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    • 1. 发明申请
    • POLYNUCLEOTIDE THERAPY
    • 多核苷酸治疗
    • WO2007147011A2
    • 2007-12-21
    • PCT/US2007/071137
    • 2007-06-13
    • BAYHILL THERAPEUTICS, INC.GARREN, HidekiLEVITEN, MichaelSOLVASON, Nanette
    • GARREN, HidekiLEVITEN, MichaelSOLVASON, Nanette
    • A61K48/00
    • C12N15/85A61K39/0008A61K48/00A61K2039/53
    • This invention provides methods of treating an autoimmune disease in a subject associated with one or more self-protein(s), polypeptide(s), or peptide(s) present in the subject non-physiologically comprising administering to the subject: a self-vector comprising an immunosuppressive vector backbone and a polynucleotide encoding the self-protein(s), polypeptide(s) or peptide(s) associated with the autoimmune disease; and a divalent cation at a concentration greater than physiological levels. Administration of the self-vector comprising a polynucleotide encoding the self-protein(s), polypeptide(s) or peptide(s) modulates an immune response to the self-protein(s), polypeptide(s) or peptide(s) expressed from administration of the self-vector. This invention further provides a method of treating multiple sclerosis by administering a self-vector comprising a BHT-1 vector backbone, for example, self-vector BHT-3009 encoding human myelin basic protein (MBP). The invention also provides a pharmaceutical composition comprising: a BHT-1 vector backbone and a polynucleotide encoding one or more self-protein(s), polypeptide(s), or peptide(s) associated with an autoimmune disease; and a divalent cation at concentrations greater than physiological levels. This invention further provides a pharmaceutical composition comprising a self-vector comprising a BHT-1 vector backbone, for example, self-vector BHT-3009 encoding human myelin basic protein (MBP), and methods of administering a BHT-1 self-vector, for example BHT-3009, to a subject.
    • 本发明提供了治疗受试者中与存在于受试者中的一种或多种自身蛋白,多肽或多肽相关的受试者的自身免疫性疾病的方法,所述自身蛋白,多肽或肽非生理学上包括对受试者施用: 包含免疫抑制载体骨架和编码与自身免疫疾病相关的自身蛋白,多肽或肽的多核苷酸的载体; 和浓度大于生理水平的二价阳离子。 施用包含编码自身蛋白,多肽或多肽的多核苷酸的自身载体调节对所表达的自身蛋白,多肽或多肽的免疫应答 从自我向量的管理。 本发明还提供了通过施用包含BHT-1载体骨架的自身载体,例如编码人髓磷脂碱性蛋白(MBP)的自身载体BHT-3009来治疗多发性硬化症的方法。 本发明还提供药物组合物,其包含:BHT-1载体骨架和编码与自身免疫疾病相关的一种或多种自身蛋白质,多肽或肽的多核苷酸; 和浓度大于生理水平的二价阳离子。 本发明进一步提供了包含BHT-1载体主链的自身载体的药物组合物,例如编码人髓磷脂碱性蛋白(MBP)的自身载体BHT-3009,以及施用BHT-1自身载体的方法, 例如BHT-3009。
    • 5. 发明申请
    • COMPOSITIONS AND METHODS FOR TREATMENT OF AUTOIMMUNE DISEASE
    • 用于治疗自身免疫疾病的组合物和方法
    • WO2007044394A2
    • 2007-04-19
    • PCT/US2006/038776
    • 2006-10-04
    • BAYHILL THERAPEUTICS, INC.SOLVASON, NanetteLEVITEN, MichaelGARREN, Hideki
    • SOLVASON, NanetteLEVITEN, MichaelGARREN, Hideki
    • A61K48/00
    • A61K39/0008A61K39/0007A61K2039/55561A61K2039/55566
    • Disclosed are improved methods for the treatment or prevention of an autoimmune disease comprising administration of a modified self-vector encoding and capable of expressing a self-polypeptide that includes one or more pathogenic epitopes associated with the autoimmune disease. The improved method of the present invention includes the administration to a subject of a modified self-vector or self-vectors comprising a polynucleotide encoding a self-polypeptide. In one aspect, the method includes a modified self-vector that allows for increased expression of the self-polypeptide associated with an autoimmune disease in a host cell relative to the unmodified vector. In another, non-mutually exclusive aspect, the method includes a modified self-vector that allows for a secreted autoantigen associated with an autoimmune disease to be encoded as a non-secreted self-polypeptide.
    • 公开了用于治疗或预防自身免疫疾病的改进方法,包括施用编码并能够表达包含与自身免疫疾病相关的一个或多个病原性表位的自身多肽的修饰的自身载体。 本发明的改进方法包括向受试者施用修饰的自身载体或包含编码自身多肽的多核苷酸的自身载体。 在一个方面,该方法包括修饰的自身载体,其允许相对于未修饰的载体增加宿主细胞中与自身免疫疾病相关的自身多肽的表达。 在另一方面,非相互排斥的方面,该方法包括允许与自身免疫疾病相关的分泌的自身抗原被编码为非分泌的自身多肽的修饰的自身载体。
    • 6. 发明公开
    • POLYNUCLEOTIDE THERAPY
    • 多核苷酸治疗
    • EP2034833A2
    • 2009-03-18
    • EP07784426.4
    • 2007-06-13
    • Bayhill Therapeutics, Inc.
    • GARREN, HidekiLEVITEN, MichaelSOLVASON, Nanette
    • A01N43/04A01N63/00C12N15/00C12N15/63C07H21/04
    • C12N15/85A61K39/0008A61K48/00A61K2039/53
    • This invention provides methods of treating an autoimmune disease in a subject associated with one or more self-protein(s), polypeptide(s), or peptide(s) present in the subject non-physiologically comprising administering to the subject: a self-vector comprising an immunosuppressive vector backbone and a polynucleotide encoding the self-protein(s), polypeptide(s) or peptide(s) associated with the autoimmune disease; and a divalent cation at a concentration greater than physiological levels. Administration of the self-vector comprising a polynucleotide encoding the self-protein(s), polypeptide(s) or peptide(s) modulates an immune response to the self-protein(s), polypeptide(s) or peptide(s) expressed from administration of the self-vector. This invention further provides a method of treating multiple sclerosis by administering a self-vector comprising a BHT-1 vector backbone, for example, self-vector BHT-3009 encoding human myelin basic protein (MBP). The invention also provides a pharmaceutical composition comprising: a BHT-1 vector backbone and a polynucleotide encoding one or more self-protein(s), polypeptide(s), or peptide(s) associated with an autoimmune disease; and a divalent cation at concentrations greater than physiological levels. This invention further provides a pharmaceutical composition comprising a self-vector comprising a BHT-1 vector backbone, for example, self-vector BHT-3009 encoding human myelin basic protein (MBP), and methods of administering a BHT-1 self-vector, for example BHT-3009, to a subject.
    • 本发明提供了治疗与存在于受试者中的一种或多种自身蛋白质,多肽或肽相关的受试者中的自身免疫性疾病的方法,其包括向受试者施用: 包含免疫抑制载体骨架和编码与自身免疫疾病相关的自身蛋白,多肽或肽的多核苷酸的载体; 和浓度高于生理水平的二价阳离子。 包含编码自身蛋白质,多肽或肽的多核苷酸的自身载体的施用调节对表达的自身蛋白质,多肽或肽的免疫应答, 从自我矢量的管理。 本发明还提供了通过施用包含BHT-1载体主链,例如编码人髓磷脂碱性蛋白(MBP)的自身载体BHT-3009的自身载体治疗多发性硬化的方法。 本发明还提供了药物组合物,其包含:BHT-1载体骨架和编码与自身免疫性疾病相关的一种或多种自身蛋白质,多肽或肽的多核苷酸; 和浓度高于生理水平的二价阳离子。 本发明进一步提供了一种药物组合物,其包含含有BHT-1载体主链,例如编码人髓磷脂碱性蛋白(MBP)的自身载体BHT-3009的自身载体和施用BHT-1自身载体的方法, 例如BHT-3009,给受试者。