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1. 发明授权

US06972124B2 Precursor of N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme 有权
标题翻译： N-乙酰半乳糖胺-4-硫酸酯酶的前体，使用所述酶的处理方法和生产和纯化所述酶的方法
公开(公告)号：US06972124B2
公开(公告)日：2005-12-06
申请号：US10704365
申请日：2003-11-07
申请人： Minmin Qin , Gary N. Zecherle , Wai-Pan Chan , Paul A. Fitzpatrick , Stuart Sweidler , John M. Henstrand , Dan J. Wendt , Lin Chen , Christopher M. Starr
发明人： Minmin Qin , Gary N. Zecherle , Wai-Pan Chan , Paul A. Fitzpatrick , Stuart Sweidler , John M. Henstrand , Dan J. Wendt , Lin Chen , Christopher M. Starr
IPC分类号： A61K38/00 , A61K38/46 , A61K48/00 , C12N9/16 , A23J1/00 , C12N1/20 , C12N5/00
CPC分类号： B01D15/327 , A61K38/00 , A61K48/00 , B01D15/3804 , B01D15/3828 , C12N9/16 , C12Y301/06012
摘要： The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.
摘要翻译：本发明提供高度纯化的重组人前体N-乙酰半乳糖胺-4-硫酸酯酶及其生物活性突变体，其片段和类似物以及包含高度纯化的重组人前体N-乙酰半乳糖胺-4-硫酸酯酶的药物制剂。本发明还提供了用于治疗全部或部分由包括MPS VI在内的人N-乙酰半乳糖胺-4-硫酸酯酶缺陷引起的疾病的方法，以及用于将重组前体酶生产和纯化成高度纯化形式的方法。

2. 发明申请

US20080260715A1 Precursor N-acetylgalactosamine-4 sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme 审中-公开
标题翻译：前体N-乙酰半乳糖胺-4-硫酸酯酶，使用所述酶的处理方法和生产和纯化所述酶的方法
公开(公告)号：US20080260715A1
公开(公告)日：2008-10-23
申请号：US11804369
申请日：2007-05-18
申请人： Minmin Qin , Gary N. Zecherle , Wai-Pan Chan , Paul A. Fitzpatrick , Stuart Swiedler , John M. Henstrand , Dan J. Wendt , Lin Chen , Christopher M. Starr
发明人： Minmin Qin , Gary N. Zecherle , Wai-Pan Chan , Paul A. Fitzpatrick , Stuart Swiedler , John M. Henstrand , Dan J. Wendt , Lin Chen , Christopher M. Starr
IPC分类号： A61K38/46
CPC分类号： B01D15/327 , A61K38/00 , A61K48/00 , B01D15/3804 , B01D15/3828 , C12N9/16 , C12Y301/06012
摘要： The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.
摘要翻译：本发明提供高度纯化的重组人前体N-乙酰半乳糖胺-4-硫酸酯酶及其生物活性突变体，其片段和类似物以及包含高度纯化的重组人前体N-乙酰半乳糖胺-4-硫酸酯酶的药物制剂。本发明还提供了用于治疗全部或部分由包括MPS VI在内的人N-乙酰半乳糖胺-4-硫酸酯酶缺陷引起的疾病的方法，以及用于将重组前体酶生产和纯化成高度纯化形式的方法。

3. 发明授权

US06866844B2 Precursor N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme 有权
标题翻译：前体N-乙酰半乳糖胺-4-硫酸酯酶，使用所述酶的处理方法和用于产生和纯化所述酶的方法
公开(公告)号：US06866844B2
公开(公告)日：2005-03-15
申请号：US10290908
申请日：2002-11-07
申请人： Minmin Qin , John M. Henstrand , Gary N. Zecherle , Dan J. Wendt , Wai-Pan Chan , Lin Chen , Paul A. Fitzpatrick , Christopher M. Starr
发明人： Minmin Qin , John M. Henstrand , Gary N. Zecherle , Dan J. Wendt , Wai-Pan Chan , Lin Chen , Paul A. Fitzpatrick , Christopher M. Starr
IPC分类号： A61K38/46 , C12N9/16 , A23J1/00 , C12N1/20 , C12N15/00
CPC分类号： B01D15/3804 , A61K38/00 , B01D15/327 , B01D15/3828 , C12N9/16 , C12Y301/06012
摘要： The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.
摘要翻译：本发明提供高度纯化的重组人前体N-乙酰半乳糖胺-4-硫酸酯酶及其生物活性突变体，其片段和类似物以及包含高度纯化的重组人前体N-乙酰半乳糖胺-4-硫酸酯酶的药物制剂。本发明还提供了用于治疗全部或部分由包括MPS VI在内的人N-乙酰半乳糖胺-4-硫酸酯酶缺陷引起的疾病的方法，以及用于将重组前体酶生产和纯化成高度纯化形式的方法。

4. 发明授权

US06569661B1 Recombinant &agr;-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof 有权
标题翻译：重组α-L-艾杜糖醛酸酶，其制备和纯化方法以及用于治疗由其缺陷引起的疾病的方法
公开(公告)号：US06569661B1
公开(公告)日：2003-05-27
申请号：US09711202
申请日：2000-11-09
申请人： Minmin Qin , Wai-Pan Chan , Lin Chen , Paul A. Fitzpatrick , John M. Hendstrand , Dan J. Wendt , Gary N. Zecherle , Christopher M. Starr , Emil D. Kakkis
发明人： Minmin Qin , Wai-Pan Chan , Lin Chen , Paul A. Fitzpatrick , John M. Hendstrand , Dan J. Wendt , Gary N. Zecherle , Christopher M. Starr , Emil D. Kakkis
IPC分类号： C12Q100
CPC分类号： C12Y302/01076 , A61K38/00 , A61K48/00 , C12N9/2402 , C12N9/88 , C12Y302/01014 , C12Y302/01075 , C12Y402/02004
摘要： The present invention provides a recombinant human &agr;-L-iduronidase and biologically active fragments and mutants thereof, large scale methods to produce and purify commercial grade recombinant human &agr;-L-iduronidase enzyme as well as methods to treat certain genetic disorders including &agr;-L-iduronidase deficiency and mucopolysaccharidosis I (MPS 1).
摘要翻译：本发明提供了重组人α-L-真蛋白酶及其生物活性片段和突变体，大规模生产和纯化商业级重组人α-L-真蛋白酶的方法以及治疗某些遗传病症（包括α-L - 糖尿病病毒缺乏和粘多糖贮积症I（MPS 1）。

5. 发明授权

US08795627B2 Treatment of liver disorders by administration of RAP conjugates 有权
标题翻译：通过施用RAP偶联物治疗肝脏疾病
公开(公告)号：US08795627B2
公开(公告)日：2014-08-05
申请号：US12441618
申请日：2007-09-18
申请人： Christopher M. Starr , Todd C. Zankel
发明人： Christopher M. Starr , Todd C. Zankel
IPC分类号： C07K14/705 , A61K38/17 , A61K47/48
CPC分类号： A61K38/17 , A61K31/704 , A61K31/715 , A61K33/24 , A61K38/168 , A61K38/177 , A61K39/395 , A61K45/06 , A61K47/64 , A61N5/1001 , C07K14/705 , Y02A50/403 , A61K2300/00
摘要： The present invention relates to the use of receptor-associate protein (RAP) and fragments and variants thereof to improve delivery of therapeutic compounds to the liver and provides methods to treat liver disorders and conditions, such as hepatic carcinoma, by administering RAP or RAP variants conjugated to active agents.
摘要翻译：本发明涉及受体相关蛋白（RAP）及其片段和变体的用途，以改善治疗化合物向肝脏的递送，并提供通过施用RAP或RAP变体来治疗肝脏疾病和病症如肝癌的方法与活性剂缀合。

6. 发明授权

US08609103B2 Megalin-based delivery of therapeutic compounds to the brain and other tissues 有权
标题翻译：基于Megalin的治疗化合物向大脑和其他组织递送
公开(公告)号：US08609103B2
公开(公告)日：2013-12-17
申请号：US12941619
申请日：2010-11-08
申请人： Todd C. Zankel , Christopher M. Starr
发明人： Todd C. Zankel , Christopher M. Starr
IPC分类号： A61K39/00
CPC分类号： C07K14/705 , A61K38/00 , A61K47/64 , C07K2319/00 , C07K2319/22 , C07K2319/23
摘要： The present invention is directed to a methods and compositions for receptor mediated drug delivery, particularly across the blood-brain barrier.
摘要翻译：本发明涉及受体介导的药物递送的方法和组合物，特别是跨血脑屏障。

7. 发明申请

US20090191178A1 Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof 审中-公开
标题翻译：高度磷酸化溶酶体酶的制备及其用途
公开(公告)号：US20090191178A1
公开(公告)日：2009-07-30
申请号：US12182818
申请日：2008-07-30
申请人： Todd C. Zankel , Christopher M. Starr
发明人： Todd C. Zankel , Christopher M. Starr
IPC分类号： A61K38/47 , C12N9/24 , C12P21/04 , C12N5/06
CPC分类号： C12P21/005 , A61K38/00 , C12N9/2408 , C12N9/2434 , C12Y302/01003 , C12Y302/0102
摘要： This invention provides compositions of highly phosphorylated lysosomal enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases.
摘要翻译：本发明提供高度磷酸化溶酶体酶的组合物，其药物组合物，生产和纯化这些溶酶体酶及其组合物的方法及其在疾病和病症的诊断，预防或治疗中的用途，特别是溶酶体贮积病。

8. 发明授权

US5591839A Polynucleotides encoding .alpha.2-3 neuraminidase 失效
标题翻译：编码α2-3神经氨酸酶的多核苷酸
公开(公告)号：US5591839A
公开(公告)日：1997-01-07
申请号：US294477
申请日：1994-08-18
申请人： Harvey I. Miller , John C. Klock , Christopher M. Starr
发明人： Harvey I. Miller , John C. Klock , Christopher M. Starr
IPC分类号： C12N9/24 , C07H21/02 , C07H21/04
CPC分类号： C12Y302/01018 , C12N9/2402
摘要： Polynucleotide sequences encoding enzymes that possess .alpha.2-3 neuraminidase activity are provided. Of particular interest are polynucleotide sequences encoding an enzyme having .alpha.2-3 neuraminidase activity and naturally produced by the bacteria Streptococcus pneumoniae. Recombinant DNA expression of enzymes possessing .alpha.2-3 specific neuraminidase activity is also described, including methods, recombinant host cells and a genetic construction. The invention also provides a purified enzyme having .alpha.2-3 neuraminidase activity from Streptococcus pneumoniae, wherein the enzyme is isolated from S. pneumoniea cultures. Another aspect of this invention is to provide methods of isolating genes encoding enzymes having neuraminidase activity, particularly .alpha.2-3 neuraminidase activity. The gene isolation methods of the invention comprise the step of labeling a hybridization probe derived from the neuraminidase coding portion of plasmid pND-1. The hybridization probe may then be used to isolate homologous genes encoding enzymes having the desired enzymatic activity.
摘要翻译：提供了编码具有α2-3神经氨酸酶活性的酶的多核苷酸序列。特别感兴趣的是编码具有α2-3神经氨酸酶活性并由细菌肺炎链球菌天然产生的酶的多核苷酸序列。还描述了具有α2-3特异性神经氨酸酶活性的酶的重组DNA表达，包括方法，重组宿主细胞和遗传构建。本发明还提供了具有来自肺炎链球菌的α2-3神经氨酸酶活性的纯化酶，其中所述酶从肺炎链球菌培养物中分离。本发明的另一方面是提供分离编码具有神经氨酸酶活性的酶的基因，特别是α2-3神经氨酸酶活性的方法。本发明的基因分离方法包括标记衍生自质粒pND-1的神经氨酸酶编码部分的杂交探针的步骤。然后可以使用杂交探针来分离编码具有所需酶活性的酶的同源基因。

9. 发明授权

US08877714B2 Compositions comprising receptor-associated protein (RAP) variants specific for LRP2 and uses thereof 有权
标题翻译：包含对LRP2特异的受体相关蛋白（RAP）变体的组合物及其用途
公开(公告)号：US08877714B2
公开(公告)日：2014-11-04
申请号：US11576506
申请日：2006-06-14
申请人： Christopher M. Starr , Todd C. Zankel
发明人： Christopher M. Starr , Todd C. Zankel
IPC分类号： C07K14/705 , A61K38/17 , A61K38/00
CPC分类号： C07K14/705 , A61K38/00
摘要： The present invention relates generally to receptor-selective variants of the low-density lipoprotein receptor-associated protein (RAP) and compositions thereof, methods of generating such variants and methods of using such receptor-selective RAP variant compositions for therapeutic purposes. The invention also relates to antibodies that bind to one or a small subset of CR-containing proteins.
摘要翻译：本发明一般涉及低密度脂蛋白受体相关蛋白（RAP）的受体选择性变体及其组合物，产生这些变体的方法和使用这种受体选择性RAP变体组合物用于治疗目的的方法。本发明还涉及结合至含有CR的蛋白质的一个或一小组的抗体。

10. 发明授权

US07829537B2 Receptor associated protein (RAP) conjugates 有权
标题翻译：受体相关蛋白（RAP）偶联物
公开(公告)号：US07829537B2
公开(公告)日：2010-11-09
申请号：US12508956
申请日：2009-07-24
申请人： Todd C. Zankel , Christopher M. Starr
发明人： Todd C. Zankel , Christopher M. Starr
IPC分类号： A61K38/18 , A61K38/30 , A61K33/24
CPC分类号： C07K14/705 , A61K38/00 , A61K47/64 , C07K2319/00 , C07K2319/22 , C07K2319/23
摘要： The present invention is directed to a methods and compositions for receptor mediated drug delivery, particularly across the blood-brain barrier.
摘要翻译：本发明涉及受体介导的药物递送的方法和组合物，特别是跨血脑屏障。

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