CTX001 is a type of cell therapy that uses the patient’s own blood stem cells (called CD34+ cells). These cells are modified using CRISPR-Cas9 technology, which is a gene-editing tool. The modified cells are then given back to the patient as a single infusion. This therapy is designed to help patients with severe sickle cell disease by altering the genes in their blood-forming cells to produce healthy red blood cells instead of the sickle-shaped cells that cause the disease.

Busulfan is typically used as a conditioning medication before cell therapy. It helps prepare the body to receive the modified stem cells by clearing space in the bone marrow. This allows the new, modified cells to establish themselves and begin producing healthy blood cells.

Plerixafor is commonly used in stem cell collection procedures. It helps mobilize stem cells from the bone marrow into the bloodstream, making it easier to collect these cells for modification.

Sickle Cell Disease (SCD) – A genetic blood disorder where red blood cells become crescent-shaped (like a sickle) instead of their normal round shape. This abnormal shape causes red blood cells to break down prematurely and can block small blood vessels. The disease is inherited when both parents pass on the abnormal hemoglobin gene to their child. People with SCD experience episodes called vaso-occlusive crises (VOCs), during which sickle-shaped cells block blood vessels, causing pain and potential organ damage. The condition typically manifests in early childhood and continues throughout life. The disease can cause anemia because the abnormal red blood cells don’t last as long as healthy ones.