The Beta Thalassaemia drugs in development market research report provides comprehensive information on the therapeutics under development for Beta Thalassaemia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Beta Thalassaemia. Buy the report here.

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Data Insights Beta Thalassaemia - Drugs In Development, 2023

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The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Beta Thalassaemia and features dormant and discontinued products.

GlobalData tracks 58 drugs in development for Beta Thalassaemia by 56 companies/universities/institutes. The top development phase for Beta Thalassaemia is preclinical with 20 drugs in that stage. The Beta Thalassaemia pipeline has 52 drugs in development by companies and six by universities/ institutes. Some of the companies in the Beta Thalassaemia pipeline products market are: Vertex Pharmaceuticals, Novo Nordisk and BRL Medicine.

The key targets in the Beta Thalassaemia pipeline products market include Hemoglobin Subunit Beta (Beta Globin or Hemoglobin Beta Chain or HBB), Hemoglobin Subunit Gamma 1 (Gamma Globin or Gamma1 Globin or Hb F Agamma or Hemoglobin Gamma 1 Chain or Hemoglobin Gamma A Chain or HBG1), and B Cell Lymphoma/Leukemia 11A (B Cell CLL/Lymphoma 11A or COUP TF Interacting Protein 1 or Ecotropic Viral Integration Site 9 Protein Homolog or Zinc Finger Protein 856 or BCL11A).

The key mechanisms of action in the Beta Thalassaemia pipeline product include Hemoglobin Subunit Beta (Beta Globin or Hemoglobin Beta Chain or HBB) Activator with nine drugs in Phase II. The Beta Thalassaemia pipeline products include six routes of administration with the top ROA being Intravenous and ten key molecule types in the Beta Thalassaemia pipeline products market including Gene-Modified Cell Therapy, and Small Molecule.

Beta Thalassaemia overview

Beta-thalassemia is a genetic blood disorder characterized by reduced or absent production of beta-globin chains, a component of hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. This deficiency leads to anemia and various complications. Beta-thalassemia is classified based on the severity of symptoms: In beta-thalassemia major (Cooley’s anemia), individuals have little or no beta-globin production, leading to severe anemia, pale skin, fatigue, bone deformities, enlarged spleen, jaundice, and growth problems. Beta-thalassemia intermedia is less severe than major but can still cause moderate to severe anemia and various complications. Beta-thalassemia is an autosomal recessive genetic disorder, meaning an affected individual must inherit two abnormal beta-globin genes (one from each parent). Carriers of one abnormal gene (thalassemia trait) generally do not have symptoms but can pass the defective gene to their children.

For a complete picture of Beta Thalassaemia’s pipeline drug market, buy the report here.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.