Hemoglobinopathies are among the most prevalent monogenic disorders. Hematopoietic stem cells (HSCs) transduced with a lentiviral vector (LVV) encoding an anti-sickling globin are being evaluated in clinical trials for beta-thalassemia and sickle cell disease. To ensure drug product quality, it is important to demonstrate consistent LVV potency in manufactured lots.<br/><br/>In this study, we present the development of LVV potency assays. qPCR was used to determine average LVV copy number from transduced healthy human donor CD34+ cells. Transduced CD34+ cells were cultured to stimulate erythroid differentiation and liquid chromatography was used to determine the amount of β-A<sup>T87Q</sup> in erythroid differentiated cells (enucleated). Both assays report percent potency relative to a well-characterized LVV reference standard. These quantitative results demonstrate consistent manufacture of LVV.

Quantitative analysis of lentiviral vector potency for gene therapy in hemoglobinopathies

2019 Spring National Meeting

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