US 11,771,745 B2
Compositions and methods for treatment of homocystinuria
Jan P. Kraus, Denver, CO (US); Tomas Majtan, Aurora, CO (US); and Erez Bublil, Ets Efraim (IL)
Assigned to THE REGENTS OF THE UNIVERSITY OF COLORADO, Denver, CO (US)
Filed by THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE, Denver, CO (US)
Filed on Jun. 29, 2021, as Appl. No. 17/362,633.
Application 17/362,633 is a continuation of application No. 16/824,931, filed on Mar. 20, 2020, granted, now 11,077,175.
Application 16/824,931 is a continuation of application No. 15/774,755, granted, now 10,653,755, issued on May 19, 2020, previously published as PCT/US2016/061050, filed on Nov. 9, 2016.
Prior Publication US 2022/0125896 A1, Apr. 28, 2022
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 38/51 (2006.01); A61K 9/00 (2006.01); A61K 31/198 (2006.01); A61K 31/205 (2006.01); A61K 31/385 (2006.01); A61K 45/06 (2006.01); A61K 47/60 (2017.01); C12N 9/88 (2006.01); C12N 9/96 (2006.01); A61P 19/10 (2006.01); A61P 43/00 (2006.01); A61P 13/02 (2006.01); A61P 17/14 (2006.01); A61P 27/02 (2006.01)
CPC A61K 38/51 (2013.01) [A61K 9/0019 (2013.01); A61K 31/198 (2013.01); A61K 31/205 (2013.01); A61K 31/385 (2013.01); A61K 45/06 (2013.01); A61K 47/60 (2017.08); C12N 9/88 (2013.01); C12N 9/96 (2013.01); C12Y 402/01022 (2013.01); A61K 2300/00 (2013.01)] 18 Claims
 
1. A method of treating homocystinuria in a human subject comprising administering an isolated human truncated cystathionine beta synthase (htCBS) mutant polypeptide, wherein:
(a) the isolated htCBS mutant polypeptide comprises amino acids 2-413 of the amino acid sequence of SEQ ID NO: 13 and has been PEGylated with an ME-200GS PEG molecule, and
(b) the isolated htCBS mutant polypeptide is administered in a dose between 0.33 mg/kg and 10 mg/kg at least once a week for at least six weeks.