A Multi-Center, Phase 2 Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell (GRASP, BMT CTN 2001)

This study aims to determine if treatment with a single infusion of autologous CD34+ HSC cells transduced with the lentiviral vector containing shmiR targeting BCL11A will lead to a complete absence of severe vaso-occlusive events (VOEs) in the period from Month 6 to Month 24 after receiving the gene therapy in participants with sickle cell disease, with genotype HbSS or HbS/B0 thalassemia. 

Study treatment includes mobilization and collection of CD34+ HSPCs, myeloablative conditioning with busulfan administered on days -5 to -2 prior to infusion of gene therapy, and one 30-45 minute infusion after standard pre-hydration and premedication. After infusion of transduced cells, red cell transfusions will be utilized to maintain healthy and stable hemoglobin levels.


National Marrow Donor Program

Status of enrollment

Accepting new patients

Ages Eligible for Study

For people ages 13-40

Disease indication

Sickle Cell Anemia

Principal Investigator

Mark Walters, MD


Marci Moriarty, RN, BSN: [email protected]


Additional study eligibility details can be found at UCSF Clinical Trials.