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1. 发明申请

WO2010115046A2 VACCINE COMPOSITIONS AND METHODS OF USE THEREOF 审中-公开
标题翻译：疫苗组合物及其使用方法
公开(公告)号：WO2010115046A2
公开(公告)日：2010-10-07
申请号：PCT/US2010029694
申请日：2010-04-01
申请人： UNIV MIAMI , DAFTARIAN PIROUZ MOHAMMAD , SERAFINI PAOLO , LEMMON VANCE PAUL , LI WEI , KAIFER ANGEL , BLOMBERG BONNIE BETH , PEREZ VICTOR L
发明人： DAFTARIAN PIROUZ MOHAMMAD , SERAFINI PAOLO , LEMMON VANCE PAUL , LI WEI , KAIFER ANGEL , BLOMBERG BONNIE BETH , PEREZ VICTOR L
IPC分类号： A61K39/145
CPC分类号： A61K39/0011 , A61K39/12 , A61K39/145 , A61K2039/53 , A61K2039/55516 , A61K2039/55555 , A61K2039/645 , C08G83/006 , C12N2760/16022 , C12N2760/16034 , G01N2333/70539
摘要： Nanoparticle -based vaccines, compositions, kits and methods are used for the effective delivery of one or more antigens in vivo for vaccination and antibody (e.g., monoclonal antibody) production, and for the effective delivery of peptides, proteins, siRNA, RNA or DNA to PAPCs or MHC class II positive cells (e.g. tumor cells). Antigens may be, for example, DNA that results in expression of the gene of interest and induction of a robust and specific immune response to the expressed protein in a subject (e.g., mammal). Antigens may also be immunogenic peptides or polypeptides that are processed and presented. In one embodiment, a nanoparticle -based method to deliver antigens in vivo as described herein includes injection of a vaccine composed of a DNA encoding at least one antigen, or at least one antigenic peptide or polypeptide conjugated to a charged dendrimer (e.g., PADRE-derivatized dendrimer) that is also conjugated to a T helper epitope (e.g., PADRE). Negatively-charged plasmids bind naturally to a positively-charged PADRE-dendrimer, while peptide or polypeptide antigens can be chemically linked to the PADRE-dendrimer if they are not negatively-charged. Alternatively, negatively-charged dendrimers may be used. The compositions, kits, vaccines and methods described herein have both prophylactic and treatment applications, i.e., can be used as a prophylactic to prevent onset of a disease or condition in a subject, as well as to treat a subject having a disease or condition. A vaccine as described herein can be used to mount an immune response against any infectious pathogen or cancer.
摘要翻译：基于纳米颗粒的疫苗，组合物，试剂盒和方法用于体内有效递送一种或多种抗原用于疫苗接种和抗体（例如单克隆抗体）生产以及有效递送肽，蛋白质，siRNA，RNA或DNA 到PAPC或MHC II类阳性细胞（例如肿瘤细胞）。例如，抗原可以是导致感兴趣基因表达并诱导受试者（例如哺乳动物）中所表达的蛋白质的强烈和特异性免疫应答的DNA。抗原也可以是经过处理和呈递的免疫原性肽或多肽。在一个实施方案中，如本文所述的体内递送抗原的基于纳米粒子的方法包括注射由编码至少一种抗原的DNA组成的疫苗或缀合至带电荷的树状聚体的至少一种抗原性肽或多肽（例如PADRE- 衍生的树枝状聚合物），其也与T辅助表位（例如，PADRE）缀合。带负电荷的质粒自然结合带正电荷的PADRE-树状聚体，而肽或多肽抗原可以化学连接至PADRE-树状聚合物（如果它们不带负电荷的话）。或者，可以使用带负电的树枝状聚合物。本文所述的组合物，试剂盒，疫苗和方法具有预防和治疗应用，即可用作预防以预防对象中疾病或病症的发作，以及治疗患有疾病或病症的对象。如本文所述的疫苗可用于产生针对任何感染性病原体或癌症的免疫应答。

2. 发明申请

WO2010115046A3 VACCINE COMPOSITIONS AND METHODS OF USE THEREOF 审中-公开
公开(公告)号：WO2010115046A3
公开(公告)日：2010-10-07
申请号：PCT/US2010/029694
申请日：2010-04-01
申请人： UNIVERSITY OF MIAMI , DAFTARIAN, Pirouz, Mohammad , SERAFINI, Paolo , LEMMON, Vance, Paul , LI, Wei , KAIFER, Angel , BLOMBERG, Bonnie, Beth , PEREZ, Victor, L.
发明人： DAFTARIAN, Pirouz, Mohammad , SERAFINI, Paolo , LEMMON, Vance, Paul , LI, Wei , KAIFER, Angel , BLOMBERG, Bonnie, Beth , PEREZ, Victor, L.
IPC分类号： A61K39/38
摘要： Nanoparticle -based vaccines, compositions, kits and methods are used for the effective delivery of one or more antigens in vivo for vaccination and antibody (e.g., monoclonal antibody) production, and for the effective delivery of peptides, proteins, siRNA, RNA or DNA to PAPCs or MHC class II positive cells (e.g. tumor cells). Antigens may be, for example, DNA that results in expression of the gene of interest and induction of a robust and specific immune response to the expressed protein in a subject (e.g., mammal). Antigens may also be immunogenic peptides or polypeptides that are processed and presented. In one embodiment, a nanoparticle -based method to deliver antigens in vivo as described herein includes injection of a vaccine composed of a DNA encoding at least one antigen, or at least one antigenic peptide or polypeptide conjugated to a charged dendrimer (e.g., PADRE-derivatized dendrimer) that is also conjugated to a T helper epitope (e.g., PADRE). Negatively-charged plasmids bind naturally to a positively-charged PADRE-dendrimer, while peptide or polypeptide antigens can be chemically linked to the PADRE-dendrimer if they are not negatively-charged. Alternatively, negatively-charged dendrimers may be used. The compositions, kits, vaccines and methods described herein have both prophylactic and treatment applications, i.e., can be used as a prophylactic to prevent onset of a disease or condition in a subject, as well as to treat a subject having a disease or condition. A vaccine as described herein can be used to mount an immune response against any infectious pathogen or cancer.

3. 发明申请

WO2010135394A1 COMPOSITIONS, KITS AND METHODS FOR IN VITRO ANTIGEN PRESENTATION, ASSESSING VACCINE EFFICACY, AND ASSESSING IMMUNOTOXICITY OF BIOLOGICS AND DRUGS 审中-公开
标题翻译：用于体外抗原介绍的组合物，组合物和方法，评估疫苗效力和评估生物学和药物的免疫毒性
公开(公告)号：WO2010135394A1
公开(公告)日：2010-11-25
申请号：PCT/US2010/035355
申请日：2010-05-19
申请人： UNIVERSITY OF MIAMI , DAFTARIAN, Pirouz, Mohammad , SERAFINI, Paolo , LEMMON, Vance, Paul , KAIFER, Angel , BLOMBERG, Bonnie, Beth , CHOWDHURY, Raquibul , KENYON, Norma
发明人： DAFTARIAN, Pirouz, Mohammad , SERAFINI, Paolo , LEMMON, Vance, Paul , KAIFER, Angel , BLOMBERG, Bonnie, Beth , CHOWDHURY, Raquibul , KENYON, Norma
IPC分类号： C12Q1/00
CPC分类号： G01N33/54346 , C12N15/87 , C12N2810/85 , G01N33/505 , G01N33/56977 , G01N33/6866 , G01N2333/57 , G01N2333/70539 , G01N2500/04
摘要： Compositions, assays, kits, methods and platforms for delivering an antigen (peptides, proteins) or a nucleic acid encoding an antigen to professional APCs (PAPCs) are described which are ued in assessing the efficacy of a vaccine (e g, a cytotoxic T lymphocyte (CTL) response to a particular antigen) or other therapy or intervention (cell-based therapy, adjuvant therapy, etc ) The composition, kits, assays and methods involve the combined use of MHC targeting, universal DR binding peptides (e g, PADRE, HA) with charged (e g, positively-charged) highly branched polymeric dendrimers (e g, PAMAM and other dendrimers) as vehicles for the targeted delivery of nucleic acids, peptides, biologies, drugs, or polypeptides to APCs.
摘要翻译：描述了用于将抗原（肽，蛋白质）或编码抗原的核酸递送到专业APC（PAPC）的组合物，测定法，试剂盒，方法和平台，其用于评估疫苗的功效（例如，细胞毒性T淋巴细胞（CTL）对特定抗原的反应）或其他治疗或干预（基于细胞的疗法，辅助治疗等）组合物，试剂盒，测定和方法涉及MHC靶向，通用DR结合肽（例如PADRE， HA），其具有带电（例如，带正电荷的）高度支化的聚合树枝状大分子（例如，PAMAM和其它树枝状大分子）作为用于向APC的靶向递送核酸，肽，生物制品，药物或多肽的载体。

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