Steven Ly Le

US Patent:

20210015906, Jan 21, 2021

Filed:

Jul 1, 2020

Appl. No.:

16/919104

Inventors:

- Torrance CA, US
Tsui-Fen Chou - Torrance CA, US
Sean Ekins - Fuquay-Varina NC, US
Shih-Hsin Kan - Torrance CA, US
Steven Le - Torrance CA, US
Derek R. Moen - Hermosa Beach CA, US

International Classification:

A61K 38/47
A61P 43/00
A61K 47/66
A61K 9/00

Abstract:

The present disclosure relates to compositions and methods for treating Sanfilippo syndrome (also known as Sanfilippo disease type D, Sanfilippo D, mucopolysaccharidosis type IIID, MPS IIID). The method can entail injecting to the spinal fluid of a MPS IIID patient an effective amount of a composition comprising a recombinant human acetylglucosamine-6-sulfatase (GNS) protein comprising the amino acid sequence of SEQ ID NO: 1 or an amino acid sequence having at least 90% sequence identity to SEQ ID NO: 1 and having the enzymatic activity of the human GNS protein. The composition can be provided in an artificial cerebrospinal fluid. About 1 mg to about 100 mg of the recombinant polypeptide may be administered to the patient once every 2 weeks to 6 months.