UniQure Hemophilia B Gene Therapy AMT-061 Has Early Phase IIb Success
Early positive data from a Phase IIb dose-confirmation study showed AMT-061 achieved and sustained therapeutic levels of Factor IX (FIX) activity in all three participating patients at six weeks after a single administration of the investigational AAV5-based gene therapy.
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Gene therapy’s promise depends on the ecosystem now being built to deliver real product to patients – safely and at quantities that match the soaring expectations fed by the curative potential of the science. To assess the state of play, In Vivo talks to the man who leads the team responsible for bringing forward the first US-approved gene therapy to treat an incurable, inherited genetic condition: John Furey, chief operating officer of Spark Therapeutics.
UniQure’s gene therapy, AMT-060, has been accepted into the European Medicines Agency’s priority medicines scheme, PRIME, a move likely to intensify the race to market of a small group of gene therapies for hemophilia B, all currently in Phase I/II studies.
Sanofi to sell most of its Regeneron stake, with much of it being re-purchased by Regeneron, but both stressed the move would not hurt their long established and very profitable drug-development collaboration.