The applications are for the company's investigational gene therapy candidate for adults with hemophilia A.
The data presented at ISTH reflected marked improvements in FVIII half-life amongst study participants.
uniQure's gene therapy candidate AMT-061 is being developed for individuals with severe and moderately severe hemophilia B.
The final data was presented for the first time at a symposium at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH).
Much of the data was gleaned from the HAVEN clinical trial program.
The findings presented at ISTH were based on an ongoing phase 1/2 clinical trial in patients with severe hemophilia A.
BioMarin will be sharing multi-year clinical trial data on valoctocogene roxaparvovec at the upcoming congress of the International Society on Thrombosis and Haemostasis Congress in Melbourne, Australia.
The two companies are looking to combine their proprietary technologies to develop gene therapy candidates for both hemophilia A and B.
The partnership with Bayer and WFH will include a donation of 50 million international units worth of rFVIII therapies to help treat hemophilia A patients in the developing world.
Sigilon and MIT researchers are developing a novel cell therapy designed to control bleeding and to forestall undesired immune responses.
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