HDAd-SB200x

HDAd-SB200x is a helper-dependent adenoviral (HDAd) vector that expresses SB200x, an activity-enhanced version of the Sleeping Beauty (SB) transposase. Developed for in vivo hematopoietic stem cell (HSC) gene therapy, it is part of a dual-vector system where HDAd-SB200x provides the transposase required to integrate a second, transposon-carrying HDAd vector into the host genome. This approach aims to treat hemoglobinopathies, such as sickle cell disease and beta-thalassemia, by enabling stable transgene expression in HSCs without the need for ex vivo manipulation or myeloablative conditioning. SB200x offers significantly higher integration efficiency compared to the earlier SB100x version, potentially reducing the number of in vivo selection cycles needed to achieve therapeutic levels of gene correction.