AMT Receives Innovation Credit From Dutch Government for Duchenne Muscular Dystrophy

By Amsterdam Molecular Therapeutics B.v, PRNE
Tuesday, January 5, 2010

AMSTERDAM, January 6 - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in
the field of human gene therapy, announced today that it will receive an
Innovation Credit of up to EUR 4 million from the Dutch government to support
the development of AMT's gene therapy treatment for Duchenne Muscular
Dystrophy (DMD). The credit is granted by SenterNovem, an agency of the Dutch
Ministry of Economic affairs.

"We are delighted to receive this credit for DMD, as these
awards are only made to innovative projects with strong commercial
prospects," said Jorn Aldag, Chief Executive Officer of AMT. "This credit
will allow us to prioritize the development of our gene therapy for this
progressive and devastating disease."

Recently, AMT has reported it has successfully treated DMD in
a preclinical model of the disease with its proprietary gene therapy product
AMT-080. These proof of concept studies demonstrated that AMT's technology
resulted in functional dystrophin synthesis in both the heart and skeletal
muscles, which prevents muscular dystrophy . These data reinforce a previous
study in which this gene therapy approach was shown to successfully restore
dystrophin in diseased human muscle cells obtained from biopsies of DMD
patients. Together, these results establish a robust basis for AMT's
therapeutic approach to DMD.

On October 8, 2009 the European Medicines Evaluation Agency
(EMEA) granted Orphan Drug Designation to AMT's gene therapy product AMT-080
for the treatment of DMD. This entitles AMT to ten years of market
exclusivity to treat DMD in Europe following marketing approval for AMT-080,
provided that this product candidate is the first such approved new drug with
a major medical benefit.

The credit, together with accrued interest, will become
repayable in mid-2013, subject to the commercial success of the project. The
credit is payable in tranches linked to the achievement of specific
milestones, and will fund 35% of the total anticipated costs of the project
during this period.

About Duchenne Muscular Dystrophy

DMD is a severe disease characterized by progressive muscle
degeneration. It affects young children, almost exclusively boys, and leads
to progressive paralysis and death in young adulthood. The disease is caused
by mutations in the dystrophin gene, as a result of which the production of
functional dystrophin protein, an important structural component within
muscle tissue, is blocked. Currently, there is no treatment to prevent the
fatal outcome of this disease. DMD affects one in 3,500 males, making it one
of the most prevalent of muscular dystrophies.

AMT is developing a gene therapy product for DMD based on
'exon skipping' technology which results in bypassing the genetic defect such
that the functional protein can be formed again. Positive long-term
therapeutic effects of this approach have been demonstrated in animals.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in
the development of human gene based therapies. Using adeno-associated viral
(AAV) vectors as the delivery vehicle of choice for therapeutic genes, the
company has been able to design and validate what is probably the first
stable and scalable AAV production platform. This safe and efficacious
proprietary platform offers a unique manufacturing capability which can be
applied to a large number of rare (orphan) diseases that are caused by one
faulty gene. Currently, AMT has a product pipeline with several AAV-based
gene therapy products in LPL Deficiency, Hemophilia B, DMD, Acute
Intermittent Porphyria and Parkinson's Disease at different stages of
research or development.

About SenterNovem and the Innovation Credit program (

The Innovation Credit is a risk-carrying credit aimed at funding
development projects that have a strong commercial potential but also a high
technical risk. The projects have to be focused at the development of new
products, processes or services. The purpose of the Innovation Credit is to
reduce the financial risk for entrepreneurs. The Innovation Credit does not
need to be repaid if the project fails. The Innovation Credit has separate
budgets for technical development projects and for clinical development

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 Amsterdam Molecular Therapeutics 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, including, but not limited to, the timely commencement and success
of AMT's clinical trials and research endeavors, delays in receiving U.S.
Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health
Canada), market acceptance of AMT's products, effectiveness of AMT's
marketing and sales efforts, development of competing therapies and/or
technologies, the terms of any future strategic alliances, the need for
additional capital, the inability to obtain, or meet, conditions imposed for
required governmental and regulatory approvals and consents. AMT expressly
disclaims any intent or obligation to update these forward-looking statements
except as required by law. For a more detailed description of the risk
factors and uncertainties affecting AMT, refer to the prospectus of AMT's
initial public offering on June 20, 2007, and AMT's public announcements made
from time to time.

For information: Jorn Aldag, Chief Executive Officer, Tel +31(0)20-566-7394, j.aldag at

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