(19)

(11)

EP 3 843 761 A1

(12)

(43) Date of publication: 07.07.2021 Bulletin 2021/27 (21) Application number: 19855584.9 (22) Date of filing: 30.08.2019

(51) International Patent Classification (IPC):  A61K 35/76(2015.01) C12N 15/00(2006.01) C12N 5/02(2006.01) C12N 7/01(2006.01) A61P 35/04(2006.01) (86) International application number: PCT/US2019/049047 (87) International publication number: WO 2020/047398 (05.03.2020 Gazette 2020/10)

(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 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: 31.08.2018 US 201862725809 P (71) Applicant: The Research Institute at Nationwide Children's Hospital Columbus, Ohio 43205 (US)	(72) Inventors: CASSADY, Kevin A. Columbus, Ohio 43212 (US) ROTH, Justin C. Birmingham, Alabama 35209 (US) (74) Representative: Mewburn Ellis LLP  Aurora Building Counterslip Bristol BS1 6BX Bristol BS1 6BX (GB)