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(11)

EP 3 389 671 A1

(12)

(43) Date of publication: 24.10.2018 Bulletin 2018/43 (21) Application number: 16876499.1 (22) Date of filing: 13.12.2016

(51) International Patent Classification (IPC):  A61K 31/7088(2006.01) C12N 15/113(2010.01) C12N 15/11(2006.01) C12Q 1/68(2018.01) (86) International application number: PCT/US2016/066414 (87) International publication number: WO 2017/106210 (22.06.2017 Gazette 2017/25)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: MA MD (30) Priority: 14.12.2015 US 201562267210 P (71) Applicants: COLD SPRING HARBOR LABORATORY Cold Spring Harbor, NY 11724 (US) Stoke Therapeutics, Inc. Bedford, Massachusetts 01730 (US)

(72) Inventors: AZNAREZ, Isabel Cambridge Massachusetts 02139 (US) NASH, Huw M. Lexington Massachusetts 02421 (US) (74) Representative: HGF Limited  1 City Walk Leeds LS11 9DX Leeds LS11 9DX (GB)