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Ropeginterferon alfa-2b for injection is under development for the treatment of polycythaemia vera (PV), a rare blood disease in which the body makes too many red blood cells. The extra red blood cells make the blood thicker than normal and as a result, blood clots can form more easily. These clots may block blood flow through arteries and veins, which can cause a heart attack or stroke. Thicker blood also does not flow as quickly and may prevent organs from getting enough oxygen. A mutation, or change, in a gene called JAK2 is the major cause of PV. This gene makes a protein that helps the body produce blood cells. PV develops slowly and may not cause symptoms for years. PV has no cure, but treatments can help control the disease and its complications.

Sutimlimab is a first-in-class monoclonal antibody in development for the treatment of cold agglutinin disease, a rare form of autoimmune haemolytic anaemia, caused by cold-reacting autoantibodies. These antibodies bind to red blood cell membranes and destroy them, leading to anaemia. Symptoms include chronic debilitating fatigue, acrocyanosis, shortness of breath and other anaemia-related symptoms, leading to a poor quality of life and increased health resource utilization. In chronic cold agglutinin disease, the patient is more symptomatic during winter months. Therapeutic options may range from using warm clothing and avoiding exposure to cold weather to blood transfusions and chemotherapy.

Givosiran is made of a short, synthetic strand of genetic material called ‘small interfering RNA’ (siRNA) that has been designed to interfere with the production of an enzyme involved in an early step in making haem. By blocking this early step of haem production in patients with AHP, givosiran is expected to prevent the next steps which produce substances that accumulate in the body and cause the symptoms of the disease.

Luspatercept 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-thalassemia 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.

The amino acid L-glutamine has been developed as an oral powder formulation to reduce the acute complication of sickle cell disease. It is thought L-glutamine works by reducing cell inflammation and promote the cellular uptake of oxygen. If licensed, L-glutamine oral powder may offer an additional therapy option for those with sickle cell disease who currently have few effective therapies available.

BPX‐501 is a medicinal product made of T‐cells, a type of white blood cell, extracted from a donor who is partly matched to a patient undergoing HSCT. These T‐cells are expected to help the patient to fight off viral infections while their immune system is being restored with the transplanted stem cells. However, the transplanted T‐cells can cause GvHD. BPX‐501 incorporates a safety mechanism where the T‐cells are genetically modified to include a ‘suicide gene’. If the patient develops GvHD, a medicine called rimiducid is given to switch on the suicide gene in the T‐cells. This causes the T cells to die, thus preventing worsening of the GvHD. BPX‐501 has the potential to reduce hospital admissions, infections and improve survival when used in combination with standard care.