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Amsterdam Molecular Therapeutics Receives Orphan Designation in the European Union for Hemophilia B Gene Therapy

AMSTERDAM, November 16, 2011 /PRNewswire/ --

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMA) has granted orphan designation for its gene therapy program for the treatment of hemophilia B. Orphan designation in the European Union provides several benefits including 10 years of market exclusivity from product launch and access to the central authorization procedure.

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). Promising data from an initial 6 patients shows that gene therapy administration resulted in a reduced need for protein replacement treatment, the standard care for hemophilia patients.  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.

"Orphan designation is an important milestone for our hemophilia B gene therapy program and will provide additional support to our negotiations as we seek potential licensing partners," said Jörn Aldag, CEO of AMT. "A successful gene therapy for hemophilia could dramatically change not only the lives of patients but also the current hemophilia market that is dominated by protein replacement therapies."  

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies. AMT has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT to pursue its strategy of focusing on this sector of the industry.  AMT was founded in 1998 and is based in Amsterdam. Further information can be found at

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of AMT only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business. AMT expressly disclaims any intent or obligation to update any forward-looking statements herein except as required by law. 

SOURCE Amsterdam Molecular Therapeutics (AMT) B.V
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