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公开(公告)号：US10280415B2
公开(公告)日：2019-05-07
申请号：US15458081
申请日：2017-03-14
Inventor： Jan P. Kraus
Abstract: Human cystathionine β-synthase variants are disclosed, as well as a method to produce recombinant human cystathionine β-synthase and variants thereof. More particularly, the role of both the N-terminal and C-terminal regions of human CBS has been studied, and a variety of truncation mutants and modified CBS homologs are described. In addition, a method to express and purify recombinant human cystathionine β-synthase (CBS) and variants thereof which have only one or two additional amino acid residues at the N-terminus are described.
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公开(公告)号：US10265387B2
公开(公告)日：2019-04-23
申请号：US15492671
申请日：2017-04-20
Inventor： Jan P. Kraus , Tomas Majtan , Erez Bublil
IPC: A61K38/51 , C12N9/96 , C12N9/88 , A61K31/205 , A61K47/48
Abstract: Provided herein are improved compositions and methods for enzyme replacement therapy using modified human cystathionine beta synthase (CBS) in the treatment of homocystinuria and related diseases and disorders.
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公开(公告)号：US20180140688A1
公开(公告)日：2018-05-24
申请号：US15571640
申请日：2016-05-03
Inventor： Jan P. Kraus
CPC classification number: A61K38/51 , A61P43/00 , C07K14/47 , C12N9/10 , C12N15/52 , C12Q1/68 , G01N33/53 , Y02A50/473
Abstract: This invention provides compositions of human propionyl-CoA carboxylase (PCC) and pharmaceutical compositons of human PCC and methods for treating conditions such as propionic acidemia (PA), propionic aciduria, propionyl-CoA carboxylase deficiency and ketotic glycinemia.
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24.发明授权公开(公告)号：US09284546B2
公开(公告)日：2016-03-15
申请号：US14663749
申请日：2015-03-20
Inventor： Jan P. Kraus
IPC: C12N9/88
CPC classification number: C12N9/88 , A61K38/00 , C12Y402/01022
Abstract: Human cystathionine β-synthase variants are disclosed, as well as a method to produce recombinant human cystathionine β-synthase and variants thereof. More particularly, the role of both the N-terminal and C-terminal regions of human CBS has been studied, and a variety of truncation mutants and modified CBS homologues are described. In addition, a method to express and purify recombinant human cystathionine β-synthase (CBS) and variants thereof which have only one or two additional amino acid residues at the N-terminus are described.
Abstract translation: 公开了人胱硫醚和bgr- -Synthase变体，以及一种产生重组人胱硫醚及其变体的方法。 更具体地，已经研究了人CBS的N-末端和C-末端区域的作用，并且描述了各种截短突变体和修饰的CBS同源物。 另外，描述了在N末端仅具有一个或两个另外的氨基酸残基的重组人胱硫醚和bgr.Synthase（CBS）及其变体的表达和纯化方法。
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Patent Agency Ranking
公开(公告)号：US09243239B2
公开(公告)日：2016-01-26
申请号：US13830494
申请日：2013-03-14
Inventor： Richard Carrillo , Jan P. Kraus , Tomas Majtan , David Naveh
IPC: C12N9/88
CPC classification number: C12N9/88 , A61K38/51 , B01D15/363 , B01D15/3823 , C12Y402/01022
Abstract: This invention provides chromatographic methods for the purification of a cystathionine β-Synthase (CBS) protein, particularly truncated variants thereof and compositions and pharmaceutical compositions prepared therefrom.
Abstract translation: 本发明提供了用于纯化胱硫醚和硫酸钠（CBS）蛋白质，特别是其截短的变体以及由其制备的组合物和药物组合物的色谱方法。
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公开(公告)号：US20230042914A1
公开(公告)日：2023-02-09
申请号：US17852246
申请日：2022-06-28
Inventor： Jan P. Kraus , Tomas Majtan , Erez Bublil
IPC: A61K38/51 , A61K31/205 , A61K47/60 , A61K9/00 , A61K45/06 , C12N9/88 , C12N9/96 , A61K31/198 , A61K31/385
Abstract: Provided herein are improved compositions and methods for enzyme replacement therapy using modified human cystathionine beta synthase (CBS) in the treatment of homocystinuria and related diseases and disorders.
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27.发明申请公开(公告)号：US20220265835A1
公开(公告)日：2022-08-25
申请号：US17622112
申请日：2020-06-26
Inventor： Erez Bublil , Frank Glavin , Marcia Sellos-Moura , Jan P. Kraus , Tomas Majtan , Randy Wanner , Orhan Causevic
Abstract: The present disclosure provides formulations for a drug product comprising a PEGylated CBS protein having the amino acid sequence of SEQ ID NO: 1. Dosages and dosing regimens are provided for treatment of homocystinuria in a subject in need thereof. Additionally, the dosages and dosing regimens are also provided to reduce the level of homocysteine (Hcy) or increase the levels of cysteine (Cys) and/or cystathionine (Cth) in a subject in need thereof.
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公开(公告)号：US20200261555A1
公开(公告)日：2020-08-20
申请号：US16605918
申请日：2018-04-17
Inventor： Tomas Majtan , Jan P. Kraus , Erez M. Bublil , Frank Glavin , Marcia Sellos-Moura
Abstract: The present invention provides a method of PEGylating a human truncated cystathionine β-synthase protein containing a mutation of a cysteine to a serine at amino acid position 15 (htCBS C15S). The htCBS C15S was PEGylated with one of 5 kDa, 10 kDa, or 20 kDa NHS ester PEG molecules. In-process monitoring of the PEGylation process was used in the method to reduce levels of unPEGylated htCBS C15S and htCBS C15S with insufficient PEGylation. Administration of the PEGylated htCBS C15S had efficacy throughout the course of treatment for homocystinuria.
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公开(公告)号：US20170224787A1
公开(公告)日：2017-08-10
申请号：US15492671
申请日：2017-04-20
Inventor： Jan P. Kraus , Tomas Majtan , Erez Bublil
IPC: A61K38/51 , C12N9/88 , C12N9/96 , A61K31/205
CPC classification number: A61K38/51 , A61K9/0019 , A61K31/198 , A61K31/205 , A61K31/385 , A61K45/06 , A61K47/48215 , A61K47/60 , A61K2300/00 , C12N9/88 , C12N9/96 , C12Y402/01022
Abstract: Provided herein are improved compositions and methods for enzyme replacement therapy using modified human cystathionine beta synthase (CBS) in the treatment of homocystinuria and related diseases and disorders.
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