AVI BioPharma Publishes Preclinical Data in Muscular Dystrophy Research
June 12 2008 - 9:30AM
Marketwired
CORVALLIS, OR today announced the publication of preclinical
results of a study designed to demonstrate the ability of AVI's
NeuGene� class of drugs to induce sustained expression of
dystrophin in the mdx mouse model of Duchenne muscular dystrophy
(DMD). Treatment with the AVI compound resulted in production of
functional dystrophin in numerous appropriate tissues, including
the heart, diaphragm and skeletal muscles; key organs for the
treatment of the disease. The findings were published in the
peer-reviewed journal, Molecular Therapy.
http://www.nature.com/mt/journal/vaop/ncurrent/abs/mt2008120a.html
The paper, titled "Sustained Dystrophin Expression Induced by
Peptide-Conjugated Morpholino Oligomers in the Muscles of mdx
Mice," was a collaborative study by scientists from AVI, the
University of North Carolina at Chapel Hill and Mahidol University,
Thailand.
In this study, a PMO-peptide conjugate, termed PPMO-B, that
showed potent activity in skeletal muscles, diaphragm and,
importantly, the heart was initially selected from a panel of other
conjugates and subsequently applied for treatment of DMD in the mdx
mouse model. The first finding of this investigation was that the
systemically delivered PPMO-B induced high level of exon skipping
in dystrophin mRNA in body-wide muscles, including diaphragm and
cardiac muscle of treated mice, without detectable toxicity. The
induced dystrophin mRNA persisted for several weeks, indicating
that the drug is retained and functional in the target organs for
an extended period of time. Furthermore, the mRNA effectively
translated substantial amounts of dystrophin protein in all target
muscles. Dystrophin protein persisted in the muscles even longer
than the RNA. Additional tests indicated improvement in muscle
integrity and, specifically, reduction of inflammation of the
heart. This is the first report of PPMO-mediated exon skipping and
functional dystrophin protein induction in the heart of treated
animals.
"For DMD patients, restoration of functional dystrophin in heart
and diaphragm is critical because respiratory and cardiovascular
complications are the ultimate cause of mortality," said Dr.
Ryszard Kole, co-author and AVI's Senior Vice President of
Discovery Research. "This is the first study to establish
peptide-conjugated NeuGene drugs as potential treatment for
DMD."
About Alternative RNA Splicing Technology
In normal genetic function, gene transcription produces a
full-length pre-messenger RNA (pre-mRNA) that is then processed to
a much shorter and functional messenger RNA (mRNA). The mRNA is the
template for creating a protein. During pre-mRNA processing,
packets of useful genetic information, called exons, are spliced
together to make the functional mRNA template, while unnecessary
fragments called introns are snipped out of the full-length RNA. In
some diseases, such as DMD, mutations derail this process and
prevent production of functional dystrophin protein. AVI's
proprietary third-generation NeuGene chemistry can be used to
target splicing sites in the pre-mRNA, thus forcing the cell
machinery to skip over targeted exons, providing altered mRNA,
which in turn produces altered proteins. Skipping a defective exon
can restore proper RNA reading frame and restore production of the
protein to overcome the devastating clinical consequences of the
mutation.
About Duchenne Muscular Dystrophy
DMD is the most common fatal genetic disorder to affect children
around the world. It is a devastating and incurable muscle-wasting
disease associated with specific inborn errors in the gene that
expresses dystrophin, a protein that is an essential component for
striated muscle function. When dystrophin is missing or
nonfunctional due to a mutation in the genetic code of the
dystrophin gene, as it is in DMD, the result is membrane leakage
and fiber damage, ultimately leading to degeneration and death of
the muscle fiber. Approximately one in 3,500 boys is born with DMD,
and an estimated 15,000 to 20,000 children are afflicted in the
United States alone.
About AVI BioPharma
AVI BioPharma develops therapeutic products for the treatment of
life-threatening diseases using third-generation NeuGene� antisense
drugs and alternative RNA splicing technology. AVI's alternative
RNA splicing technology is initially being applied to potential
treatments for Duchenne muscular dystrophy. AVI's NeuGene compounds
are also designed to treat post-operative cardiovascular
restenosis. In addition to targeting specific genes in the body,
AVI's antiviral program uses NeuGene antisense compounds to combat
disease by targeting single-stranded RNA viruses, including Marburg
Musoke and Ebola Zaire viruses. More information about AVI is
available at www.avibio.com.
"Safe Harbor" Statement under the Private Securities Litigation
Reform Act of 1995: The statements that are not historical facts
contained in this release are forward-looking statements that
involve risks and uncertainties, including, but not limited to, the
results of research and development efforts, the results of
preclinical and clinical testing, the effect of regulation by the
FDA and other agencies, the impact of competitive products, product
development, commercialization and technological difficulties, and
other risks detailed in the company's Securities and Exchange
Commission filings.
AVI Press and Investor Contact: Michael Hubbard Director of
Corporate Communications Email Contact (503) 227-0554
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