A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease

This study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (lovotibeglogene autotemcel, also known as LentiGlobin BB305 Drug Product for SCD, or bb1111), an autologous CD34+ cell-enriched population from patients with sickle cell disease that contains hematopoietic stem cells transduced with BB305 lentiviral vector encoding the β A-T87Q -globin gene. 

The main goal of this study is to evaluate the efficacy of treatment with bb1111 in subjects with severe sickle cell disease, defined as complete resolution of vaso-occlusive events (VOEs), between 6 months and 18 months after drug product infusion. Safety of the treatment will also be assessed. 

Status of enrollment

Not accepting new patients

Ages Eligible for Study

12-50 years of age

Genders Eligible for Study

Females and males

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.