Advanced therapy medicinal product (ATMP)
Development and Regulatory status
Phase III Clinical Trials
Aug 19 · Pivotal PIII trial (NCT03587116) is still recruiting with an estimated primary completion date of October 2021 
Single infusion of a gene therapy that uses an adeno-associated viral vector to deliver a codon-optimised, high-activity gene for human Factor IX to liver cells
Haemophilia B has a prevalence of around 1 in 30,000 live births (about five times rarer than haemophilia A). There are usually carrier females and affected males. The reference level of factor IX is 5 μg/mL but the normal range is from half to twice that level. Severe disease occurs with a factor IX level below 1% of the reference and accounts for about 50% of cases. Moderate severity occurs with a level of 1-5% and accounts for around 30% of cases .