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