AVI BioPharma Strengthens Patent Position in Exon Skipping
November 24 2008 - 8:05AM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs,
today announced the signing of an exclusive worldwide license
agreement with the University of Western Australia (UWA) to a
patent application related to the treatment of Duchenne Muscular
Dystrophy (DMD). The patent application, "Antisense
Oligonucleotides for Inducing Exon Skipping and Methods of Use
Thereof" (U.S. Patent publication number US2008/0200409 A1 and
foreign counterparts) claims compositions and methods for treating
DMD in humans by skipping exons in the dystrophin gene using
antisense oligomers. Among the inventors on the licensed patent
application is Stephen D. Wilton, Ph.D., Head of the Molecular
Genetic Therapies Group at UWA, a renowned pioneer in the use of
exon skipping to treat DMD.
"Dr. Wilton is a longtime collaborator of AVI, and our securing
of this license to UWA's patent application further strengthens
AVI's leading position in the field of exon skipping for DMD," said
Leslie Hudson, Ph.D., President and Chief Executive Officer of AVI
BioPharma.
In addition to the UWA patent application, AVI's patent position
in exon skipping includes exclusive rights to Dr. Ryszard Kole's
general RNA splice altering patents gained though AVI's acquisition
of Ercole Biotech earlier this year, as well as other AVI-filed
patents and in-licensed intellectual property specific to exon
skipping of the dystrophin gene as a therapeutic target.
AVI is currently evaluating the exon skipping therapeutic
AVI-4658 for the treatment of DMD. Preclinical studies have
demonstrated sustained production of functional dystrophin in
numerous tissues, including the heart, diaphragm and skeletal
muscles. A clinical trial is currently underway at the Imperial
College of London where patients with DMD are receiving a
single-dose, intramuscular administration of AVI-4658. The Company
was granted orphan drug designation for AVI-4658 by the U.S. Food
and Drug Administration in November of 2007 and has been
recommended for orphan product designation by the European
Medicines Agency (EMEA) Committee for Orphan Medicinal
Products.
Dr. Wilton and AVI researchers have collaborated on research in
exon skipping and have published articles detailing their research.
The most recently published research includes an article appearing
in the December issue of the Journal of Gene Medicine titled
"By-passing the Nonsense Mutation in the 4(CV) Mouse Model of
Muscular Dystrophy by Induced Exon Skipping." A link to the preview
of this publication, posted online in advance of print publication,
can be found under the "Publications" section of the AVI BioPharma
website at www.avibio.com
About Duchenne Muscular Dystrophy (DMD)
DMD is one of the most common fatal genetic disorders 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 a range of promising therapies in or moving into
development.
About AVI BioPharma
AVI BioPharma is focused on the discovery and development of
RNA-based drugs utilizing proprietary derivatives of its antisense
chemistry (morpholino-modified phosphorodiamidate oligomers or
PMOs) that can be applied to a wide range of diseases and genetic
disorders through several distinct mechanisms of action. Unlike
other RNA therapeutic approaches, AVI's antisense technology has
been used to directly target both messenger RNA (mRNA) and its
precursor (pre-mRNA), allowing for both up- and down-regulation of
targeted genes and proteins. AVI's RNA-based drug programs are
being evaluated for the treatment of Duchenne muscular dystrophy as
well as for the treatment of cardiovascular restenosis through our
partner Global Therapeutics, a Cook Group Company. AVI's antiviral
programs have demonstrated promising outcomes in Ebola Zaire and
Marburg Musoke virus infections and may prove applicable to other
viral targets such as HCV or Dengue viruses. For more information,
visit 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: Julie Rathbun Investor Relations
(541) 224-2575 Investorrelations@avibio.com
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