VECTORS COMBINING ANTI-SICKLING BETA-AS3-GLOBIN WITH ANTI BCEL11A SH RNA MIR TO TREAT BETA-HEMOGLOBINOPATHIES
Publication
EP4490302A2
Kind: A2
Mar 18, 2026
Applicants
The Regents of University of California, The Children's Medical Center Corporation, Dana-Farber Cancer Institute, Inc.
Inventors
KOHN, Donald B., HOLLIS, Roger Paul, HART, Kevyn, WILLIAMS, David, BRENDEL, Christian, LIU, Boya
IPC Classifications
C12N 15/867 20060101AFI20260210BHEP
A61K 38/42 20060101ALI20260210BHEP
A61P 7/06 20060101ALI20260210BHEP
C12N 15/113 20100101ALI20260210BHEP
C12N 5/00 20060101ALI20260210BHEP
Designated 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