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EP 4 573 090 A1

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(43) Date of publication: 25.06.2025 Bulletin 2025/26 (21) Application number: 23765598.0 (22) Date of filing: 17.08.2023

(51) International Patent Classification (IPC):  C07D 413/14(2006.01) A61P 37/00(2006.01) A61P 29/00(2006.01) A61K 31/506(2006.01) (52) Cooperative Patent Classification (CPC): A61P 29/00; A61P 37/00; A61K 45/06; C07D 413/14 (86) International application number: PCT/US2023/072366 (87) International publication number: WO 2024/040155 (22.02.2024 Gazette 2024/08)

(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 ME MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA Designated Validation States: KH MA MD TN (30) Priority: 19.08.2022 EP 22315187 (71) Applicant: GENZYME CORPORATION Cambridge, Massachusetts 02141 (US)

(72) Inventors: DEFOSSA, Elisabeth 65926 Frankfurt am Main (DE) HEINELT, Uwe 65926 Frankfurt am Main (DE) RACKELMANN, Nils 65926 Frankfurt am Main (DE) MENDEZ PEREZ, Maria 65926 Frankfurt am Main (DE) MATTER, Hans 65926 Frankfurt am Main (DE) GRIESANG, Niels 65926 Frankfurt am Main (DE) SCHIELL, Matthias 65926 Frankfurt am Main (DE) PANCZEL, Jozsef 65926 Frankfurt am Main (DE) (74) Representative: Ipsilon NNY  11 rue Saint-Georges 75009 Paris 75009 Paris (FR)