First-in-human pilot trial improves stem cell collection and delivery for sickle cell gene therapy

Patients with sickle cell disease are often hesitant to undergo transformative gene therapy—the current lengthy process requires multiple hospital visits to collect enough stem cells. Researchers from Boston Children's Hospital have helped smooth the road to gene therapy with a new approach that collects enough of the patient's stem cells in a single hospital admission for most patients, improving the turnaround time for genetically altering and infusing the cells back into the patient. The long-term follow-up results of the first-in-human phase I pilot clinical trial were recently published in Blood.