Imetelstat is in clinical development for the treatment of relapsed/refractory low or intermediate-1 risk myelodysplastic syndrome (MDS) in transfusion-dependent patients, following erythropoiesis-stimulating agent (ESA) treatment. MDS are a group of disorders in which red blood cells, white blood cells, and platelets produced by the bone marrow do not grow and mature normally. MDS are long-term debilitating and life-threatening diseases. MDS patients may require repeated blood transfusions and currently have few treatment options.
Imetelstat sodium is an intravenously administered drug that works by blocking the activity of an enzyme called telomerase. Telomerase is involved in regulating cell growth and division. By blocking the activity of telomerase, this medicine is expected to stop the uncontrolled division of abnormal immature blood cells, therefore slowing the progression of MDS. If licensed, imetelstat will provide an additional treatment option for relapsed/refractory low or intermediate-1 risk MDS in transfusion-dependent patients, following ESA Treatment.
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 …