“This is the first time we’ve been able to measure improvements in quality of life after treatment with gene editing technology,” said Josu de la Fuente, Ph.D., director of the Pediatric Blood and Marrow Transplantation Program at Imperial College London Healthcare NHS Trust, professor of practice (Cell & Gene Therapy) at Imperial College London, and lead author of the beta thalassemia study.

“Most research on new therapies like exa-cel tends to focus on improvements in laboratory values, but what is most important to patients with severe SCD or transfusion-dependent beta thalassemia are improvements in their daily life.”

Participants in both studies reported clinically meaningful improvements in overall quality of life, including physical, social/family, functional, and emotional well-being. Sustained improvements were observed starting as early as six months following exa-cel infusion.