AM20-30: Supporting Genetic Therapies for Sickle Cell Disease (ASH/AABB Joint Session)

Description

Presented in collaboration with Be The Match BioTherapies®

Cellular therapies for treating SCD are expanding at a rapid pace, with multiple approaches in trials and under development. Most of the therapeutic approaches use stem cell transplantation with genetically modified HSC, to generate red cells resistant to sickling. Emerging therapies include gene addition of beta globin, Bcl11a modification as well as other gene and base editing techniques being performed at both academic and industry research laboratories. While there are promising early results from clinical trials using gene therapy, more work is required to understand how to optimize stem cell collection, transplantation and engraftment.

To develop novel cell therapies, a concerted effort to link clinicians, patients, researchers and industry to move the field forward. In our session, we will review the current landscape of SCD therapies and to discuss how the initiative functions to identify and support initiatives. We will discuss the therapeutic clinical trials and review the clinical indicators used to assess successful outcomes. Lastly, we will discuss preparative transfusion therapies to optimize mobilization and collection of stem cells and to improve outcome of stem cell transplantation. The latter therapies heavily rely on the expertise of transfusion medicine specialists. Together, this presentation seeks to demonstrate the multimodal approaches of cell therapy for sickle cell disease.

The target audience is wide and includes: 

• Blood bank technicians and managers to understand transfusion needs
• Apheresis clinicians who perform red cell exchange and stem cell collection
• Hematologists and Stem Cell Transplant clinicians interested in trials and clinical therapeutic endpoints
• Patients and their advocates to understand the multi-modal nature of the novel cell therapies
• Scientific program managers that allocate resources and balance efforts within an initiative

Learning Objectives:

• Appreciate the wide range of participants to effect novel cell therapies for sickle cell disease
• Update of clinical trials and assess outcomes of clinical trials for sickle cell disease
• Review transfusion therapies and stem cell collection for cellular therapy of sickle cell disease

Speaker(s):

• Dr. John Manis, MD, Boston Children's Hospital
• Dr. Mark Walters, MD, UCSF Benioff Chidren's Hospital
• Lis Welniak, NHLBI

Disclosures

• John Manis, MD : Consultant: SQZ Biotech, Sanofi
• Mark Walters, MD : Consultant: Veevos, Editas Medical; Medical Director: AllCells Inc.
• Lis Welniak : Nothing to disclose