Patent SG-11201805680S-A (SG)
Title: Methods and compositions for the treatment of neurologic disease
Inventor: DEKELVER RUSSELL
IPC: A61P3/00
Filed: 2017-01-12
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization MD 110111101110101011111 HO 11111011101110111100 11110011111110111011# International Bureau .. .... ..Yejd ..... .....!;,,, (10) International Publication Number (43) International Publication Date WO 2017/123757 Al 20 July 2017 (20.07.2017) WIPO I PCT (51) (21) (22) (25) Filing Language: (26) Publication Language: (30) (71) Applicants: (72) International International International [US/US]; fornia ter, Inventors: peutics, Patent Classification: (US). A61K 48/00 (2006.01) C12N 9/40 (2006.01) sity A61P 3/00 (2006.01) C12N 15/09 (2006.01) Street C12N 5/10 (2006.01) (74) Agent: PASTERNAK, Dahna; Pasternak Patent Law, Application Number: 1900 PCT/1J52017/013189 94303 Filing Date: (81) Designated 12 January 2017 (12.01.2017) kind AO, English BZ, English DO, HN, Priority Data: KP, 62/279,394 15 January 2016 (15.01.2016) US MD, 62/300,271 26 February 2016 (26.02.2016) US NI, 62/328,925 28 April 2016 (28.04.2016) US RU, SANGAMO THERAPEUTICS, INC. TH, 501 Canal Blvd., Suite A100, Richmond, Cali- ZA, ZM, ZW. 94804 (US). REGENTS OF THE UNIVERSITY (84) Designated OF MINNESOTA [US/US]; 600 McNamara Alumni Cen- kind 200 Oak Street SE, Minneapolis, Minnesota 55455- GM, 2020 (US). TZ, DEKELVER, Russell; c/o Sangamo Thera- TJ, Inc., 501 Canal Blvd., Suite A100, Richmond, DK, California 94804 (US). MCIVOR, R. Scott; c/o Regents LV, of the University of Minnesota, 600 McNamara Alumni SM, Center, 200 Oak Street SE, Minneapolis, Minnesota GW, KM, ML, MR, NE, SN, TD, TG). 55455-2020 (US). OU, Li; c/o Regents of the University of Published: Minnesota, 600 McNamara Alumni Center, 200 Oak Street SE, Minneapolis, Minnesota 55455-2020 (US). with WECHSLER, Thomas; c/o Sangamo Therapeutics, Inc., 501 Canal Blvd., Suite A100, Richmond, California 94804 WHITLEY, Chester B.; c/o Regents of the Univer- of Minnesota, 600 McNamara Alumni Center, 200 Oak SE, Minneapolis, Minnesota 55455-2020 (US). Embarcadero Rd., Suite 211, Palo Alto, California (US). States (unless otherwise indicated, for every of national protection available): AE, AG, AL, AM, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, States (unless otherwise indicated, for every of regional protection available): ARIPO (BW, GH, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, international search report (Art. 21(3)) = = = _ = = = = = = = = ei disease. Title: METHODS AND COMPOSITIONS -a e Figure I -month so 24?? FOR THE TREATMENT OF 1A 4-month NEUROLOGIC DISEASE 1--Mid type, Formuladon 2 ?RS I rink, Yonnunaion 3 MPS I cl ..,:t! 'ormladon 4 MPS' . n. , ZINiDonot 5 MPS I Auto. EFN , Dottor 6 - MPS I, Donor Only — (54) = _ s-1 0') IN M ei Il IN 1-1 (57) 0 such : as 1 2 3 4 5 6 1 2 3 4 5 Group Group Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic IDUA or IDS protein an enzyme into a cell, thereby providing proteins or cell therapeutics 6 for treatment and/or prevention of MPS I or MPS II O