December 30, 2011
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 (
“U.S. orphan designation provides additional support for our hemophilia B gene therapy program and supplements the designation in the EU received in November,” said Jörn Aldag, CEO of AMT. “The early clinical success seen with the program to date by our partners is very encouraging. We will build on this success in the coming months.”
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