INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 08 November 2018 (08.11.2018) WIPO I PCT ~ill IIIIIIIIImillion ono o Holonmoinoiliflom oimIE (10) International Publication Number WO 2018/204786 Al (51) International Patent Classification: A61K 31/713 (2006.01) Cl 2N 15/11 (2006.01) A61K 48/00 (2006.01) Cl 2N 15/113 (2010.01) A61P 25/28 (2006.01) Cl 2N 15/86 (2006.01) (21) International Application Number: PCT/US2018/031089 (22) International Filing Date: 04 May 2018 (04.05.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/501,788 05 May 2017 (05.05.2017) US 62/507,927 18 May 2017 (18.05.2017) US 62/520,100 15 June 2017 (15.06.2017) US 62/566,609 02 October 2017 (02.10.2017) US (71) Applicant: VOYAGER THERAPEUTICS, INC. [US/US]； 75 Sidney Street, Cambridge, MA 02139 (US). (72) Inventors: SAH, Dinah, Wen-Yee； 75 Sidney Street, Cam- bridge, MA 02139 (US). CHEN, Qingmin； 75 Sidney Street, Cambridge, MA 02139 (US). HOU, Jinzhao； 75 Sidney Street, Cambridge, MA 02139 (US). 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)) — before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amen
