CTX001 for severe sickle cell disease

CTX001 is in clinical development for patients with severe sickle cell disease (SCD). SCD is a group of inherited disorders where the red blood cells become hard and sticky and look like a C-shaped farm tools called a “sickle” and these sickle red blood cells die early. Individuals with SCD have inherited the gene for sickle haemoglobin from one parent and a gene for an abnormal haemoglobin variant from their other parent. Sickle-shaped red blood cells do not flow easily through the blood vessels and can cause episodic blockages (vaso-occlusive crises) in different parts of the body. Currently the only curative treatment option available for severe SCD patients is a stem cell transplant from a donor but many people will require lifelong treatment. Management focuses on reducing the chances of experiencing a sickle cell crisis by avoiding dehydration, sudden changes in temperature and infection.
CTX001 is an ex vivo CRISPR investigational gene-edited therapy that is administered intraveneously. The patient’s haematopoietic stem cells are genetically engineered to produce high levels of fetal haemoglobin (HbF) in red blood cells. The elevation of HbF by CTX001 has the potential to reduce painful and debilitating sickle crises for SCD patients. If licensed CTX001 will offer a treatment for patients with severe SCD.