(Approved in EU)

Disease:  Beta-Thalassemia

Indication:  Zynteglo is indicated for the treatment of patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

Company:  Bluebird Bio


  • IPO $116.1M Jun 2016
  • Market Cap $3.713B Oct 2020

Notable Transactions:  Priced @ €1.58m / patient

Methods:  Hematopoietic Stem Cells (HSCs) are collected from the patient, then treated with a lentiviral vector to introduce functional genes for beta-globulin. HSCs are then re-introduced to the patient.

Link to the clinical trial:  NCT01745120

Link to company R&D pipeline:  https://www.bluebirdbio.com/our-science/pipeline

Notes: Bluebird previously said it planned to file its FDA application for Zynteglo by year-end 2019, but it has yet to send the therapy to U.S. regulators.

Current Phase:  FDA Approved

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