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Spectacular results for bluebird’s gene therapy in thalassemia
April 20, 2018Patients with the rare blood disorders transfusion-dependent β-thalassemia (TDT) have shown spectacular results in clinical trials of bluebird bio’s gene therapy, raising hopes the treatment could be a cure for the condition. -
Spark Therapeutics acquires licence to supply viral gene therapy outside US
January 29, 2018Spark Therapeutics has signed a licensing and supply agreement with Novartis Pharmaceuticals to develop and commercialise investigational gene therapy voretigene neparvovec outside the US. -
FDA Approval Brings First Gene Therapy to the U.S.
September 01, 2017The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).
Pharma Sources Insight January 2025
