#AMT’s #HemophiliaB #GeneTherapy gains #OrphanDrug Designation

AMT's  gene therapy program for the treatment of hemophilia B has today gained orphan drug designation in the US from the FDA. AMT’s hemophilia B program, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is being investigated in a Phase I/II study conducted by St. Jude’s Children’s Research Hospital (Memphis, USA) and University College …