Luspatercept is under clinical development for the treatment of adult patients with beta-thalassaemia who don’t regularly require blood transfusion. Thalassaemia is a commonly inherited blood disorder resulting from an abnormality in one of the genes that affects the production of haemoglobin, a protein in red blood cells that carries oxygen throughout the body . Beta-thalassaemia is a subtype caused by a specific gene mutation. People with thalassaemia produce either little or no normal haemoglobin. Current treatment options for beta-thalassaemia are limited to blood transfusions with its associated risks and complications.
Luspatercept, administered via subcutaneous injection, is a recombinant engineered protein designed to attach to certain proteins that slow down the maturation of red blood cells. This leads to the production of healthy red blood cells and increased haemoglobin levels, leading to improved symptoms in patients with beta-thalassaemia intermedia and major. Luspatercept is a novel approach for treating anaemia, with potential to improve many patients’ lives by reducing or eliminating the need for frequent and lifelong blood transfusions.
OTL-103 is administered intravenously. It is made up of immature bone marrow cells (called CD34+ cells) taken from the patient. It works by correcting cells using a modified virus that contains the correct gene for the WAS protein. When these corrected cells are transplanted back into the patient, they populate the bone marrow and produce healthy platelets and immune cells that produce the WAS protein, thereby relieving the symptoms of the disease. If licensed, OTL-103 will provide a treatment option for patients with WAS.