The 2022 Tandem Meetings | Benefits of beti-cel gene therapy for transfusion-dependent β-thalassemia
Andreas E. Kulozik, MD, PhD, Hopp-Children’s Cancer Research Center (KiTZ), University of Heidelberg, Heidelberg, Germany, outlines the advantages of betibeglogene autotemcel (beti-cel) lentiviral gene therapy over gammaretroviral gene therapy in transfusion-dependent β-Thalassemia. Lentiviral gene therapy reduces the risk of insertional mutagenesis as it integrates randomly in the genome rather than in transcriptionally active regions. In addition, beti-cel contains the endogenous promoter and regulatory regions of the beta-globin gene, which limits the risk of activating genes close to the integration sites. This interview took place at the Transplantation & Cellular Therapy (TCT) Meetings of ASTCT™ and CIBMTR® 2022 in Salt Lake City, Utah.
