AVI BioPharma to Co-Host Exon Skipping Conference for Duchenne Muscular Dystrophy
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.
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SOURCE: AVI BioPharma, Inc.