	US 11,986,515 B2
	Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS)
Stephen Joseph Pakola, Irvington, NY (US); Paulo Falabella, Potomac, MD (US); and Marie-Laure Nevoret, Virginia Beach, VA (US)
Assigned to REGENXBIO Inc., Rockville, MD (US)
Filed by REGENXBIO Inc., Rockville, MD (US)
Filed on Jul. 21, 2023, as Appl. No. 18/356,961.
Application 18/356,961 is a continuation of application No. PCT/US2022/014526, filed on Jan. 31, 2022.
Claims priority of provisional application 63/256,805, filed on Oct. 18, 2021.
Claims priority of provisional application 63/242,250, filed on Sep. 9, 2021.
Claims priority of provisional application 63/210,610, filed on Jun. 15, 2021.
Claims priority of provisional application 63/180,361, filed on Apr. 27, 2021.
Claims priority of provisional application 63/148,093, filed on Feb. 10, 2021.
Prior Publication US 2024/0058426 A1, Feb. 22, 2024
Int. Cl. A61P 25/28 (2006.01); A61K 38/46 (2006.01); C12N 15/86 (2006.01); G01N 33/53 (2006.01)

CPC A61K 38/465 (2013.01) [A61P 25/28 (2018.01); C12N 15/86 (2013.01); C12Y 301/06013 (2013.01); G01N 33/5308 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01); G01N 2400/00 (2013.01); G01N 2800/04 (2013.01)]

30 Claims

1. A method of treating mucopolysaccharidosis II (MPS II) in a human subject, the method comprising:

a) measuring the level of D2S6 present in a biological sample obtained from the human subject;

b) diagnosing the human subject with MPS II when the level of D2S6 in the biological sample is higher than a reference; and

c) delivering an effective amount of an active human iduronate-2-sulfatase (hIDS) to the central nervous system (CNS) of the human subject diagnosed with MPS II, wherein the method further comprises calculating a ratio of D2S6 to total heparan sulfate disaccharides (HS) in the biological sample, wherein the total HS comprises disaccharides D2S6, D0A0, D0S0, and D0A6.