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1.发明申请THERAPY REGIMENS, DOSING REGIMENS AND STABLE MEDICAMENTS FOR THE TREATMENT OF POMPE DISEASE 审中-公开治疗方案，治疗方案和稳定药物治疗轻度疾病公开(公告)号：US20100119502A1
公开(公告)日：2010-05-13
申请号：US12616670
申请日：2009-11-11
Applicant: Hung V. Do , Ken Valenzano , David J. Lockhart
Inventor： Hung V. Do , Ken Valenzano , David J. Lockhart
CPC classification number: A61K38/47 , A61K2300/00
Abstract: The present application provides a method for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject a GAA enzyme in combination with an ASSC for the GAA enzyme. The present application also provides methods for increasing the in vitro and in vivo stability of a GAA enzyme formulation.
Abstract translation: 本申请提供了在有需要的受试者中治疗庞培病的方法，其包括将GAA酶与GAA酶的ASSC组合给受试者的方法。 本申请还提供了增加GAA酶制剂的体外和体内稳定性的方法。
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2.发明授权公开(公告)号：US08592362B2
公开(公告)日：2013-11-26
申请号：US12855468
申请日：2010-08-12
Applicant: Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
Inventor： Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
IPC: C07K14/00
CPC classification number: A61K31/445 , C07K14/00 , C12Q1/34 , G01N33/6893 , G01N2333/47 , G01N2333/94 , G01N2800/38 , G01N2800/52
Abstract: The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
Abstract translation: 本发明提供了确定患有溶酶体储存障碍的患者是否将受益于用特定药物伴侣的治疗的方法。 本发明举例说明了在表达来自α-半乳糖苷酶A的突变体的细胞系中测定α-半乳糖苷酶A对药物分子伴侣如1-脱氧半乳糖吉非霉素的反应性的体外方法。本发明还提供了一种用于诊断患者中法布里病的方法 怀疑患有法布里病。
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3.发明申请公开(公告)号：US20110136151A1
公开(公告)日：2011-06-09
申请号：US12920856
申请日：2009-03-12
Applicant: Brandon Wustman , Hung V. Do
Inventor： Brandon Wustman , Hung V. Do
IPC: G01N33/53
CPC classification number: G01N33/6893 , G01N2333/928 , G01N2800/042 , G01N2800/52
Abstract: Provided are in vitro, ex vivo and in vivo methods for determining whether a patient with Pompe disease will respond to treatment with a specific pharmacological chaperone.
Abstract translation: 提供了用于确定具有庞培病的患者是否将对具有特定药物分子伴侣的治疗作出反应的体外，离体和体内方法。
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公开(公告)号：US20120083010A1
公开(公告)日：2012-04-05
申请号：US13212917
申请日：2011-08-18
Applicant: Hung V. Do , Brian E. Rane , John Flanagan
Inventor： Hung V. Do , Brian E. Rane , John Flanagan
IPC: C12Q1/34
CPC classification number: C12Q1/34 , G01N33/5047 , G01N33/577 , G01N2333/924 , G01N2800/042 , G01N2800/52
Abstract: Provided is a method for diagnosing Pompe disease in a patient by measuring acid α-glucosidase activity in a sample from the patient. The invention also provides a method for monitoring the treatment of Pompe disease with specific pharmacological chaperones by measuring acid α-glucosidase activity in a sample from the patient.
Abstract translation: 提供了一种通过测量来自患者的样品中的酸性α-葡萄糖苷酶活性来诊断患者庞波病的方法。 本发明还提供了通过测量来自患者的样品中的酸性α-葡萄糖苷酶活性来监测具有特定药理学分子伴侣的庞毕病治疗的方法。
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5.发明授权公开(公告)号：US09206457B2
公开(公告)日：2015-12-08
申请号：US12952036
申请日：2010-11-22
Applicant: Hung V. Do
Inventor： Hung V. Do
IPC: C12P21/02 , C07D201/00 , C12N5/07 , C12N5/10 , C12N9/00 , C07K5/00 , C07K7/00 , C07K16/00 , C07K17/00 , A61K38/00 , C07K1/14 , C07K1/22
CPC classification number: C12P21/02 , C07D201/00 , C07K1/14 , C07K1/22 , C12Y302/01054
Abstract: The present invention provides methods for improving the production of recombinant proteins through the use of pharmacological chaperones for the recombinant proteins. As exemplified by the present invention, the binding of a pharmacological chaperone to a recombinant protein expressed by a cell can stabilize the protein and increase export of the protein out of the cell's endoplasmic reticulum, and increase secretion of the protein by the cell.
Abstract translation: 本发明提供了通过使用用于重组蛋白的药理学伴侣来改善重组蛋白质生产的方法。 如本发明所示，药物伴侣与由细胞表达的重组蛋白的结合可稳定蛋白质并增加蛋白质从细胞内质网中的输出，并增加细胞分泌蛋白质。
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Patent Agency Ranking
6.发明申请公开(公告)号：US20110212996A1
公开(公告)日：2011-09-01
申请号：US12855468
申请日：2010-08-12
Applicant: Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
Inventor： Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
CPC classification number: A61K31/445 , C07K14/00 , C12Q1/34 , G01N33/6893 , G01N2333/47 , G01N2333/94 , G01N2800/38 , G01N2800/52
Abstract: The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
Abstract translation: 本发明提供了确定患有溶酶体储存障碍的患者是否将受益于用特定药物伴侣的治疗的方法。 本发明例举了在表达来自α-半乳糖苷酶A的突变体的细胞系中测定α-半乳糖苷酶A对药物分子伴侣如1-脱氧半乳糖吉非霉素的反应性的体外方法。本发明还提供了一种诊断患者法布里病的方法 怀疑患有法布里病。
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7.发明申请TREATMENT OF POMPE DISEASE WITH SPECIFIC PHARMACOLOGICAL CHAPERONES AND MONITORING TREATMENT USING SURROGATE MARkERS 审中-公开用特定药理学治疗轻度疾病的治疗和使用血糖检测的监测治疗公开(公告)号：US20110189710A1
公开(公告)日：2011-08-04
申请号：US12920864
申请日：2009-03-12
Applicant: Brandon Wustman , Hung V. Do
Inventor： Brandon Wustman , Hung V. Do
CPC classification number: G01N33/6893 , G01N33/5091 , G01N2333/49 , G01N2333/515 , G01N2333/5412 , G01N2333/5418 , G01N2333/5421 , G01N2800/042 , G01N2800/52
Abstract: Provided is a method of monitoring the treatment of Pompe disease with specific pharmacological chaperones using systemic and/or cellular surrogate markers.
Abstract translation: 提供了一种使用全身和/或细胞代谢标记物监测具有特定药物伴侣的庞毕病治疗的方法。
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公开(公告)号：US20110152319A1
公开(公告)日：2011-06-23
申请号：US12860611
申请日：2010-08-20
Applicant: Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
Inventor： Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
IPC: A61K31/445 , C12Q1/34 , A61P3/00
CPC classification number: C12Q1/34 , G01N2333/94 , G01N2800/04 , G01N2800/52
Abstract: The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
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9.发明申请Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics 审中-公开利用药物伴侣改善生物制剂的制造和纯化公开(公告)号：US20110070643A1
公开(公告)日：2011-03-24
申请号：US12788068
申请日：2010-05-26
Applicant: Hung V. Do
Inventor： Hung V. Do
IPC: C12N5/07
CPC classification number: C12N9/96 , C12N9/2402 , C12P21/02 , Y02E50/17
Abstract: The present invention provides methods for improving the production of recombinant proteins through the use of pharmacological chaperones for the recombinant proteins. As exemplified by the present invention, the binding of a pharmacological chaperone to a recombinant protein expressed by a cell can stabilize the protein and increase export of the protein out of the cell's endoplasmic reticulum, and increase secretion of the protein by the cell.
Abstract translation: 本发明提供了通过使用用于重组蛋白的药理学伴侣来改善重组蛋白质生产的方法。 如本发明所示，药物伴侣与由细胞表达的重组蛋白的结合可稳定蛋白质并增加蛋白质从细胞内质网中的输出，并增加细胞分泌蛋白质。
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10.发明授权Compositions and methods for the expression of factor VIII polypeptides and uses therefor 失效用于表达因子VIII多肽的组合物和方法及其用途公开(公告)号：US06517830B1
公开(公告)日：2003-02-11
申请号：US09633020
申请日：2000-08-04
Applicant: John S. Lollar , Hung V. Do , John F. Healey , Edmund K. Waller
Inventor： John S. Lollar , Hung V. Do , John F. Healey , Edmund K. Waller
IPC: A01N6300
CPC classification number: C12P21/02 , A61K48/00 , C07K14/755
Abstract: Compositions and methods are provided for the in vivo gene delivery of nucleic acid sequences encoding the factor VIII protein to the liver endothelial sinusoidal cells (LSECs). Compositions and methods are also provided for the ex vivo gene transfer of nucleic acid sequences encoding the factor VIII protein to cultured LSECs and the implantation of the transformed LSECs in vivo. These methods and compositions increase the level of factor VIII in the blood stream and find use in the gene therapy treatment of hemophilia A.
Abstract translation: 提供了将编码因子VIII蛋白的核酸序列的体内基因递送给肝内皮正弦细胞（LSEC）的组合物和方法。 还提供了组合物和方法用于将编码因子VIII蛋白的核酸序列的体外基因转移到培养的LSECs和体内转化的LSEC的植入。 这些方法和组合物增加血液中血液中VIII因子的水平，并可用于血友病A的基因疗法治疗。
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