The Non-Malignant Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
The Thalassemia Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
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.
