AMT Successful in Preclinical Treatment of DMD

By Amsterdam Molecular Therapeutics B.v, PRNE
Tuesday, November 10, 2009

AMSTERDAM, November 11 - 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.


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

Biotechnology News

Amsterdam Molecular Therapeutics B.V News

will not be displayed