Patent SG-11201805598W-A (SG)
Title: Methods and compositions for treating hunter syndrome
Inventor: OKUYAMA TORAYUKI
IPC: A61K38/43
Filed: 2016-12-21
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization M11111011101010111110101111 101E3 01111 0011011111111101111011# 11 International Bureau .. .... ..Yejd (10) International Publication Number (43) International Publication Date ..... .....!;,,, WO 2017/116066 Al 6July 2017 (06.07.2017) WI P0 1 PCT (51) International Patent Classification: 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 A61K 38/43 (2006.01) A61K 9/00 (2006.01) (KR). (21) International Application Number: (74) Agent: DOOHO IP LAW FIRM; (Seocho-dong, Daehy- PCT/KR2016/015060 un Blue Tower) 7th Floor, 1, Gangnam-daero 51-gil, (22) International Filing Date: Seocho-gu, Seoul 06628 (KR). 21 December 2016 (21.12.2016) (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (25) Filing Language: English AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (26) Publication Language: English BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (30) Priority Data: HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, 62/272,843 30 December 2015 (30.12.2015) US KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, 62/369,970 2 August 2016 (02.08.2016) US MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (71) Applicants: GREEN CROSS CORPORATION NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, [KR/KR]; (Bojeong-dong) 107, Ihyeon-ro 30beon-gil, Gi- RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, heung-gu, Yongin-si, Gyeonggi-do 16924 (KR). MEDI- TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, GENEBIO CORPORATION [KR/KR]; (Bojeong-dong) ZA, ZM, ZW. 107, lhyeon-ro 30beon-gil, Giheung-gu, Yongin-si, (84) Designated States (unless otherwise indicated, for every Gyeonggi-do 16924 (KR). kind of regional protection available): ARIPO (BW, GH, (72) Inventors: OKUYAMA, Torayuki; 2-10-1, Okura, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, Setagaya-ku, Tokyo, Tokyo 157-8535 (JP). JIN, Thong- TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, — Gyu; E-3806, 57, Eonju-ro 30-gil, Seoul 06293 (KR). TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, BYUN, Han-Yeul; (Bojeong-dong) 107, Ihyeon-ro DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, = (KR). SEO, Jin-Wook; (Bojeong-dong) 107, Ihyeon-ro SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 GW, KM, ML, MR, NE, SN, TD, TG). (KR). LEE, Byoung-Ju; (Bojeong-dong) 107, Ihyeon-ro Published: = 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 — (KR). KIM, Yong-Chul; (Bojeong-dong) 107, lhyeon-ro with international search report (Art. 21(3)) = 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 — with sequence listing part of description (Rule 5.2(a)) = (KR). JANG, In-Young; (Bojeong-dong) 107, Ihyeon-ro = = = = = = = Title: METHODS AND COMPOSITIONS FOR TREATING HUNTER SYNDROME = (54) Brain GAG (D7) Brain GAG (D14) Brain GAG (D28) S' b. En 3 t - h. E 2 3 I E 2 1-1 .4 a g • • a 0 < Pt (.0 0 Il g , 0 • ti` ,e , ,,, , b ., , f t , , f , 0 c,' ''. 5 , ; . ' g 0 4' ,a , .,, , f , ,,,O , 4 ,4 , -4. , ,,,i ,; b • S , Q ‹> 41' e At` , ..,, , f° ,0% . 5 ,4 , -e .) 2 f , i e 11 KO mice KO nice KO mica Ir ... ' 1-1 (57) : The present invention provides, among other things, compositions and methods for CNS delivery of Idursulfase-beta, 0 a human recombinant iduronate-2-sulfatase protein, for effective treatment of Hunter Syndrome. The compositions and methods \" provided by the present invention effectively reduce symptoms not only in brain and spinal cord but also in peripheral tissues includ - 0 ing heart, liver, spleen, lung, and kidney.