Zahara & Kesha Weeks

Gene therapies are redefining possibilities for people living with Sickle Cell Disease (SCD). For many years, care focused on managing symptoms. Today, gene therapies offer the potential to address the disease at its source. Safe, effective, and widely available cures aren’t just a scientific milestone, they’re a pathway to improved quality of life for people living with SCD.

Cure Sickle Cell Initiative (CureSCi) activities focus on:

  • Identifying and prioritizing optimal ways to assess patient readiness and resiliency pre-gene therapy, and to provide psychosocial support during and post-gene therapy.
  • Studying the safety and clinical benefits of potential gene therapies through patient-centric clinical trials.
  • Ensuring access to best practices in apheresis for the preparation and collection of healthy, viable stem cells for gene therapies.
  • Evaluating the risk of clonal hematopoiesis and myelodysplastic syndromes/acute myeloid leukemia to better align patients to the right SCD treatment.
  • Addressing gene therapy long-term follow-up by not only tracking adverse events but also studying treatment benefits, safety, and durability of gene therapies.

Learn about CureSCi research studies that are advancing gene therapies by viewing the Publications Page.