A Phase 1/2 Study of nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease

In this study includes studying nula-cel (nulabeglogene autogedtemcel, also referred to as KMAU001) in sickle cell disease (SCD). Some of your blood stem cells will be collected and sent to a lab for gene correction and editing. The lab will use a process to change the mutated beta-globin gene into a normal beta-globin gene in the blood stem cells. The corrected cells are called nula-cel , which is the study drug product (DP) in this study. Your DP is made only for you. It will not be given to anyone else. The purpose of this study is to find out whether this gene correction process improves or prevents the symptoms and organ damage that result from SCD. This study is the first to evaluate nula-cel (DP) in humans and has not been approved by the Food and Drug Administration (FDA).

Are you Eligible? (Inclusion Criteria)

• Must be 18 years old or older. - Must be 40 year old or less. - Must not have an available 10/10 HLA-matched sibling donor