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News List

Spectacular results for bluebird’s gene therapy in thalassemia pharmaphorum
April 20, 2018
Patients 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 pharmaceutical-technology
January 29, 2018
Spark 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. americanpharmaceuticalreview.
September 01, 2017
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).