Тремтелектоген эмпогедитемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

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.

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