TREMTELECTOGENE EMPOGEDITEMCEL

UNII:	CT5FM8JN3S
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	TREMTELECTOGENE EMPOGEDITEMCEL
GSRS Full Record:	CT5FM8JN3S i

Tremtelectogene Empogeditemcel
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 ≥90% CD34 purity and ≥50% on-target editing of CD33
tremtelectogene empogeditemcel [INN]

Note

UNIIs are generated based on scientific identity characteristics using ISO 11238 data elements. UNII availability does not imply any regulatory review or approval. Synonyms and mappings are based on the best public information available at the time of publication. Please report any problems/errors associated with this data to FDA-SRS@fda.hhs.gov.

TREMTELECTOGENE EMPOGEDITEMCEL

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