5 Questions for Gene Therapy in 2023 

Experts believe gene therapy will have a pivotal year ahead of it. It will bring not only new treatments but challenges.  

Just imagine a patient with the inherited blood disease beta-thalassemia will receive an infusion containing hundreds of millions of his own stem cells at the end of March this year. The drug manufacturing facility will equip these cells with a modified gene that will be able to correct the condition of this patient.  

It will be the first commercial use of gene therapy, and the procedure has already been approved by the Food and Drug Administration (FDA) in August.  

For more than 10 years, biotechnological company Bluebird bio conducted research, which resulted in Zynteglo. Over the past two years, gene therapy has faced safety concerns and clinical and regulatory setbacks. The approval of the procedure offered validation to Bluebird and the broader gene therapy field.     

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