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Curative Options in Sickle Cell Disease Plenary Session
Lovotibeglogene autotemcel (lovo-cel) Gene Therapy for Sickle Cell Disease
Jen Leiding, MD: No financial relationships to disclose
Learning Objectives:
Understand the key milestones of the lovo-cel clinical program, including clinical study primary endpoints, enrollment, and age range of participants.
Define the key phases of gene therapy with lovo-cel, including mobilization, apheresis, HSCT preparation, infusion.
Describe at a high level clinical data from HGB-206 Group C--results of which are supporting the submitted Biologics License Application (BLA); including efficacy outcomes (severe vaso-occlusive events [VOEs], safety (adverse and severe adverse events); and patient reported outcomes (measures of pain, fatigue, physical functioning.
.jpg "Jen Leiding, MD (she/her/hers) photo")