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

#AMT, #AmsterdamMolecularTherapeutics, #HemophiliaB, #gene therapy, #OrphanDrugAMT'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 London (UK).  AMT is preparing for additional clinical development work to establish safety, tolerability and proof-of-concept with a factor IX gene therapy produced using its proprietary AAV production system.


To find out more about the scientific research being carried out by AMT and the nuts and bolts of their recent failure to achieved approval for their gene therapy from Dr Harald Petry and Jörn Aldag, then you must attend the World Gene Therapy Congress taking place on the 22-23 May 2012 in London.


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