公开(公告)号：US20230039591A1

公开(公告)日：2023-02-09

申请号：US17713891

申请日：2022-04-05

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

IPC: A61K38/51 ,  A61K47/60 ,  C12N9/88

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.

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

IPC: A61K38/51 ,  A61K47/60 ,  C12N9/88

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.

公开(公告)号：US20220265835A1

公开(公告)日：2022-08-25

申请号：US17622112

申请日：2020-06-26

Applicant: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE

Inventor： Erez Bublil ,  Frank Glavin ,  Marcia Sellos-Moura ,  Jan P. Kraus ,  Tomas Majtan ,  Randy Wanner ,  Orhan Causevic

IPC: A61K47/60 ,  A61K47/26 ,  A61K47/02 ,  A61K9/19 ,  A61P43/00

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.

公开(公告)号：US20200261555A1

公开(公告)日：2020-08-20

申请号：US16605918

申请日：2018-04-17

Applicant: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE ,  ORPHAN TECHNOLOGIES, LTD.

Inventor： Tomas Majtan ,  Jan P. Kraus ,  Erez M. Bublil ,  Frank Glavin ,  Marcia Sellos-Moura

IPC: A61K38/51 ,  C12N9/88 ,  A61K47/60

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.