Amsterdam, The Netherlands (ots/PRNewswire) -
- Production Increase Brings Large Disease Areas Within Reach
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the
field of human gene therapy, today announced that it obtained a
Cooperative Research and Development Agreement from the National
Heart, Lung, and Blood Institute (NHLBI) of the National Institutes
of Health (NIH), Bethesda, Maryland, that promises to substantially
enhance the power of AMT's gene therapy platform.
The Cooperative Research and Development Agreement (CRADA) with
the NIH gives AMT the option to license the exclusive rights to the
recombinant adeno-associated virus (rAAV) baculoviral manufacturing
technology developed for treating Duchenne Muscular Dystrophy (DMD)
by Robert Kotin, Ph.D., and colleagues at the NHLBI Laboratory of
Biochemical Genetics. While AMT's current production platform is
based on a 50-liter bioreactor set-up, the application of the NIH
technology has been scaled-up to 250 liters, and allows for further
expansion to 1,000 liters. These production levels are unique in the
gene therapy field today and are relevant in the context of some of
the products that AMT has under development that affect large patient
populations, such as Factor IX for hemophilia B, IGF-I for liver
cirrhosis, and AMT-011 for NASH. The scale-up is also important for
treatment of Duchenne Muscular Dystrophy (DMD) for which AMT has just
closed an agreement with La Sapienza University in Rome, Italy, since
this indication requires a relatively high dose of gene therapy
vector in order to correct the defect in all affected muscles.
Ronald Lorijn, CEO of AMT said: "This CRADA with the NIH provides
AMT with a technology that in combination with our proprietary
in-house platform adds a whole new dimension to our manufacturing
platform. Our combined effort has the potential to greatly increase
the therapeutic reach of our gene therapies to benefit far larger
patient groups than was hitherto thought possible. In addition, it
would allow us to attempt the treatment of diseases that require the
systemic (as opposed to local) expression of therapeutic genes.
Duchenne Muscular Dystrophy is such a disease, and together with La
Sapienza in Rome we will work very hard to develop a treatment for
this wasting disorder for which there is currently no treatment."
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that to date 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 that are
caused by one faulty gene. AMT currently has a product pipeline with
six products at different stages of 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.
ots Originaltext: Amsterdam Molecular Therapeutics B.V
Im Internet recherchierbar: http://www.presseportal.ch
For information: André Verwei, CFO, +31-20-566-5686,
email@example.com; Rob Janssen, Director Corporate
Communications & Investor Relations, +31-20-566-7509,