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(11)

EP 4 816 540 A1

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(43) Date of publication: 07.10.2026 Bulletin 2026/41 (21) Application number: 24817296.7 (22) Date of filing: 29.11.2024

(51) International Patent Classification (IPC):  C12N 15/86(2006.01) A61K 48/00(2006.01) (52) Cooperative Patent Classification (CPC): C12N 15/86; C12N 2750/14143; A61K 47/26; A61K 9/0019; A61K 48/0041; A61K 47/40 (86) International application number: PCT/EP2024/084069 (87) International publication number: WO 2025/114524 (05.06.2025 Gazette 2025/23)

(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: GE KH MA MD TN (30) Priority: 30.11.2023 EP 23213403 (71) Applicant: uniQure biopharma B.V. 1105 BP Amsterdam (NL)	(72) Inventors: SUN, Feilong 1105 BP Amsterdam (NL) MATHEW, Simi 1105 BP Amsterdam (NL) BERTHIER, Eddy 1105 BP Amsterdam (NL) APETRI, Maria Mihaela 1105 BP Amsterdam (NL) LJUBOVIC-COUTEAU, Edina 1105 BP Amsterdam (NL) (74) Representative: Nederlandsch Octrooibureau  P.O. Box 29720 2502 LS The Hague 2502 LS The Hague (NL)