TREATMENT OF MUCOPOLYSACCHARIDOSIS II WITH RECOMBINANT HUMAN IDURONATE-2 SULFATASE (IDS) PRODUCED BY HUMAN NEURAL OR GLIAL CELLS

摘要

ITR Chicken -actin intron tintglifiditionat&. Cytomegalovirus Immediate-early enhancer Chicken p-actin promoter Rabbit p- globin poly A signal FIG. 5 ITR (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 18 October 2018 (18.10.2018) W I PO I PCT 0111111010 0111 °nolo mu Oil IIIII Kuno oimIE (10) International Publication Number WO 2018/191666 Al (51) International Patent Classification: A61K 38/43 (2006.01) A61P 25/00 (2006.01) A61K 47/26 (2006.01) Cl 2N 15/86 (2006.01) A61K 48/00 (2006.01) (21) International Application Number: PCT/US2018/027568 (22) International Filing Date: 13 April 2018 (13.04.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/485,659 14 April 2017 (14.04.2017) US 62/573,921 18 October 2017 (18.10.2017) US 62/574,355 19 October 2017 (19.10.2017) US 62/579,686 31 October 2017 (31.10.2017) US (71) Applicant: REGENXBIO INC. [US/US]; 9712 Medical Center Drive, Suite 100, Rockville, MD 20850 (US). (72) Inventors: YOO, Stephen; 5521 Southwick Street, Bethes- da, MD 20817 (US). REINHARDT, Rickey, Robert; 108 Apple Grove Road, Silver Spring, MD 20904 (US). SIM- PSON, Curran, Matthew; 2149 Thurston Road, Frederick, MD 21704 (US). WU, Zhuchun; 14716 Soft Wind Drive, North Potomac, MD 20878 (US). (74) Agent: ELBERT, Maya et al.; Jones Day, 250 Vesey Street, New York, NY 10281-1047 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, 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, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a)) (54) Title: TREATMENT OF MUCOPOLYSACCHARIDOSIS II WITH RECOMBINANT HUMAN IDURONATE-2 SULFATASE (IDS) PRODUCED BY HUMAN NEURAL OR GLIAL CELLS CB7 promoter (57) : Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).