Дазагамглоген аутогедтемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) collected by leukapheresis and genetically modified ex vivo by a pair of zinc finger nucleases (ZFN) directed against the genomic sequence of the erythroid- specific enhancer (ESE) located in the second intron of the B-cell lymphoma/leukemia 11A (BCL11A) gene on chromosome 2. The ZFN editing components comprise two messenger RNAs (mRNAs) with one mRNA encoding the left optimized ZFN (mRENH1), and the other mRNA encoding the right optimized ZFN (mRENH2). The two mRNAs are introduced ex vivo into the target cell population via electroporation. Subsequent endogenous non- homologous end joining DNA repair results in an insertion or deletion (indel) event thus permanently disrupting the BCL11A erythroid enhancer region.
The cell suspension is enriched for CD34+ cells using magnetic bead separation and cultured in media containing Fms-related tyrosine kinase 3 ligand (Flt3L), stem cell factor (SCF), and thrombopoietin (TPO), prior to electroporation.
The substance consists of cells with ≥80% CD34+ purity and detectable on-target editing

Поделиться этой страницей