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EP 3 452 045 A1

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(43) Date of publication: 13.03.2019 Bulletin 2019/11 (21) Application number: 17793335.5 (22) Date of filing: 04.05.2017

(51) International Patent Classification (IPC):  A61K 31/519(2006.01) A61P 25/08(2006.01) A61P 25/18(2006.01) A61P 25/28(2006.01) A61P 15/00(2006.01) A61P 25/16(2006.01) A61P 25/24(2006.01) A61P 43/00(2006.01) (86) International application number: PCT/US2017/031044 (87) International publication number: WO 2017/192841 (09.11.2017 Gazette 2017/45)

(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: 04.05.2016 US 201662331539 P (71) Applicant: Lysosomal Therapeutics Inc. Cambridge, MA 02139 (US)	(72) Inventors: SKERLJ, Renato, T. West Newton, MA 02465 (US) LANSBURY, Peter, T. Brookline, MA 02445 (US) BOURQUE, Elyse Marie, Josee L'etang-du-nord, Quebec G4T 3G6 (CA) GOOD, Andrew, C. Wallingford, CT 06492 (US) (74) Representative: Graham Watt & Co LLP  St. Botolph's House 7-9 St. Botolph's Road Sevenoaks TN13 3AJ Sevenoaks TN13 3AJ (GB)