利索-全球专利检索

  1. 登录
  2. 注册

搜索结果

39 条记录 (耗时 0.036 秒)

    Delivery of therapeutic compounds to the brain and other tissues
    2.
    发明授权
    Delivery of therapeutic compounds to the brain and other tissues 有权

    公开(公告)号：US09044473B2

    公开(公告)日：2015-06-02

    申请号：US11630446

    申请日：2004-08-30

    申请人： Emil D. Kakkis

    发明人： Emil D. Kakkis

    IPC分类号： A61K38/47 ,  C12N9/14 ,  C12Q1/34

    CPC分类号： A61K38/465 ,  A61K9/0085 ,  A61K38/47 ,  C12Y301/06008 ,  C12Y302/0105

    摘要： The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human α-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.

    METHODS AND COMPOSITIONS FOR THE TREATMENT OF PULMONARY HYPERTENSION OF THE NEWBORN
    3.
    发明申请
    METHODS AND COMPOSITIONS FOR THE TREATMENT OF PULMONARY HYPERTENSION OF THE NEWBORN 审中-公开
    治疗新生儿肺动脉高压的方法和组合

    公开(公告)号：US20100130500A1

    公开(公告)日：2010-05-27

    申请号：US11720815

    申请日：2005-12-08

    申请人： Emil D. Kakkis

    发明人： Emil D. Kakkis

    IPC分类号： A61K31/4985 ,  A61P9/12

    CPC分类号： A61K31/505

    摘要： The present invention is directed to a novel methods and compositions for the therapeutic intervention in persistent pulmonary hypertension of the newborn (PPHN). More specifically, the specification describes methods and compositions for treating various types of PPHN using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.

    摘要翻译： 本发明涉及用于新生儿持续性肺动脉高压（PPHN）的治疗性干预的新颖方法和组合物。 更具体地说，说明书描述了使用包含BH4的组合来处理各种类型的PPHN的方法和组合物。 预期BH4和其他治疗方案的组合疗法。

    Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof
    4.
    发明授权
    Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof 有权
    原核苯丙氨酸氨裂解酶的组合物及其组合物的使用方法

    公开(公告)号：US07531341B1

    公开(公告)日：2009-05-12

    申请号：US11451999

    申请日：2006-06-12

    申请人： Michel Claude Vellard ,  Paul Andrew Fitzpatrick ,  Emil D. Kakkis

    发明人： Michel Claude Vellard ,  Paul Andrew Fitzpatrick ,  Emil D. Kakkis

    IPC分类号： C12N9/88 ,  A61K38/00

    CPC分类号： C12N9/88 ,  A61K38/00 ,  C12Y403/01005

    摘要： The present invention is directed to phenylalanine ammonia lyase (PAL) produced by prokaryotes, wherein such prokaryotic PAL wherein the PAL variant has a greater phenylalanine-converting activity as compared to a wild-type PAL. The invention thus provides compositions of bacterial PAL and biologically active fragments, mutants, variants and analogs thereof, as well as methods for the production, purification, and use of such compositions for therapeutic and industrial purposes.

    摘要翻译： 本发明涉及由原核生物产生的苯丙氨酸氨裂解酶（PAL），其中与野生型PAL相比，PAL变体具有更大的苯丙氨酸转化活性的原核PAL。 因此，本发明提供细菌PAL和生物活性片段，突变体，变体和类似物的组合物，以及用于制备，纯化和使用这些组合物用于治疗和工业目的的方法。

    Induction of antigen specific immunologic tolerance
    5.
    发明授权
    Induction of antigen specific immunologic tolerance 失效
    诱导抗原特异性免疫耐受

    公开(公告)号：US07485314B2

    公开(公告)日：2009-02-03

    申请号：US10141668

    申请日：2002-05-06

    申请人： Emil D. Kakkis ,  Merry Passage ,  Thomas Lester ,  Rebecca Yang ,  Christopher Tanaka

    发明人： Emil D. Kakkis ,  Merry Passage ,  Thomas Lester ,  Rebecca Yang ,  Christopher Tanaka

    IPC分类号： A61K45/00

    CPC分类号： A61K39/00 ,  A61K31/522 ,  A61K31/7024 ,  A61K38/00 ,  A61K38/11 ,  A61K38/13 ,  A61K2300/00

    摘要： Antigen specific immune tolerance is induced in a mammalian host by administration of a toleragen in combination with a regimen of immunosuppression. The methods optionally include a preceding conditioning period, where immunosuppressive agents are administered in the absence of the toleragen. After the tolerizing regimen, the host is withdrawn from the suppressive agents, but is able to maintain specific immune tolerance to the immunogenic epitopes present on the toleragen. Optimally, the toleragen will have high uptake properties that allow uptake in vivo at low concentrations in a wide variety of tolerizing cell types.

    摘要翻译： 通过与免疫抑制方案组合施用耐受性，在哺乳动物宿主中诱导抗原特异性免疫耐受性。 所述方法任选地包括前述调节期，其中在不存在耐受性的情况下施用免疫抑制剂。 在耐受方案之后，宿主从抑制剂中排出，但能够对存在于耐受性上的免疫原性表位保持特异性的免疫耐受性。 最佳地，耐受性将具有高吸收性质，其允许在多种耐受细胞类型中以低浓度体内摄取。

    Delivery of therapeutic compounds to the brain and other tissues
    6.
    发明授权
    Delivery of therapeutic compounds to the brain and other tissues 有权
    向大脑和其他组织递送治疗性化合物

    公开(公告)号：US07442372B2

    公开(公告)日：2008-10-28

    申请号：US10651493

    申请日：2003-08-29

    申请人： Emil D. Kakkis

    发明人： Emil D. Kakkis

    IPC分类号： A61K38/47 ,  C12N9/14 ,  C12Q1/34

    CPC分类号： A61K38/465 ,  A61K9/0085 ,  A61K38/47 ,  C12Y301/06008 ,  C12Y302/0105

    摘要： The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human α-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.

    摘要翻译： 本发明涉及鞘内（IT）给予重组酶以治疗溶酶体储存障碍。 在一个示例性实施方案中，在MPS I受影响的动物中鞘内施用人α-L-艾杜糖醛酸苷酶（rhIDU）注射导致显着的酶摄取，脑和脑膜中显着的rh-艾杜糖醛酸酶活性和糖胺聚糖（GAG）储存在细胞中的降低 我接受正常科目的MPS。 在减轻MPS I症状时，鞘内给药证明比静脉内治疗更有效，表明它是治疗溶酶体储存障碍的有用方法。

    Delivery of therapeutic compounds to the brain and other tissues
    7.
    发明授权
    Delivery of therapeutic compounds to the brain and other tissues 有权
    向大脑和其他组织递送治疗性化合物

    公开(公告)号：US09089566B2

    公开(公告)日：2015-07-28

    申请号：US13399787

    申请日：2012-02-17

    申请人： Emil D. Kakkis

    发明人： Emil D. Kakkis

    IPC分类号： A61K38/47

    CPC分类号： A61K38/465 ,  A61K9/0085 ,  A61K38/47 ,  C12Y301/06008 ,  C12Y302/0105

    摘要： The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human α-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.

    摘要翻译： 本发明涉及鞘内（IT）给予重组酶以治疗溶酶体储存障碍。 在一个示例性实施方案中，在MPS I受影响的动物中鞘内施用人α-L-艾杜糖醛酸酶（rhIDU）注射导致显着的酶摄取，脑和脑膜中显着的rh-艾杜糖醛酸酶活性和糖胺聚糖（GAG）储存在细胞中 我接受正常科目的MPS。 在减轻MPS I症状时，鞘内给药证明比静脉内治疗更有效，表明它是治疗溶酶体储存障碍的有用方法。