According to a story from Business Wire, the drug developer Bioverativ, Inc., recently announced its intention to present data related to its latest developments in blood disorder therapies. This data will relate to projects focused on the treating hemophilia and sickle cell anemia. This information will be presented at the Annual Meeting of the American Society of Hematology, which is slated to take place in San Diego from December 1st – 4th. Bioverativ is committed to developing innovative therapies for rare blood disorders.
About Sickle Cell Anemia
Sickle cell anemia is a genetic, inherited disorder of the blood. This disorder is characterized by an abnormality affecting hemoglobin, which is found in red blood cells and is responsible for carrying oxygen. The abnormality causes blood cells to lose their typical circular shape and instead become take on an elongated, sickle-like appearance. Symptoms begin to appear at around six months old and include swelling of the hands and feet, stroke, bacterial infections, and acute episodes of severe pain termed sickle cell crisis. Life expectancy is between 40 and 60.
Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures.
Presentations will include data from an ongoing clinical trial of BIVV001, and experimental factor VIII therapy for hemophilia A. This therapy has the potential to provide protection from bleeding for a longer period than current therapies, with dosing necessary only once per week or possibly even less frequently.
Another presentation will focus on a long term effectiveness in safety study of ELOCTATE® and ALPROLIX.® These treatments are intended for hemophilia A and hemophilia B, respectively. These studies have occurred over a period of four years and highlight the long term effectiveness and safety of these two products.
ELOCTATE will also be featured in another presentation which will focus on the drugs use in immune tolerance induction for hemophilia A patients with inhibitors. Inhibitors prevent recombinant clotting factors from working for patients, so addressing this problem is a serious need for this patient community.
Bioverativ will also present early, pre-clinical data focusing on another one of its experimental therapies, BIVV003. This gene editing cell therapy intended to treat sickle cell anemia has been accepted for clinical trials by the US Food and Drug Administration.