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,
