for mapping a biologically active cysteine site on a protein and screening a small molecule fragment for interaction with a cysteine containing protein.

8. 发明公开

EP2717882A4 RNA INTERFERENCE MEDIATED INHIBITION OF ISOCITRATE DEHYDROGENASE (IDH1) GENE EXPRESSION 审中-公开
标题翻译： RNA-INTERFERENZ-VERMITTELTE HEMMUNG DER ISOCITRAT-DEHYDROGENASE（IDH1）-GENEXPRESSION
公开(公告)号：EP2717882A4
公开(公告)日：2015-03-25
申请号：EP12796872
申请日：2012-06-01
申请人： SIRNA THERAPEUTICS INC
发明人： MA YONG , BROWN DUNCAN , BARTZ STEVEN
IPC分类号： A61K31/7088 , C07H21/02
CPC分类号： C12N15/1137 , C07H21/02 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/332 , C12N2310/3521 , C12N2310/3533 , C12Y101/01042 , C12N2310/322
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of IDH1 and mutant IDH1 gene expression and/or activity, and/or modulate an IDH1 or mutant IDH1 gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules, including small nucleic acid molecules such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules, that are capable of mediating or that mediate RNA interference (RNAi) against IDH1 or mutant IDH1 gene expression.
摘要翻译：本发明涉及用于研究，诊断和治疗响应于IDH1和突变体IDH1基因表达和/或活性的调节和/或调节IDH1或突变体的性状，疾病和病症的化合物，组合物和方法 IDH1基因表达途径。具体地说，本发明涉及双链核酸分子，包括小核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA）分子，其能够介导或介导针对IDH1或突变体IDH1基因表达的RNA干扰（RNAi）。

9. 发明公开

EP2717882A1 RNA INTERFERENCE MEDIATED INHIBITION OF ISOCITRATE DEHYDROGENASE (IDH1) GENE EXPRESSION 审中-公开
标题翻译：异柠檬酸脱氢酶（IDH1）的基因表达的RNA干扰介导的抑制
公开(公告)号：EP2717882A1
公开(公告)日：2014-04-16
申请号：EP12796872.5
申请日：2012-06-01
申请人： Merck Sharp & Dohme Corp.
发明人： MA, Yong , BROWN, Duncan , BARTZ, Steven
IPC分类号： A61K31/7088 , C07H21/02
CPC分类号： C12N15/1137 , C07H21/02 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/332 , C12N2310/3521 , C12N2310/3533 , C12Y101/01042 , C12N2310/322
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of IDH1 and mutant IDH1 gene expression and/or activity, and/or modulate an IDH1 or mutant IDH1 gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules, including small nucleic acid molecules such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules, that are capable of mediating or that mediate RNA interference (RNAi) against IDH1 or mutant IDH1 gene expression.

10. 发明公开

EP3148565A4 THERAPEUTIC COMPOSITIONS INCLUDING FRATAXIN, LACTOFERRIN, AND MITOCHONDRIAL ENERGY GENERATING ENZYMES, AND USES THEREOF 审中-公开
公开(公告)号：EP3148565A4
公开(公告)日：2018-06-06
申请号：EP15799189
申请日：2015-05-27
申请人： STEALTH BIOTHERAPEUTICS CORP
发明人： WILSON D TRAVIS
IPC分类号： A61K38/07 , A61K31/353
CPC分类号： A61K38/10 , A61K38/05 , A61K38/06 , A61K38/07 , A61K38/08 , A61K38/13 , A61K38/1709 , A61K38/40 , A61K38/44 , A61K38/443 , A61K38/45 , A61K38/46 , A61K38/51 , A61K38/53 , A61K47/48315 , A61K47/645 , C07K5/06078 , C07K5/06095 , C07K5/06156 , C07K5/0812 , C07K5/0815 , C07K5/0817 , C07K5/0821 , C07K5/1016 , C07K5/1021 , C12Y101/01037 , C12Y101/01042 , C12Y102/04002 , C12Y103/05001 , C12Y106/05003 , C12Y109/03001 , C12Y110/02002 , C12Y203/03001 , C12Y306/03014 , C12Y402/01002 , C12Y402/01003 , C12Y602/01004 , C12Y604/01001
摘要： Disclosed herein are methods and compositions for the treatment and/or prevention of diseases or conditions comprising administration of a therapeutic biological molecule, and/or naturally or artificially occurring derivatives, analogues, or pharmaceutically acceptable salts thereof, alone or in combination with one or more active agents (e.g., an aromatic-cationic peptide). The present technology provides compositions related to aromatic-cationic peptides linked to a therapeutic biological molecule and uses of the same. In some embodiments, the aromatic-cationic peptide comprises 2′,6′-dimethyl-Tyr-D-Arg-Phe-Lys-NH2, Phe-D-Arg-Phe-Lys-NH2, or D-Arg-2′,6′-Dmt-Lys-Phe-NH2.

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