AVI BioPharma and Collaborators Demonstrate In Vivo Effectiveness of PPMO-Based Splice Switching Oligomers (SSOs) in Genetic Dis
January 12 2009 - 5:10PM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs,
announced today new pre-clinical results published in Proceedings
of the National Academy of Sciences (Saovaros Svasti et al (January
12, 2009) Proc. Natl Acad. Sci. USA, 10.1073/pnas 0805434105)
demonstrating the effectiveness of a systemically delivered
PPMO-based splice switching oligomer or SSO (a morpholino oligomer
conjugated to an arginine-rich peptide) in vivo in a mouse model of
an inherited blood disorder. The results show that PPMO-based SSOs
may be effective in vivo not only in muscle cells for DMD
treatment, but also for another genetic disease and in target cells
more challenging than muscle fibers.
The paper's co-authors include Drs. Ryszard Kole and Hong
Moulton from AVI as well as Nobel laureate Dr. Oliver Smithies of
the University of North Carolina. The research was conducted by
scientists at the Departments of Pharmacology, Pathology and
Lineberger Comprehensive Cancer Center, University of North
Carolina and the Thalassemia Research Center, Mahidol University,
Bangkok, Thailand in collaboration with AVI scientists.
"The significance of these findings includes confirmation that
our PPMO-based SSOs are effective in additional tissues and cells,
establishing the potential that this technology may be selectively
applied to commercially viable genetic disease targets in the
future," said Ryszard Kole, Ph.D., SVP of Discovery Research at AVI
BioPharma. "The results from this research were particularly
impressive since immature red blood cells are challenging targets
and other oligonucleotides that were tested were ineffective."
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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