AMT-061

Unassigned

New Medicines

Severe and moderately severe haemophilia B

Information

Advanced therapy medicinal product (ATMP)
uniQure
uniQure

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials

Category

Gene therapy using AAV5 gene vector
Haemophilia B has a prevalence of around 1 in 30,000 live births (around 20% incidence of haemophilia A) and affected males have a deficiency of Factor IX clotting factor. Severe disease occurs with a factor IX level below 1% of the reference and accounts for about 50% of cases [2].
Severe and moderately severe haemophilia B
Intravenous infusion