Credit: Image by Arek Socha from Pixabay 

Author- Simpal K. Suman(Biotech communicator)

First gene therapy procedure for β-thalassemia has been approved in Europe . ZYNTEGLO™ is the first gene product for transfusion-dependent β-thalassemia (TDT) . It has developed by the Bluebird Bio, a Biotech company, based on gene therapy . Bluebird Bio accepts potential challenges to combat severe genetic diseases and cancer. Bluebird bio has received conditional marketing authorization from the European Commission(EC) for ZYNTEGLO™ in June 2019. This gene therapy is for the treatment of patients 12 years and older with transfusion-dependent beta-thalassemia(TDT) who have no other curative medical options .

Key points: TDT

  • A potentially severe, genetic blood disease

  • Reduced or no production of β-globin

  • β-globin, a critical component of hemoglobin protein

  • Hemoglobin: Oxygen-carrying protein, red in colour(Fe,Iron present)

  • Mutations in the β-globin (HBB) gene causes β-thalassemia

Key points: Gene therapy

  • An experimental technique

  • to treat or prevent disease

  • Replace faulty or defective gene by correct copy of the gene

  • Gene editing technique includes insertion, knock out or silence of the the gene.

 Key points: ZYNTEGLO™ 

β-globin gene (autologous CD34+ cells encoding βA-T87Q-globin gene)

A modified gene to be inserted into β-thalassemia patient.

Globally, β-thalassemia is genetically inherited problem . Till now this scientific world greatly affected in regions like Mediterranean countries, North Africa, the Middle East, India, Central Asia, and Southeast Asia. In the general population, it is estimated to affect approximately 1 in 100,000 individuals globally.


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