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公开(公告)号:US10160962B2
公开(公告)日:2018-12-25
申请号:US14970814
申请日:2015-12-16
Inventor: Richard Carrillo , Jan P. Kraus , Tomas Majtan , David Naveh
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.
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公开(公告)号:US11324811B2
公开(公告)日:2022-05-10
申请号:US16605918
申请日:2018-04-17
Applicant: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE , Travere Therapeutics Switzerland GmbH
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:US11077175B2
公开(公告)日:2021-08-03
申请号:US16824931
申请日:2020-03-20
Inventor: Jan P. Kraus , Tomas Majtan , Erez Bublil
IPC: A61K38/51 , A61K47/60 , A61K9/00 , A61K31/198 , A61K31/205 , A61K31/385 , A61K45/06 , C12N9/88 , C12N9/96 , A61P43/00 , A61P27/02 , A61P19/10 , A61P17/14 , A61P13/02
Abstract: Provided are 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: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:US09675678B2
公开(公告)日:2017-06-13
申请号:US14935690
申请日:2015-11-09
Inventor: Jan P Kraus , Tomas Majtan , Erez Bublil
IPC: A61K38/54 , A61K38/00 , C12N9/96 , C12N9/88 , C07H21/04 , C07K1/00 , A61K38/51 , A61K31/205 , A61K47/48 , A61K9/00 , A61K45/06 , A61K31/198 , A61K31/385
CPC classification number: A61K38/51 , A61K9/0019 , A61K31/205 , A61K45/06 , A61K47/60 , C12N9/88 , C12N9/96 , C12Y402/01022 , A61K31/198 , A61K2300/00 , 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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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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18.
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:US10046036B2
公开(公告)日:2018-08-14
申请号:US15235355
申请日:2016-08-12
Inventor: Jan P Kraus , Tomas Majtan , Erez Bublil
IPC: A61K38/54 , A61K38/00 , C12N9/96 , C12N9/88 , C07H21/04 , C07K1/00 , A61K38/51 , A61K45/06 , A61K31/198 , A61K31/205 , A61K31/385 , A61K9/00 , A61K47/48
Abstract: The invention provides reagents and methods for enzyme replacement therapy using chemically modified species of human cystathionine β-synthase (CBS) to treat homocystinuria and other related diseases and disorders.
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