Genetics

uniQure N.V. (NASDAQ:QURE) announced that its proprietary, investigational gene therapy in patients with severe hemophilia B,AMT-060, received Breakthrough Therapy designation by the USFDA. As quoted in the press release: This designation is based on results from the ongoing, dose-ranging Phase 1-2 study that show sustained increases in Factor IX (FIX), reductions in FIX replacement usage and a …

uniQure N.V. (NASDAQ:QURE) announced that its proprietary, investigational gene therapy in patients with severe hemophilia B,AMT-060, received Breakthrough Therapy designation by the USFDA.
As quoted in the press release:

This designation is based on results from the ongoing, dose-ranging Phase 1-2 study that show sustained increases in Factor IX (FIX), reductions in FIX replacement usage and a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up.
“We are very pleased the FDA has designated AMT-060 a Breakthrough Therapy for patients with hemophilia B,” stated Matthew Kapusta, chief executive officer of uniQure. “The FDA’s decision to prioritize and expedite the review of AMT-060 is an important milestone for uniQure and we are committed to working closely with the FDA to rapidly advance our hemophilia B program into late-stage development.”

Click here for the full press release.

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