AVI BioPharma to Co-Host Exon Skipping Conference for Duchenne Muscular Dystrophy
October 14 2008 - 12:00PM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs,
today announced that the Company -- along with The Foundation to
Eradicate Duchenne, the CureDuchenne Foundation, and Prosensa --
will co-host an exon skipping conference for Duchenne muscular
dystrophy (DMD) from October 14-17, 2008 at the Banbury Center of
Cold Spring Harbor Laboratory, Cold Spring Harbor, NY. Invited
participants will be drawn from all over the world and from all
areas of research, clinical development, regulatory affairs and key
DMD disease foundations to review the advances in oligonucleotides
as therapeutic agents for DMD.
AVI's contribution to this international meeting will include
presentations from Drs. Peter O'Hanley and Ryszard Kole -- who are
respectively SVP of Clinical Development and Discovery Research at
the Company -- on AVI's clinical trials using a PMO candidate drug
(AVI-4658) in DMD as well as the recently published work on
improved PPMO compounds currently undergoing preclinical
development. Several of the Company's key collaborators -- who are
internationally-recognized experts in their own right -- will
present updates on their work in collaboration with AVI. These
include Professor Francesco Muntoni, UCL Institute of Child Health,
London, Professor Steve D. Wilton, University of Western Australia,
Perth and Professor Qi L. Lu, Carolinas Medical Center, Charlotte,
NC.
In recent years, there have been significant advances in
oligonucleotide research that have led to exon skipping drug
candidates that retain sequence-specific splice-skipping activity,
while showing little or no protein binding or associated off-target
effects. In addition, recent developments in chemistry have
improved intracellular delivery and other features important for
drug-like character, suggesting that the Company and others may
have overcome many of the hurdles which were significant barriers
to the clinical development of RNA-based therapeutics.
About Duchenne Muscular Dystrophy (DMD)
DMD is the most common fatal genetic disorder to affect children
around the world. Approximately one in every 3,500 boys worldwide
is afflicted with Duchenne muscular dystrophy with 20,000 new cases
reported each year. It is a devastating and incurable
muscle-wasting disease associated with specific inborn errors in
the gene that codes for dystrophin, a protein that plays a key
structural role in muscle fiber function. Symptoms usually appear
in male children before age 6. Progressive muscle weakness of the
legs and pelvis eventually spreads to the arms, neck, and other
areas. By age 10, braces may be required for walking, and most
patients are confined to a wheelchair by age 12. Eventually, this
progresses to complete paralysis and increasing difficulty in
breathing. The condition is terminal and death usually occurs
before the age of 30. The outpatient cost of care for a
non-ambulatory DMD boy is among the highest of any disease. There
is currently no cure for DMD, but for the first time in decades,
there are promising therapies moving into development.
About AVI BioPharma
AVI BioPharma is focused on the discovery and development of
RNA-based drugs using the company's expanded portfolio of
proprietary antisense compounds (PMOs). The company's technology
applications leverage distinct mechanisms of action in a range of
genetic diseases, genetic disorders and the genetic code of
disease-causing organisms. The emerging field of directed
alternative RNA splicing represents AVI's newest and most exciting
application based on the company's core antisense technology.
Functional attributes of this approach may include correcting
genetic defects (RNA mutations; which AVI believes could produce
promising treatments for Duchenne muscular dystrophy), coding for
novel soluble receptors (an exciting and novel approach which could
have application in the treatment of inflammatory diseases such as
rheumatoid arthritis), and the reduction in activity of immune
modulators in disease states (currently being applied to IL-10).
AVI's RNA-based drug programs also include blocking mRNA
translation. In AVI's biodefense program, this application has been
successful against the single-stranded RNA viruses Ebola Zaire and
Marburg Musoke in non-human primates and may have value against
other viral targets such as HCV, Dengue, Junin, influenza and RSV
viruses. This application also will be evaluated in the clinic for
the treatment of cardiovascular restenosis by our partner Cook
Medical. 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 (503) 227-0554 Email Contact
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