Gene therapy's roller coaster ride (Pink Sheet - Informa) - May 11, 2015 - "Baxter AG is expected to file a BLA in 2017-2018 for its hemophilia B candidate AskBio009 (scAAV2/8-LP1-hFIXco), a recombinant adeno-associated virus (rAAV) based gene therapy, which corrects a defect in the gene encoding coagulation Factor IX."
Anticipated BLA • Hemophilia
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