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

By Amsterdam Molecular Therapeutics B.v, PRNE
Sunday, March 6, 2011

AMSTERDAM, March 7, 2011 - 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 Netherlands, 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
www.amtbiopharma.com.

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.

PRN NLD

Jörn Aldag, CEO, AMT, Tel : +31-20-566-7394, j.aldag at amtbiopharma.com; Mike Sinclair, Partner, Halsin Partners, Tel : +44-20-7318-2955, msinclair at halsin.com

Health Care / Hospitals News

March 7 News

YOUR VIEW POINT
NAME : (REQUIRED)
MAIL : (REQUIRED)
will not be displayed
WEBSITE : (OPTIONAL)
YOUR
COMMENT :