Abstract: The present invention provides a method of PEGylating a human truncated cystathionine ?-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.
Type:
Grant
Filed:
April 17, 2018
Date of Patent:
May 10, 2022
Assignees:
THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE, TRAVERE THERAPEUTICS SWITZERLAND GMBH
Inventors:
Tomas Majtan, Jan P. Kraus, Erez M. Bublil, Frank Glavin, Marcia Sellos-Moura