(19)

(11)

EP 4 680 741 A2

(12)

(88) Date of publication A3: 24.10.2024 (43) Date of publication: 21.01.2026 Bulletin 2026/04 (21) Application number: 24720350.8 (22) Date of filing: 14.03.2024

(51) International Patent Classification (IPC):  C12N 15/113(2010.01) (52) Cooperative Patent Classification (CPC): C12N 15/113; C12N 2320/33; C12N 2310/11; C12N 2320/32 (86) International application number: PCT/US2024/019995 (87) International publication number: WO 2024/192281 (19.09.2024 Gazette 2024/38)

(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: 15.03.2023 US 202363452393 P 16.03.2023 US 202363452630 P 16.05.2023 US 202363466934 P 21.08.2023 US 202363520787 P 23.10.2023 US 202363592393 P 16.01.2024 US 202463621465 P (71) Applicant: RegenxBio Inc. Rockville, MD 20850 (US)

(72) Inventors: FOLTZ, Steven Rockville, MD 20850 (US) QIAN, Randolph Rockville, MD 20850 (US) JIN, Lan Rockville, MD 20850 (US) LIU, Ye Rockville, MD 20850 (US) QIAO, Chunping Chapel Hill, NC 27514 (US) DANOS, Olivier Rockville, MD 20850 (US) (74) Representative: Appleyard Lees IP LLP  15 Clare Road Halifax HX1 2HY Halifax HX1 2HY (GB)