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.