On December 12, we hosted an R&D Day in New York City. Alnylam management and key opinion leaders discussed the latest progress as well as plans for the future development of our RNAi therapeutics pipeline. At this event, we announced our pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease.
Alnylam has a number of other programs in its genetic medicine pipeline, including the development of an RNAi therapeutic for beta-thalassemia and iron overload disorders, as well as an RNAi therapeutic for primary hyperoxaluria (PH1).
Beta-thalassemia is an inherited blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body. In people with beta-thalassemia, insufficient hemoglobin, also known as anemia, leads to a lack of oxygen in many parts of the body.
Reduced hemoglobin production also leads to iron overload, a condition in which the body absorbs excessive amount of iron from the diet. Affected individuals suffer from weakness, fatigue, organ failure, and other more serious complications. People with beta thalassemia are also at an increased risk of developing abnormal blood clots and skeletal deformities.
In general, iron overload is an acquired or inherited disorder where the body absorbs excessive amounts of iron from food and drink. In beta-thalassemia, it is one of the primary causes of disease severity. In beta-thalassemia patients with moderate anemia (Thalassemia Intermedia), iron overload results from excess dietary iron uptake. In beta-thalassemia patients with severe anemia (Thalassemia Major), the need for chronic blood transfusions further exacerbates iron overload and accounts for the majority of excess iron accumulation. Iron overload can damage the liver, heart, pancreas, and other organs, ultimately leading to severe morbidity and increased mortality.