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61. 发明申请

WO2015101414A2 ARTIFICIAL NUCLEIC ACID MOLECULES 审中-公开
标题翻译：人工核酸分子
公开(公告)号：WO2015101414A2
公开(公告)日：2015-07-09
申请号：PCT/EP2014/003480
申请日：2014-12-30
申请人： CUREVAC GMBH
发明人： THESS, Andreas
IPC分类号： C12N15/67
CPC分类号： C12N15/85 , C12N15/67
摘要： The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3'-untranslated region element (3'-UTR) element comprising a nucleic acid sequence which is derived from the 3'-UTR of a ribosomal protein gene. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, the invention relates to the use of a 3'-UTR element comprising a nucleic acid sequence which is derived from the 3'-UTR of a ribosomal protein gene for enhancing, stabilizing and/or prolonging protein expression from a nucleic acid sequence comprising such 3'-UTR element.
摘要翻译：本发明涉及包含至少一个开放阅读框和至少一个3'-非翻译区域元件（3'-UTR）元件的人造核酸分子，其包含衍生自核糖体的3'-UTR的核酸序列蛋白质基因。本发明还涉及这种人工核酸分子在基因治疗和/或基因疫苗接种中的用途。此外，本发明涉及包含衍生自核糖体蛋白基因的3'-UTR的核酸序列的3'-UTR元件用于增强，稳定和/或延长来自包含以下的核酸序列的蛋白质表达的用途：这样的3'-UTR元件。

62. 发明申请

WO2013174409A1 REVERSIBLE IMMOBILIZATION AND/OR CONTROLLED RELEASE OF NUCLEIC ACID CONTAINING NANOPARTICLES BY (BIODEGRADABLE) POLYMER COATINGS 审中-公开
标题翻译：（可生物降解）聚合物涂层可逆稳定和/或控制含有纳米粒子的核酸的释放
公开(公告)号：WO2013174409A1
公开(公告)日：2013-11-28
申请号：PCT/EP2012/002257
申请日：2012-05-25
申请人： CUREVAC GMBH , BAUMHOF, Patrick , WENDEL, Hans-Peter , NOLTE, Andrea , WALKER, Tobias
发明人： BAUMHOF, Patrick , WENDEL, Hans-Peter , NOLTE, Andrea , WALKER, Tobias
IPC分类号： A61K47/34 , A61K9/51 , A61K47/48 , A61K48/00 , A61L27/34 , A61L27/54 , A61L27/58 , A61L31/10 , A61L31/14 , A61L31/16
CPC分类号： A61K48/0041 , A61K9/5146 , A61K9/5153 , A61K9/5192 , A61K47/34 , A61K47/645 , A61K47/6937 , A61L27/34 , A61L27/54 , A61L31/10 , A61L31/148 , A61L31/16 , A61L2300/258 , A61L2300/604 , A61L2300/606 , A61L2300/62 , A61L2300/802 , A61L2400/12 , C12N9/0069 , C12N15/88 , C12Y113/12005 , C08L67/04
摘要： The present invention relates to nanoparticles comprising nucleic acids coated with a (biodegradable) polymer for reversible immobilization and/or controlled release of the nucleic acid comprising nanoparticles. Furthermore, the present invention is directed to medical or diagnostic devices, particularly stents and implants coated by a (biodegradable) polymer with the nucleic acid comprising nanoparticles for reversible immobilization and/or controlled release. Furthermore, the present invention is directed to the use of these nanoparticles coated with a (biodegradable) polymer and to the use of medical devices and implants coated by the (biodegradable) polymer with these nucleic acid comprising nanoparticles in the prophylactic or therapeutic treatment of diseases, particularly in the prevention or treatment of restenosis, calicification, foreign body reaction, or inflammation. Additionally, the present invention is directed to a method of preparing these nucleic acid comprising nanoparticles coated with a (biodegradable) polymer and to a method for coating nucleic acid comprising nanoparticles by a (biodegradable) polymer on medical or diagnostic devices.
摘要翻译：本发明涉及包含用可生物降解的聚合物涂覆的核酸的纳米颗粒，用于可逆地固定和/或控制释放包含纳米颗粒的核酸。此外，本发明涉及医疗或诊断装置，特别是由可生物降解的聚合物涂覆的支架和植入物，其中核酸包含用于可逆固定和/或控制释放的纳米颗粒。此外，本发明涉及涂覆有（可生物降解的）聚合物的这些纳米颗粒的用途，以及使用包含（可生物降解的）聚合物的医疗装置和植入物，其中包含纳米颗粒的这些核酸在预防或治疗疾病中特别是在预防或治疗再狭窄，加压，异物反应或炎症中。另外，本发明涉及一种制备这些核酸的方法，所述核酸包含涂覆有（可生物降解的）聚合物的纳米颗粒，以及通过医学或诊断装置上的（可生物降解的）聚合物涂覆包含纳米颗粒的核酸的方法。

63. 发明申请

WO2013143700A2 ARTIFICIAL NUCLEIC ACID MOLECULES COMPRISING A 5'TOP UTR 审中-公开
标题翻译：包含5'TOP UTR的人工核酸分子
公开(公告)号：WO2013143700A2
公开(公告)日：2013-10-03
申请号：PCT/EP2013000938
申请日：2013-03-27
申请人： CUREVAC GMBH , THES ANDREAS
发明人： THES ANDREAS
IPC分类号： C12N15/67
CPC分类号： A61K48/0066 , A61K38/00 , A61K39/00 , C12N15/67 , C12N15/85 , C12N2830/50 , C12N2830/85 , C12N2840/105
摘要： The invention relates to an artificial nucleic acid molecule comprising at least one 5'UTR element which is derived from a TOP gene, at least one open reading frame and optionally at least one 3'UTR element comprising a nucleic acid sequence which is preferably derived from the 3'UTR of a gene providing a stable mRNA, such as an albumin gene, or from a variant of the 3'UTR of a gene providing a stable mRNA. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination.
摘要翻译：本发明涉及一种人造核酸分子，其包含至少一个源自TOP基因的5'UTR元件，至少一个可读框和任选的至少一个3'UTR元件，所述3'UTR元件包含优选来源于提供稳定mRNA的基因如白蛋白基因的3'UTR，或来自提供稳定mRNA的基因的3'UTR的变体。本发明还涉及这种人造核酸分子在基因治疗和/或基因疫苗接种中的用途。

64. 发明申请

WO2012019780A1 NUCLEIC ACID COMPRISING OR CODING FOR A HISTONE STEM-LOOP AND A POLY(A) SEQUENCE OR A POLYADENYLATION SIGNAL FOR INCREASING THE EXPRESSION OF AN ENCODED PROTEIN 审中-公开
标题翻译：包含或编码用于增强编码蛋白质表达的聚（A）序列或聚烯丙基化信号的核酸
公开(公告)号：WO2012019780A1
公开(公告)日：2012-02-16
申请号：PCT/EP2011/004077
申请日：2011-08-12
申请人： CUREVAC GMBH , THESS, Andreas , SCHLAKE, Thomas , PROBST, Jochen
发明人： THESS, Andreas , SCHLAKE, Thomas , PROBST, Jochen
IPC分类号： C12N15/67
CPC分类号： A61K48/0066 , A61K39/0011 , A61K2039/53 , A61K2039/575 , C07K14/435 , C12N15/67
摘要： The present application describes a coding nucleic acid sequence, particularly a messenger RNA (mRNA), comprising or coding for a histone stem-loop and a poly(A) sequence or a polyadenylation signal and the use thereof for increasing the expression of an encoded protein. It also discloses its use for the preparation of a pharmaceutical composition, especially a vaccine e.g. for the use in the treatment of tumours and cancer diseases, cardiovascular diseases, infectious diseases, autoimmune diseases or genetic diseases, or in gene therapy. The present invention further describes an in vitro transcription method, in vitro methods for increasing the expression of a protein using the nucleic acid comprising or coding for a histone stem-loop and a poly(A) sequence or a polyadenylation signal and an ex vivo and in vivo method.
摘要翻译：本申请描述了包含或编码组蛋白茎环和聚（A）序列或聚腺苷酸化信号的编码核酸序列，特别是信使RNA（mRNA），及其用途用于增加编码蛋白质的表达。它还公开了其用于制备药物组合物，特别是例如疫苗的用途。用于治疗肿瘤和癌症疾病，心血管疾病，感染性疾病，自身免疫疾病或遗传疾病，或用于基因治疗。本发明进一步描述了体外转录方法，使用包含或编码组蛋白茎环和聚（A）序列或聚腺苷酸化信号的核酸和离体的方法增加蛋白质表达的体外方法，和体内方法。

65. 发明申请

WO2011026641A9 DISULFIDE-LINKED POLYETHYLENEGLYCOL/PEPTIDE CONJUGATES FOR THE TRANSFECTION OF NUCLEIC ACIDS 审中-公开
标题翻译：二硫键连接的聚乙二醇/肽共轭物用于转移核酸
公开(公告)号：WO2011026641A9
公开(公告)日：2011-11-17
申请号：PCT/EP2010005438
申请日：2010-09-03
申请人： CUREVAC GMBH , BAUMHOF PATRICK , SCHLAKE THOMAS
发明人： BAUMHOF PATRICK , SCHLAKE THOMAS
IPC分类号： A61K47/48 , A61P43/00
CPC分类号： A61K48/0041 , A61K47/60 , A61K47/6455
摘要： The present invention is directed to an inventive polymeric carrier molecule according to generic formula (I) and variations thereof, which allows for efficient transfection of nucleic acids into cells in vivo and in vitro, a polymeric carrier cargo complex formed by a nucleic acid and the inventive polymeric carrier molecule, but also to methods of preparation of this inventive polymeric carrier molecule and of the inventive polymeric carrier cargo complex. The present invention also provides methods of application and use of this inventive polymeric carrier molecule and the inventive polymeric carrier cargo complex as a medicament, for the treatment of various diseases, and in the preparation of a pharmaceutical composition for the treatment of such diseases.
摘要翻译：本发明涉及根据通式（I）及其变体的本发明的聚合物载体分子，其允许核酸在体内和体外有效地转染入细胞，由核酸和本发明的聚合物载体分子，还涉及制备本发明的聚合物载体分子和本发明的聚合物载体货物复合物的方法。本发明还提供了本发明的聚合物载体分子和本发明的聚合物载体货物复合物作为药物，用于治疗各种疾病以及制备用于治疗这些疾病的药物组合物的应用和使用方法。

66. 发明申请

WO2011069587A1 LYOPHILIZATION OF NUCLEIC ACIDS IN LACTATE-CONTAINING SOLUTIONS 审中-公开
标题翻译：在含有LACTAT的溶液中的核酸的分解
公开(公告)号：WO2011069587A1
公开(公告)日：2011-06-16
申请号：PCT/EP2010/006789
申请日：2010-11-08
申请人： CUREVAC GMBH , MUTZKE, Thorsten , KETTERER, Thomas , VON DER MÜLBE, Florian
发明人： MUTZKE, Thorsten , KETTERER, Thomas , VON DER MÜLBE, Florian
IPC分类号： C12N15/10 , A61K48/00
CPC分类号： C12N15/1003 , C12N1/04
摘要： The present invention is directed to the lyophilization of nucleic acids in a lactate-containing solution or formulation. The present invention is particularly suitable for enhancing and improving storage and shipping capabilities of nucleic acids for multiple purposes. The present invention is furthermore directed to methods of lyophilization suitable to prepare such inventive lyophilized nucleic acids, to the use of a lactate-containing solution or formulation for the preparation of such lyophilized nucleic acids or the use of a solution containing at least one nucleic acid (sequence) and (free) lactate for lyophilization of at least one nucleic acid (sequence), to the use of such lyophilized nucleic acids in the preparation of pharmaceutical compositions, to first and second medical indications using such lyophilized nucleic acids and to kits, particularly to kit of parts, comprising such lyophilized nucleic acids.
摘要翻译：本发明涉及在含乳酸的溶液或制剂中冻干核酸。本发明特别适用于增强和改进用于多个目的的核酸的储存和运输能力。本发明还涉及适于制备本发明的冻干核酸的冷冻干燥方法，使用含乳酸盐的溶液或制剂用于制备这种冻干核酸或使用含有至少一种核酸的溶液（序列）和（游离）乳酸盐，用于冻干至少一种核酸（序列），使用这样的冻干核酸在药物组合物的制备中，使用这种冻干核酸的第一和第二种医学适应症和试剂盒，特别是包含这种冻干核酸的试剂盒。

67. 发明申请

WO2010088927A1 USE OF PEI FOR THE IMPROVEMENT OF ENDOSOMAL RELEASE AND EXPRESSION OF TRANSFECTED NUCLEIC ACIDS, COMPLEXED WITH CATIONIC OR POLYCATIONIC COMPOUNDS 审中-公开
标题翻译：使用PEI改进内源性释放和表达转移的核酸，与阳离子或多元化合物复合
公开(公告)号：WO2010088927A1
公开(公告)日：2010-08-12
申请号：PCT/EP2009/000886
申请日：2009-02-09
申请人： CUREVAC GMBH , BAUMHOF, Patrick , SCHLAKE, Thomas
发明人： BAUMHOF, Patrick , SCHLAKE, Thomas
IPC分类号： C12N15/87 , A61K48/00
CPC分类号： C12N15/87 , A61K48/0041
摘要： The present invention is directed to complexed precomplexed nucleic acids, preferably mRNAs, which have been precomplexed in a first step with PEI in an N/P ratio between 0.05 and 2, preferably in an N/P ratio between 0.1 and 1, and which have been further complexed in a second step with a cationic compound. The present invention is furthermore directed to the use of such complexed precomplexed nucleic acids for the improvement of the endosomal release of nucleic acids and optionally the improvement of expression of an encoded protein or peptide. It is also directed to compositions comprising such complexed precomplexed nucleic acids and to the use of such complexed precomplexed nucleic acids or compositions thereof for gene therapy and/or the treatment of various diseases as mentioned herein, e.g. by vaccination. The present invention is also directed to methods for preparing and administering these complexed precomplexed nucleic acids or compositions thereof and to kits, comprising these complexed precomplexed nucleic acids or compositions thereof.
摘要翻译：本发明涉及复合的预复合核酸，优选mRNA，其在第一步中已经被复合，PEI以N / P比为0.05和2，优选N / P比在0.1和1之间，其具有在第二步中与阳离子化合物进一步复合。本发明还涉及这种复合的预复合核酸用于改善核酸的内体内释放和任选地改进编码的蛋白质或肽的表达的用途。还涉及包含这种络合的预复合核酸的组合物，以及这些复合的预复合核酸或其组合物用于基因治疗和/或治疗如本文所述的各种疾病的用途，例如，通过疫苗接种。本发明还涉及用于制备和施用这些络合的预复合核酸或其组合物的方法，以及包含这些络合的预复合核酸或其组合物的试剂盒。

68. 发明申请

WO2010037539A1 COMPOSITION COMPRISING A COMPLEXED (M)RNA AND A NAKED MRNA FOR PROVIDING OR ENHANCING AN IMMUNOSTIMULATORY RESPONSE IN A MAMMAL AND USES THEREOF 审中-公开
标题翻译：包含复合（M）RNA的组合物和用于提供或增强哺乳动物的免疫应答的NANA MRNA及其用途
公开(公告)号：WO2010037539A1
公开(公告)日：2010-04-08
申请号：PCT/EP2009/007032
申请日：2009-09-30
申请人： CUREVAC GMBH , FOTIN-MLECZEK, Mariola , VOSS, Söhnke
发明人： FOTIN-MLECZEK, Mariola , VOSS, Söhnke
IPC分类号： A61K39/39 , A61P35/00 , A61K47/00
CPC分类号： A61K39/39 , A61K39/0011 , A61K2039/53 , A61K2039/55511 , Y02A50/491
摘要： The present invention relates to an immunostimulatory composition comprising a) an adjuvant component, comprising or consisting of at least one (m)RNA, complexed with a cationic or polycationic compound, and b) at least one free mRNA, encoding at least one therapeutically active protein, antigen, allergen and/or antibody, wherein the immunostimulatory composition is capable to elicit or enhance an innate and optionally an adaptive immune response in a mammal. The inventive immunostimulatory composition may be a pharmaceutical composition or a vaccine. The invention furthermore relates to a method of preparation of the inventive immunostimulatory composition. The invention also relates to the use of the inventive immunostimulatory composition or its components (for the preparation of a pharmaceutical composition or a vaccine) for the treatment of various diseases. Finally, the invention relates to kits containing the inventive immunostimulatory composition, its components and/or the pharmaceutical composition or vaccine.
摘要翻译：本发明涉及免疫刺激组合物，其包含a）佐剂组分，其包含或与由阳离子或多阳离子化合物络合的至少一种（m）RNA组成，和b）至少一种游离mRNA，其编码至少一种治疗活性蛋白质，抗原，变应原和/或抗体，其中所述免疫刺激组合物能够引发或增强哺乳动物中的先天和任选的适应性免疫应答。本发明的免疫刺激组合物可以是药物组合物或疫苗。本发明还涉及本发明的免疫刺激组合物的制备方法。本发明还涉及本发明的免疫刺激组合物或其组分（用于制备药物组合物或疫苗）用于治疗各种疾病的用途。最后，本发明涉及包含本发明的免疫刺激组合物，其组分和/或药物组合物或疫苗的试剂盒。

69. 发明申请

WO2009095226A3 NUCLEIC ACIDS COMPRISING FORMULA (NuGlXmGnNv)a AND DERIVATIVES THEREOF AS AN IMMUNOSTIMULATING AGENTS /ADJUVANTS 审中-公开
公开(公告)号：WO2009095226A3
公开(公告)日：2009-08-06
申请号：PCT/EP2009/000546
申请日：2009-01-28
申请人： CUREVAC GMBH , KRAMPS, Thomas , VOSS, Söhnke , PROBST, Jochen , HOERR, Ingmar
发明人： KRAMPS, Thomas , VOSS, Söhnke , PROBST, Jochen , HOERR, Ingmar
IPC分类号： C12N15/09 , C12N15/11 , A61K39/39
摘要： The present invention relates to nucleic acids of the general formula (I): (N u G l X m G n N v ) a and derivatives thereof as an immunostimulating agent/adjuvant and to compositions containing same, optionally comprising an additional adjuvant. The present invention furthermore relates to a pharmaceutical composition or to a vaccine, each containing nucleic acids of formula (I) above and/or derivatives thereof as an immunostimulating agent, and optionally at least one additional pharmaceutically active component, e.g. an antigenic agent. The present invention relates likewise to the use of the pharmaceutical composition or of the vaccine for the treatment of cancer diseases, infectious diseases, allergies and autoimmune diseases etc. Likewise, the present invention includes the use of nucleic acids of the general formula (I): (N u G l X m G n N v ) a and/or derivatives thereof for the preparation of a pharmaceutical composition for the treatment of such diseases.

70. 发明申请

WO2009046975A1 COMPOSITION FOR TREATING PROSTATE CANCER (PCa) 审中-公开
标题翻译：治疗前列腺癌（PCa）的组合物
公开(公告)号：WO2009046975A1
公开(公告)日：2009-04-16
申请号：PCT/EP2008/008504
申请日：2008-10-08
申请人： CUREVAC GMBH , PROBST, Jochen , HOERR, Ingmar , LANDER, Thomas
发明人： PROBST, Jochen , HOERR, Ingmar , LANDER, Thomas
IPC分类号： A61K39/00
CPC分类号： A61K39/0011 , A61K39/00 , A61K39/39 , A61K2039/53 , A61K2039/55516 , A61K2039/572 , A61K2039/575 , A61K2039/70
摘要： The present invention relates to an active (immunostimulatory) composition comprising at least one RNA, preferably an mRNA, encoding at least two (preferably different) antigens capable of eliciting an (adaptive) immune response in a mammal wherein the antigens are selected from the group consisting of PSA (Prostate-Specific Antigen), PSMA (Prostate- Specific Membrane Antigen), PSCA (Prostate Stem Cell Antigen), and STEAP (Six Transmembrane Epithelial Antigen of the Prostate). The invention furthermore relates to a vaccine comprising said active (immunostimulatory) composition, and to the use of said active (immunostimulatory) composition (for the preparation of a vaccine) and/or of the vaccine for eliciting an (adaptive) immune response for the treatment of prostate cancer (PCa), preferably of neoadjuvant and/or hormone-refractory prostate cancers, and diseases or disorders related thereto.. Finally, the invention relates to kits, particularly to kits of parts, containing the active (immunostimulatory) composition and/or the vaccine.
摘要翻译：本发明涉及包含至少一种RNA（优选mRNA）的活性（免疫刺激性）组合物，其编码至少两种（优选不同的）能够在哺乳动物中引发（适应性）免疫应答的抗原，其中所述抗原选自组由PSA（前列腺特异性抗原），PSMA（前列腺特异性膜抗原），PSCA（前列腺干细胞抗原）和STEAP（前列腺的六跨膜上皮抗原）组成。本发明还涉及包含所述活性（免疫刺激）组合物的疫苗，以及所述活性（免疫刺激性）组合物（用于制备疫苗）和/或用于引发（适应性）免疫应答的疫苗的用途治疗前列腺癌（PCa），优选新辅助和/或激素难治性前列腺癌，以及与之相关的疾病或病症。最后，本发明涉及试剂盒，特别是涉及包含活性（免疫刺激）组合物的试剂盒， /或疫苗。

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