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EP 4 408 534 A1

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(43) Date of publication: 07.08.2024 Bulletin 2024/32 (21) Application number: 22873938.9 (22) Date of filing: 27.09.2022

(51) International Patent Classification (IPC):  A61P 27/02(2006.01) C12N 15/113(2010.01) A61K 9/00(2006.01) C12N 15/86(2006.01) (52) Cooperative Patent Classification (CPC): C12N 15/113; A61P 27/02; C12N 15/86; C12N 2800/40; C12N 2750/14143; C12N 15/102; C12N 2310/20 (86) International application number: PCT/US2022/077100 (87) International publication number: WO 2023/049931 (30.03.2023 Gazette 2023/13)

(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: KH MA MD TN (30) Priority: 27.09.2021 US 202163248808 P (71) Applicants: The Trustees of Columbia University in the City of New York New York, NY 10027 (US) The Broad Institute, Inc. Cambridge, MA 02142 (US) President And Fellows Of Harvard College Cambridge, Massachusetts 02138 (US)

(72) Inventors: QUINN, Peter M.J. New York, New York 10027 (US) LOPES DA COSTA, Bruna New York, New York 10027 (US) TSANG, Stephen H. New York, New York 10027 (US) LIU, David R. Cambridge, Massachusetts 02142 (US) (74) Representative: CH Kilger Anwaltspartnerschaft mbB  Fasanenstraße 29 10719 Berlin 10719 Berlin (DE)