Aug 25, 2026 —
Researchers at Boston Children’s Hospital have reported a first-in-human pilot trial of a new approach that streamlines stem cell collection for sickle cell disease gene therapy, potentially removing a major barrier to treatment.
Autologous gene therapy for sickle cell disease requires collecting large numbers of a patient’s hematopoietic stem cells — a lengthy process that can involve multiple hospital visits and deter patients from pursuing treatment. The new approach enabled collection of enough stem cells in a single hospital admission for most participants, improving the turnaround time for the subsequent steps of genetically modifying and reinfusing the cells.
By simplifying and shortening collection, the strategy could make ex vivo gene therapy more accessible for patients with sickle cell disease. The work was supported by the National Institutes of Health.
