	US 11,857,641 B2
	Method for the treatment of mucopolysaccharidosis type I
Dale Ando, Richmond, CA (US); Cheryl Wong Po Foo, Richmond, CA (US); Sagar A. Vaidya, Richmond, CA (US); and Shelley Q. Wang, Richmond, CA (US)
Assigned to Sangamo Therapeutics, Inc., Brisbane, CA (US)
Filed by Sangamo Therapeutics, Inc., Richmond, CA (US); and Roselia C. Santana, Walnut Creek, CA (US)
Filed on Aug. 7, 2019, as Appl. No. 16/534,302.
Claims priority of provisional application 62/802,568, filed on Feb. 7, 2019.
Claims priority of provisional application 62/802,110, filed on Feb. 6, 2019.
Prior Publication US 2020/0246486 A1, Aug. 6, 2020
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 48/00 (2006.01); A61P 3/00 (2006.01); G01N 33/66 (2006.01); C12Q 1/34 (2006.01); A61K 38/47 (2006.01)

CPC A61K 48/005 (2013.01) [A61K 38/47 (2013.01); A61P 3/00 (2018.01); C12Q 1/34 (2013.01); C12Y 301/06013 (2013.01); C12Y 302/01076 (2013.01); G01N 33/66 (2013.01)]

31 Claims

1. A method of reducing, delaying and/or eliminating: the need for additional treatment procedures, the onset, progression and/or severity of symptoms in a subject with MPS I, the method comprising administering to the subject a composition comprising first, second and third AAV vectors, the first AAV vector comprising a sequence encoding a left ZFN comprising SEQ ID NO: 23, the second AAV vector comprising a sequence encoding a right ZFN comprising SEQ ID NO: 26 and the third AAV vector comprising a sequence encoding iduronidase (IDUA).