Mount Sinai Will be Site for Gene Therapy Study for Hemophilia B

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A promising new gene therapy study for hemophilia B is getting started and Mount Sinai will be one of two initial study sites.  Asklepios Biopharmaceutical (AskBio) in collaboration with several investigators has developed a gene therapy treatment using adeno-associated virus, type 8 (AAV8) to deliver factor IX genes to the liver.  AskBios is using a method similar method to that used in the St. Jude/University College London study we reported on at the end of 2011.  One difference is that AskBio has a more highly purified AAV8 vector.  That means that there are more active vector particles in a given dose, so a dose can potentially transform a larger number of cells to produce factor IX, hopefully with fewer side effects.  AskBio is also using a modified factor IX gene to produce a protein that has a significantly higher clotting activity than normal factor IX.  AskBio believes they can eventually increase patient’s blood levels of factor IX to 40 – 50% of normal.  That’s enough to essentially provide a cure for hemophilia.


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