Ловотибеглоген аутотемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

autologous CD34+ hematopoietic stem cells (HSCs) obtained from mobilised peripheral blood, from patients with sickle cell disease. The cells are transduced with betibeglogene darolentivec, a self-inactivating human immunodeficiency virus (HIV-1)-derived lentiviral vector encoding a T87Q-mutated form of the human hemoglobin subunit beta (HBB, beta-globin) gene under the control of a human beta-globin promoter and a 3' beta-globin enhancer; the vector genome is flanked by 5' and 3' long terminal repeats (LTRs) and contains a ψ packaging signal, a Rev response element (RRE), and two polypurine tract (PPT) sequences. The CD34+ cell population was enriched using immunomagnetic microbeads, and then cultured in a stem cell growth medium, supplemented with recombinant human cytokines such as stem cell factor (SCF), FMS-related tyrosine kinase 3 ligand (Flt-3L) and thrombopoietin (TPO). The cells consist of CD34+ cells (≥70%), of which ≥56% express the transgene.

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