The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.
uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated clinical data on the three patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. In addition, the company presented up to 3.5 years of follow-up data on the 10 patients in the Phase I/II trial of AMT-060, its first-generation gene therapy for the treatment of hemophilia B. These clinical data were presented on Saturday, July 6 in oral presentations at the 27th Congress of the International Society on Thrombosis and Haemostasis, taking place in Melbourne, Australia.
The Phase IIb study of AMT-061 is an open-label, single-dose, single-arm, multi-center trial being conducted in the United States. Three patients with severe hemophilia (endogenous Factor IX (FIX) activity less than or equal to one percent) were enrolled in the study and received a single intravenous infusion of 2×1013 vc/kg. Prior to the administration of AMT-061, all three patients showed low levels of pre-existing neutralizing antibodies to AAV5 but were not excluded from the trial on that basis. Patients in the Phase IIb study will be followed for 52 weeks to assess FIX activity, bleeding rates and usage of FIX replacement therapy, and will be monitored for five years to evaluate the safety of AMT-061.
Read the press release in its entirety here.聽