Product Code: 26EL-662-2035
Please note: This is a Single Viewer eCast registration for one participant for this eCast for both the live and on-demand version of this eCast. If you are interested in presenting this program to a group, please see the “eCast Registration Form– Group Viewing” form on the eCast program page.
Access instructions for the live eCast will be provided via email approximately 2-3 business days prior to the live program date. Access instructions for the on-demand version of this program will be provided via email approximately 3-10 business days after the live program has occurred and the on-demand version is available.
Program Description: Gene therapy offers a revolutionary approach to treating a broad spectrum of diseases, with some therapies already FDA-approved and many more in clinical trials. This cutting-edge treatment often relies on apheresis to collect a substantial quantity of hematopoietic progenitor cells (HPCs), the essential starting material. These HPCs are then genetically modified outside the body (ex vivo) to create a therapeutic product. In patients with hemoglobinopathies, their underlying disease can affect mobilization and can make collection more challenging. Managing these challenges is crucial for successful gene therapy manufacturing and ultimately, for delivering effective treatments to patients with hemoglobinopathies.
This program will provide an overview of gene therapy for hemoglobinopathies like sickle cell disease and beta-thalassemia illustrating gene editing strategies. The faculty will also explore the challenges inherent in the complex logistics of preparing, mobilizing, and collecting these patients and discuss potential solutions for optimizing these processes.