AMT Successful in Preclinical Treatment of DMD

November 11, 2009

AMSTERDAM, November 11 /PRNewswire-FirstCall/ — Amsterdam Molecular
Therapeutics (Euronext: AMT), a leader in the field of human gene therapy,
announced today that it has successfully treated Duchenne muscular dystrophy
(DMD) in an animal model with its proprietary gene therapy. The proof of
concept studies were performed in collaboration with the group of Professor

Irene Bozzoni (University of Rome, La Sapienza, Italy) and demonstrated
effectiveness in the heart as well as in skeletal muscles. In a previous
study, AMT’s gene therapy approach was shown to be successful in the
treatment of diseased human muscle cells obtained from biopsies of DMD
patients. These data establish a robust basis for AMT’s therapeutic approach
to DMD.

“We are proud to establish proof of concept with our gene therapy for
Duchenne muscular dystrophy, as it is an important new step in developing a
treatment for this progressive and devastating disease,” said Jörn Aldag,
Chief Executive Officer of AMT. “In particular, our positive results in the
heart and respiratory muscles bring hope that we might be able to prevent the
fatal outcome of this disease in the future, potentially through a long-term
remedy with a single treatment.”

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe disease characterized by
progressive muscle degeneration. It affects young children, almost
exclusively boys, and leads to 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 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 has a unique gene therapy platform that appears to circumvent many if
not all of the obstacles that have prevented gene therapy from becoming a
mainstay of clinical medicine. 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. As such, AMT’s proprietary platform holds tremendous
promise for thousands of rare (orphan) diseases, especially diseases that are
caused by one faulty gene. Currently, AMT has a pipeline with nine products
at different stages of research or 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.

SOURCE Amsterdam Molecular Therapeutics B.V

Source: newswire

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