AMT Receives Grant From Dutch Parents Organization for Duchenne Muscular Dystrophy Gene Therapy

March 6, 2011

AMSTERDAM, March 7, 2011 /PRNewswire-FirstCall/ — Amsterdam Molecular
Therapeutics (AMT) (Euronext: AMT), a leader in the field of human gene
therapy, announced today that the Duchenne Parent Project, based in the
, has awarded AMT a grant of EUR 145,000 to support the
development of AMT-080, AMT’s gene therapy for Duchenne Muscular Dystrophy
(DMD). DMD is a severe, fatal disease affecting young children, almost
exclusively boys, characterized by progressive muscle degeneration. It is
caused by mutations in the dystrophin gene, which blocks the production of
functional dystrophin protein, an important structural component within
muscle tissue. The Duchenne Parent Project was established by the parents of
children with DMD to provide information and vital support for families
affected by the disease.

AMT-080 is based on ‘exon skipping’ technology, which effectively
bypasses the genetic defect so that functional dystrophin protein can be
produced. AMT plans to begin a Phase I/II trial with AMT-080 by the end of
2012 following successful preclinical studies where AMT-080 has shown
efficacy in models of DMD. These proof of concept studies demonstrated that
AMT-080 produced functional dystrophin synthesis in both the heart and
skeletal muscles, leading to the prevention of muscular dystrophy in these
models. These data are strengthened by a study in which this gene therapy
approach was shown to successfully restore dystrophin activity in diseased
human muscle cells obtained from biopsies of DMD patients.

“Support such as this grant received from the Duchenne Parent Project, as
well as other similar patient-led organizations, is greatly appreciated by
the whole team at AMT. We are all committed to progressing this treatment
into clinical trials as soon as possible and ultimately making it available
to patients,” said Jorn Aldag, Chief Executive Officer of AMT. “This generous
grant and the support previously given by SenterNovem has ensured priority
development for this innovative potential treatment for DMD within AMT.”

In January 2010 Agentschap NL(formerly SenterNovem), an agency of the
Dutch Ministry of Economic Affairs, awarded AMT an Innovation Credit of up to
EUR 4 million, and in October 2009 and September 2010 AMT received Orphan
Drug designation from the European Medicines Agency and the US Food and Drug
Administration respectively.

About Duchenne Muscular Dystrophy (DMD)

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.

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies.
The company’s lead product Glybera(R), a gene therapy for the treatment of
lipoprotein lipase deficiency (LPLD), is currently under review by the
European Medicines Agency (EMA). If approved, Glybera will be the first gene
therapy product to be marketed in Europe. AMT also has a product pipeline of
several gene therapy products in development for hemophilia B, Duchenne
Muscular Dystrophy, acute intermittent porphyria, and Parkinson’s disease.
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 B.V

Source: newswire

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