The first two hemophilia patients treated with AMT-060, about 12 and 20 weeks after treatment, are now producing 4.5 percent and 5.5 percent, respectively, of normal Factor IX. To put that in context, these patients have severe or moderately severe hemophilia, meaning they typically produce less than 1 to 2 percent of these levels, and rely on frequent infusions to get those numbers up.
Shares of UniQure jumped about 12 percent in pre-market trading on Thursday.
Bumping those Factor IX figures up by a few percentage points might not seem like a big deal, but getting a patient to produce even 5 to 10 percent of their normal levels of clotting factor can turn a severe case of hemophilia into a mild one, or significantly lower the risk of a spontaneous bleed.
SOURCE - Xconomy