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    PLANT LECTINS AS CARRIERS OF ASSOCIATED DRUG SUBSTANCES INTO ANIMAL AND HUMAN CELLS
    1.
    发明申请
    PLANT LECTINS AS CARRIERS OF ASSOCIATED DRUG SUBSTANCES INTO ANIMAL AND HUMAN CELLS 审中-公开
    植物细胞作为相关药物作为动物和人类细胞的载体

    公开(公告)号：WO2013181454A1

    公开(公告)日：2013-12-05

    申请号：PCT/US2013/043454

    申请日：2013-05-30

    Applicant: BIOSTRATEGIES LC

    Inventor： RADIN, David, N.

    IPC: A61K38/16 ,  A61K38/17 ,  A61K48/00 ,  A61P25/00

    CPC classification number: C12N9/2497 ,  A61K38/00 ,  A61K47/48261 ,  A61K47/6415 ,  C07K14/5434 ,  C07K16/16 ,  C07K2317/76 ,  C07K2319/00 ,  C07K2319/02 ,  C12N9/14 ,  C12N9/2402 ,  C12Y302/01076 ,  C12Y302/02022 ,  C12Y310/01001

    Abstract: The current invention involves the use of protein lectins produced by plants including the non-toxic carbohydrate binding subunits (B subunits) of plant "AB toxins" (PTB lectins) as delivery vehicles for mobilizing associated drug substances for delivery to animal and human cells. The resulting protein fusions or conjugates retain lectin carbohydrate specificity for binding to cells and cellular trafficking activity so as to deliver an associated drug compound to the site of disease manifestation. One embodiment of this invention concerns the ability of ricin toxin B subunit, as a model PTB lectin, to deliver enzyme replacement therapeutic drugs to cells of several organs of the body including the brain and central nervous system, eyes, ears, lungs, bone, heart, kidney, liver, and spleen for treating lysosomal diseases.

    Abstract translation: 本发明涉及使用由植物产生的蛋白质凝集素，包括植物“AB毒素”（PTB凝集素）的无毒性碳水化合物结合亚基（B亚基）作为用于调动相关药物以递送至动物和人细胞的递送载体。 所得到的蛋白质融合物或缀合物保留凝集素碳水化合物特异性以结合细胞和细胞转运活性，以将相关药物化合物递送至疾病表现的部位。 本发明的一个实施方案涉及蓖麻毒素B亚基作为模型PTB凝集素将酶替代治疗药物递送至身体的几个器官的细胞的能力，包括脑和中枢神经系统，眼睛，耳朵，肺，骨骼， 心脏，肾脏，肝脏和脾脏用于治疗溶酶体疾病。

    MODIFIED SULFAMIDASE AND PRODUCTION THEREOF
    2.
    发明申请
    MODIFIED SULFAMIDASE AND PRODUCTION THEREOF 审中-公开
    改性磺化酶及其生产

    公开(公告)号：WO2015150490A1

    公开(公告)日：2015-10-08

    申请号：PCT/EP2015/057256

    申请日：2015-04-01

    Applicant: SWEDISH ORPHAN BIOVITRUM AB (PUBL)

    Inventor： BERGHARD, Charlotta ,  NORDLING, Erik ,  SVENSSON GELIUS, Stefan ,  TJERNBERG, Agneta

    IPC: C07K14/435

    CPC classification number: C12N9/14 ,  A61K38/00 ,  C12N9/96 ,  C12Y310/01001

    Abstract: Disclosed herein are a modified sulfamidase, a composition comprising a modified sulfamidase, as well as methods for preparing a modified sulfamidase and therapeutic use of such a sulfamidase. In particular, the present disclosure relates to a modified sulfamidase comprising substantially no epitopes for glycan recognition receptors, thereby enabling transportation of said sulfamidase across the blood brain barrier of a mammal, wherein said sulfamidase has catalytic activity in the brain of said mammal.

    Abstract translation: 本文公开了改性的磺酰胺酶，包含改性的磺酰胺酶的组合物，以及制备修饰的磺酰胺酶的方法和这种磺酰胺酶的治疗用途。 具体地说，本公开内容涉及基本上不含聚糖识别受体的表位的修饰的磺酰胺酶，从而使所述磺酰胺酶能够跨越哺乳动物的血脑屏障传递，其中所述的磺酰胺酶在所述哺乳动物的脑中具有催化活性。

    MODIFIED LYSOSOMAL PROTEIN AND PRODUCTION THEREOF
    3.
    发明申请
    MODIFIED LYSOSOMAL PROTEIN AND PRODUCTION THEREOF 审中-公开
    改良的LYSOSOMAL蛋白质及其生产

    公开(公告)号：WO2017055570A1

    公开(公告)日：2017-04-06

    申请号：PCT/EP2016/073452

    申请日：2016-09-30

    Applicant: SWEDISH ORPHAN BIOVITRUM AB (PUBL)

    Inventor： NORDLING, Erik ,  STRÖMBERG, Patrik ,  SVENSSON GELIUS, Stefan

    IPC: C12P21/02 ,  A61K38/43 ,  C12P21/00 ,  A61K38/47

    CPC classification number: C12N9/14 ,  A61K38/00 ,  C12Y301/06013 ,  C12Y302/01076 ,  C12Y310/01001

    Abstract: Disclosed herein are a modified lysosomal protein, methods for preparing a modified lysosomal protein and therapeutic use of such a modified protein. Further disclosed herein is a method of treating a mammal afflicted with a lysosomal storage disease.In particular, the present disclosure relates to a method of preparing a modified lysosomal protein, said method comprising reacting a glycosylated lysosomal protein with an alkali metal periodate and reacting said lysosomal protein with an alkali metal borohydride for a time period of no more than 2 h, thereby modifying glycan moieties of the lysosomal protein and reducing the activity of the lysosomal protein with respect to glycan recognition receptors.

    Abstract translation: 本文公开了修饰的溶酶体蛋白质，制备修饰的溶酶体蛋白质的方法和这种修饰蛋白质的治疗用途。 本文进一步公开的是治疗患有溶酶体贮积病的哺乳动物的方法。特别地，本公开内容涉及制备修饰的溶酶体蛋白的方法，所述方法包括使糖基化溶酶体蛋白与碱金属高碘酸酯反应并使所述 溶酶体蛋白与碱金属硼氢化物的时间不超过2小时，从而修饰溶酶体蛋白的聚糖部分并降低溶酶体蛋白质相对于聚糖识别受体的活性。

    METHODS AND COMPOSITIONS FOR INCREASING ENZYME ACTIVITY IN THE CNS
    5.
    发明申请
    METHODS AND COMPOSITIONS FOR INCREASING ENZYME ACTIVITY IN THE CNS 审中-公开
    在CNS中增加酶活性的方法和组合物

    公开(公告)号：WO2015012944A1

    公开(公告)日：2015-01-29

    申请号：PCT/US2014/038660

    申请日：2014-05-19

    Applicant: ARMAGEN TECHNOLOGIES, INC

    Inventor： PARDRIDGE, William, M. ,  BOADO, Ruben, J.

    IPC: C07K16/18 ,  C07K16/28 ,  A61K38/43

    CPC classification number: C07K16/2869 ,  A61K2039/505 ,  C07K16/26 ,  C07K2317/24 ,  C07K2317/565 ,  C07K2317/92 ,  C07K2319/00 ,  C07K2319/30 ,  C12N9/14 ,  C12Y310/01001

    Abstract: Provided herein are methods and compositions for treating a subject suffering from an enzyme deficiency in the central nervous system (CNS). The bifunctional fusion antibodies provided herein comprise an antibody to an endogenous blood brain barrier (BBB) receptor and an enzyme deficient in mucopolysaccharidosis III (MPS-III). The fusion antibodies provided herein comprise N- sulfoglucosamine sulfohydrolase (SGSH), alpha-N-acetylgulcosaminidase (NAGLU), heparin-alpha- glucosaminide N-acetyltransferase (HGSNAT), or N-acetylglucosamine-6-sulfatase (GNS). The methods of treating an enzyme deficiency in the CNS comprise systemic administration of a fusion antibody provided herein.

    Abstract translation: 本文提供了用于治疗患有中枢神经系统（CNS）中的酶缺乏的受试者的方法和组合物。 本文提供的双功能融合抗体包含针对内源性血脑屏障（BBB）受体的抗体和缺陷型粘多糖贮积症III（MPS-III）的酶。 本文提供的融合抗体包含N-磺基葡糖胺磺基水解酶（SGSH），α-N​​-乙酰氨基糖苷酶（NAGLU），肝素-α-葡糖胺-N-乙酰转移酶（HGSNAT）或N-乙酰葡糖胺-6-硫酸酯酶（GNS）。 在CNS中治疗酶缺乏症的方法包括全身给予本文提供的融合抗体。

    INTRANASAL ADMINISTRATION OF GUANIDINYLATED AMINOGLYCOSIDES
    6.
    发明申请
    INTRANASAL ADMINISTRATION OF GUANIDINYLATED AMINOGLYCOSIDES 审中-公开
    内分泌氨基酸的内在管理

    公开(公告)号：WO2014159878A2

    公开(公告)日：2014-10-02

    申请号：PCT/US2014025382

    申请日：2014-03-13

    Applicant: UNIV CALIFORNIA

    Inventor： ESKO JEFFREY D ,  TOR YITZHAK ,  TONG WENYONG

    IPC: A61P31/04

    CPC classification number: A61K47/67 ,  A61K9/0043 ,  A61K38/46 ,  A61K38/47 ,  A61K39/39533 ,  A61K47/48092 ,  A61K47/48115 ,  A61K47/549 ,  A61K47/552 ,  A61K47/62 ,  C12Y302/01076 ,  C12Y310/01001

    Abstract: This disclosure relates to intranasal administration of conjugates comprising guanidinylated aminoglycosides ("guanidinoglycosides") and a polypeptide (e.g., an enzyme, antibody, or polypeptide growth factor). For example, such administration methods are useful for delivering a polypeptide to the brain and/or cerebrospinal fluid. Such methods are useful for treating a lysosomal storage disease through intranasal administration of a conjugate comprising one or more guanidinoglycosides and an enzyme useful for treating a lysosomal storage disease.

    Abstract translation: 本公开涉及鼻内施用包含胍基化氨基糖苷（“胍基糖苷”）和多肽（例如酶，抗体或多肽生长因子）的缀合物。 例如，这种给药方法可用于将多肽递送至脑和/或脑脊液。 这样的方法可用于通过鼻内施用包含一种或多种胍胍糖苷和可用于治疗溶酶体贮积病的酶的结合物来治疗溶酶体贮积病。

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