January 09, 2016

Gene therapy seems able to convert severe hemophilia into mild hemophilia

Dutch firm UniQure presented results from an early study of AMT-060, a gene therapy it’s developing for patients with hemophilia B. To be clear, these are numbers from just the first handful of patients in UniQure’s study, treated with a low dose of the gene therapy. And they’re early—the patient treated the longest is now 20 weeks post-treatment. For gene therapy to become a viable option for hemophilia, the effects will have to hold up for a long period of time, stop the dangerous, spontaneous bleeds that patients can suffer, and eliminate the need for frequent infusions of the recombinant factors that patients have to take to clot their blood.

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

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