Amsterdam Molecular Therapeutics Receives US Orphan Designation for Hemophilia B Gene Therapy

Amsterdam Molecular Therapeutics Receives US Orphan Designation for Hemophilia
                                B Gene Therapy

  PR Newswire

  AMSTERDAM, January 4, 2012

AMSTERDAM, January 4, 2012 /PRNewswire/ --

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of
human gene therapy, announced today that the U.S. Food and Drug Administration
(FDA) has granted orphan drug designationto its gene therapy program for the
treatment of hemophilia B. Orphan designation in the U.S. could provide up to
seven years market exclusivity on regulatory approval. Orphan designation for
AMT's hemophilia program in the European Union was granted in November 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 (Memphis, USA)
and University College London (UK). Promising data from an initial 6 patients,
recently published in the New England Journal of Medicine (N Engl J Med 2011;
365:2357-2365), 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.

"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." 

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development ofhuman 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. 


Contact: For further enquiries: Jörn Aldag, CEO, AMT, Tel : +31-20-566-7394,; Mike Sinclair, Partner, Halsin Partners, Tel :
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