Preparative Apheresis and Transfusion Support for Sickle Cell Disease Gene Therapy (25EL-527)

For many patients with sickle cell disease (SCD), hematopoietic stem cell transplantation is unavailable due to lack of a matched donor. Gene therapy using autologous gene-edited stem cells has emerged as an option for these patients. Two gene therapy regimens have been approved by the FDA and additional clinical trials are underway. This program will provide background on gene therapy for SCD and an update on the status of SCD gene-editing clinical trials. The program will also address challenges in preparing products for patients enrolled in these trials and for the transfusion services providing transfusion support.

Presented by

Elizabeth St. Lezin, MD
Director/Moderator
Clinical Professor of Laboratory Medicine Emeritus, UCSF
San Francisco, CA

John Manis, MD
Speaker
Associate Professor Pathology, Harvard Medical School & Boston Children’s Hospital
Boston, MA

Learning objectives

After participating in this educational activity, participants should be able to:

• Demonstrate understanding of the role of gene therapy and gene-editing in the treatment of SCD.
• Identify current FDA-approved gene therapy regimens and key gene-editing clinical trials in SCD.
• Apply understanding of preparative transfusions for mobilization and collection of stem cells for SCD cell therapies.

Registration

Registration includes access to both the live and on-demand version of this eCast. Registration for the live eCast will close 1 hour prior to the eCast start time. If you register after this time, you will receive access to the on-demand eCast when it is available.

For additional information and to register as a single viewer or group, visit the AABB event webpage. Please note: Registration fees are required for AABB events.