We will continue to work as hard as we can to push this area forward and perhaps in ten years it will be available to everyone. BeneFIX was licensed in the U.S. last year and has been used successfully in treating patients. However, one of the issues surrounding the product is that it exhibits a lower recovery than plasma-derived Factor IX products. Recovery refers to the amount of Factor IX that is seen in the bloodstream after an injection. Genetics Institute, the manufacturer, has been doing extensive studies, both in the laboratory and with patients, to determine the cause of the lower recovery.
Translation is the process a cell uses to make a protein from the recipe given in the gene.
Post-translational modifications are changes that the cell makes to a protein after the basic molecule has been formed. These modifications are often one of the main stumbling blocks in trying to produce proteins by genetic engineering. Often the cells used to produce a recombinant protein in large amounts do not modify the protein exactly the same way that the body's cells do. This often does not affect the function of the protein, but sometimes it can. However, the usefulness of any drug is not determined by whether it is identical to another, but rather whether it does its job in a predictable manner.