Sickle Cell Gene Therapy 'Truly Transformative'

SAN DIEGO — A newly approved gene therapy product for sickle cell disease, lovotibeglogene autotemcel, led to durable disease remissions for up to 5 years and almost complete elimination of dangerous and debilitating vaso-occlusive events, according to results of a long-term follow-up study.

More specifically, a single infusion of lovo-cel led to complete resolution of vaso-occlusive events in 88% of patients, with 94% achieving complete resolution of severe events. All 10 adolescents in the study achieved complete resolution of vaso-occlusive events. Most patients remained free of vaso-occlusive events at their last follow-up.

"This is a one-time, truly transformative treatment with lovo-cel," lead author Julie Kanter, MD, director of the adult sickle cell clinic at the University of Alabama in Birmingham, said in a media briefing here at the American Society of Hematology (ASH) 2023 annual meeting. The gene therapy can essentially eliminate vaso-occlusive events in patients with sickle cell disease and lead to normal hemoglobin levels, Kanter added. 

Source: MEDspace