Amsterdam, The Netherlands (ots/PRNewswire)
Molecular Therapeutics (Euronext: AMT), a leader in the field of
human gene therapy, today announced that it obtained a license from
La Sapienza University in Rome, Italy, to their advanced small
nuclear RNA (snRNA)-based exon-skipping technology for the treatment
of Duchenne muscular dystrophy (DMD). The combination with AMT's
proprietary adeno-associated virus (AAV) gene therapy platform
potentially makes up a long-term treatment for this seriously
debilitating disease with a single administration of the product.
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy, caused by mutations in the
dystrophin gene (essential for muscle function) is a severely
debilitating neuromuscular disease. It affects young children, is
progressive and leads to death in young adulthood. In the USA and
Europe about 120,000 people suffer from it. Today, there is no
treatment to prevent the fatal outcome.
The group of Prof. Irene Bozzoni at La Sapienza has done seminal
work to develop a treatment for DMD. Using adeno-associated viral
vectors, AMT's gene therapy platform of choice, and an exon-skipping
snRNA program Bozzoni and co-workers demonstrated long term systemic
and therapeutic effect in animals after a single administration. One
of the important advantages of the Bozzoni technology is that a
single systemic delivery of the AAV vector containing the required
antisense RNA-coding construct will result in long-term treatment of
Duchenne muscular dystrophy.
Formalizing relationship with leading RNA research group of
prof. Irene Bozzoni
Ronald Lorijn, CEO of AMT said: "We are very excited to have
inlicenced an exciting therapeutic approach, which has shown to
provide a life-long cure in rodents suffering from Duchenne's
disease. In Duchenne's disease in particular the combined application
of snRNA and AAV vectors has shown tremendous promise. Access to La
Sapienza's RNA technology perfectly complements our gene and vector
therapy platform. It adds a project to AMT's R&D pipeline that will
start its pre-clinical phase this year."
Exon skipping in DMD
Exon skipping is a technology to neutralize genetic defects by
preventing the faulty parts of the gene being used. At the cellular
level, a molecule called messenger RNA (mRNA) reads off (transcribes)
the protein instructions from the gene. It then transports these
instructions to the ribosomes in the cell where the protein is
assembled. Messenger RNA is not a transcript of the complete gene
sequence, but only of the exons, which are the sections of the gene
that code for a portion of the protein. If one of the exons contains
an error, this process may be halted, and the production of the
full-length dystrophin protein cannot take place. However, when the
faulty exon is eliminated (i.e. skipped), protein synthesis does take
place and leads to a functional, albeit shorter, dystrophin protein.
By using the exon-skipping technique the mutated exons in the
dystrophin mRNA that contain errors are "skipped" and as a result the
muscle cells are able to produce functional dystrophin protein.
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,
; Rob Janssen, Director Corporate
Communications & Investor Relations, +31-20-566-7509,