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EP 4 770 675 A1

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(43) Date of publication: 08.07.2026 Bulletin 2026/28 (21) Application number: 24861123.8 (22) Date of filing: 30.08.2024

(51) International Patent Classification (IPC):  A61K 47/14(2017.01) A61P 31/04(2006.01) A61P 15/02(2006.01) A61K 45/06(2006.01) (52) Cooperative Patent Classification (CPC): A61P 15/02; A61P 31/04; A61K 9/0034; A61K 31/20; A61K 31/201 (86) International application number: PCT/US2024/044631 (87) International publication number: WO 2025/049879 (06.03.2025 Gazette 2025/10)

(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: 01.09.2023 US 202363536186 P 28.03.2024 US 202463570876 P (71) Applicants: The General Hospital Corporation Boston, Massachusetts 02114 (US) The Broad Institute Inc. Cambridge, MA 02142 (US) Massachusetts Institute of Technology Cambridge, MA 02139-4301 (US)

(72) Inventors: KWON, Douglas S. Somerville, Massachusetts 02145 (US) BLAINEY, Paul Cambridge, Massachusetts 02142 (US) BLOOM, Seth M. Brookline, Massachusetts 02446 (US) ZHU, Meilin Cambridge, Massachusetts 02142 (US) (74) Representative: Maiwald GmbH  Elisenhof Elisenstraße 3 80335 München 80335 München (DE)