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
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.
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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