Beta-Thalassemia Disease Pipeline Drugs Assessment Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2019 - 2030

Beta-Thalassemia is an inherited blood disorder characterized by decreased levels of functional hemoglobin, which is the iron-containing protein present in red blood cells that carries oxygen to the cells throughout the body. People with Beta-Thalassemia will be suffering from lack of oxygen supply in body and also shortage of red blood cells and will be at an increased risk of developing abnormal blood clots.

Symptoms of Beta-Thalassemia include pale skin, jaundice, enlarged spleen, liver and heart, and delayed puberty. Beta-Thalassemia can be diagnosed by molecular and genetic tests, blood tests, etc. Treatment of Beta-Thalassemia includes frequent blood transfusions, bone marrow transplant, surgery to remove spleen or gallbladder and medications.

Key Developments:

• In October 2017, Sangamo Therapeutics and Bioverativreceived U.S.FDA IND acceptance to begin human clinical studies of ST-400, a gene-edited cell therapy candidate for people with transfusion-dependent beta-thalassemia.
• In September 2016, bluebird bio, Inc. commenced a phase III global multi center study of its HGB-207  in patients with transfusion-dependent beta-thalassemia with non-genotypes
• In May 2015, Luspatercept of Celgene Corporation and Acceleron Pharma Inc. was granted Fast track designations by U.S. FDA for two separate indications, for the treatment of patients with transfusion-dependent beta-thalassemia and patients with non-transfusion dependent beta-thalassemia. Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members of the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation