(INND)
TREMTELEKTOGEN EMPOGEDITEMCEL(INNC)
ALLOGENEIC HUMAN CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS (HSPC) OBTAINED FROM PERIPHERAL BLOOD BY LEUKAPHERESIS FROM HUMAN LEUKOCYTE ANTIGEN (HLA)-MATCHED HEALTHY DONORS, GENETICALLY MODIFIED EX VIVO BY CRISPR/CAS9 (CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS (CRISPR)-ASSOCIATED PROTEIN-9 (CAS9)) NUCLEASE, COMPLEXED WITH A GUIDE RNA (GRNA) THAT BINDS TO A 20-BASE PAIR TARGET DNA SEQUENCE AT THE INTRON 2/EXON 3 BOUNDARY OF THE SIALIC ACID BINDING IG-LIKE LECTIN 3 (SIGLEC-3, CD33) GENE, WHICH INTRODUCES A PREMATURE STOP CODON AND DEGRADATION OF THE MRNA SUCH THAT NO FUNCTIONAL CD33 IS EXPRESSED. THE CELL SUSPENSION IS ENRICHED FOR CD34+ CELLS USING MAGNETIC BEAD SEPARATION. FOLLOWING ELECTROPORATION, THE CELLS ARE CULTURED IN MEDIA CONTAINING STEM CELL FACTOR (SCF), FMS-RELATED TYROSINE KINASE 3 LIGAND (FLT-3L), AND THROMBOPOIETIN (TPO). THE SUBSTANCE CONSISTS OF CELLS WITH MORE OR EQUAL TO 90% CD34 PURITY AND MORE OR EQUAL TO 50% ONTARGET EDITING OF CD33.(WHO)
TREMTELECTOGENUM EMPOGEDITEMCELUM(INN)