Press Release
Home > Press Release > Marketwire
AVI BioPharma to Co-Host Exon Skipping Conference for Duchenne Muscular Dystrophy
Therapeutic Strategies Using Oligonucleotide-Directed Splicing
CORVALLIS, OR -- (Marketwire) -- 10/14/08 -- AVI BioPharma, Inc. (NASDAQ: AVII), adeveloper of RNA-based drugs, today announced that the Company -- alongwith The Foundation to Eradicate Duchenne, the CureDuchenne Foundation, andProsensa -- will co-host an exon skipping conference for Duchenne musculardystrophy (DMD) from October 14-17, 2008 at the Banbury Center of ColdSpring Harbor Laboratory, Cold Spring Harbor, NY. Invited participantswill be drawn from all over the world and from all areas of research,clinical development, regulatory affairs and key DMD disease foundations toreview the advances in oligonucleotides as therapeutic agents for DMD.
AVI's contribution to this international meeting will include presentationsfrom Drs. Peter O'Hanley and Ryszard Kole -- who are respectively SVP ofClinical Development and Discovery Research at the Company -- on AVI'sclinical trials using a PMO candidate drug (AVI-4658) in DMD as well as therecently published work on improved PPMO compounds currently undergoingpreclinical development. Several of the Company's key collaborators -- whoare internationally-recognized experts in their own right -- will presentupdates on their work in collaboration with AVI. These include ProfessorFrancesco Muntoni, UCL Institute of Child Health, London, Professor SteveD. 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 oligonucleotideresearch that have led to exon skipping drug candidates that retainsequence-specific splice-skipping activity, while showing little or noprotein binding or associated off-target effects. In addition, recentdevelopments in chemistry have improved intracellular delivery and otherfeatures important for drug-like character, suggesting that the Company andothers may have overcome many of the hurdles which were significantbarriers 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 theworld. Approximately one in every 3,500 boys worldwide is afflicted withDuchenne muscular dystrophy with 20,000 new cases reported each year. Itis a devastating and incurable muscle-wasting disease associated withspecific inborn errors in the gene that codes for dystrophin, a proteinthat plays a key structural role in muscle fiber function. Symptomsusually appear in male children before age 6. Progressive muscle weaknessof the legs and pelvis eventually spreads to the arms, neck, and otherareas. By age 10, braces may be required for walking, and most patients areconfined to a wheelchair by age 12. Eventually, this progresses tocomplete paralysis and increasing difficulty in breathing. The condition isterminal and death usually occurs before the age of 30. The outpatient costof 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-baseddrugs using the company's expanded portfolio of proprietary antisensecompounds (PMOs). The company's technology applications leverage distinctmechanisms of action in a range of genetic diseases, genetic disorders andthe genetic code of disease-causing organisms. The emerging field ofdirected alternative RNA splicing represents AVI's newest and most excitingapplication based on the company's core antisense technology. Functionalattributes of this approach may include correcting genetic defects (RNAmutations; which AVI believes could produce promising treatments forDuchenne muscular dystrophy), coding for novel soluble receptors (anexciting and novel approach which could have application in the treatmentof inflammatory diseases such as rheumatoid arthritis), and the reductionin activity of immune modulators in disease states (currently being appliedto IL-10). AVI's RNA-based drug programs also include blocking mRNAtranslation. In AVI's biodefense program, this application has beensuccessful against the single-stranded RNA viruses Ebola Zaire and MarburgMusoke in non-human primates and may have value against other viral targetssuch as HCV, Dengue, Junin, influenza and RSV viruses. This applicationalso will be evaluated in the clinic for the treatment of cardiovascularrestenosis by our partner Cook Medical. More information about AVI isavailable at www.avibio.com.
"Safe Harbor" Statement under the Private Securities Litigation Reform Actof 1995: The statements that are not historical facts contained in thisrelease are forward-looking statements that involve risks anduncertainties, including, but not limited to, the results of research anddevelopment efforts, the results of preclinical and clinical testing, theeffect of regulation by the FDA and other agencies, the impact ofcompetitive products, product development, commercialization andtechnological difficulties, and other risks detailed in the company'sSecurities and Exchange Commission filings.
AVI Press and Investor Contact:Michael HubbardDirector of Corporate Communications(503) 227-0554Email Contact
In recent years, there have been significant advances in oligonucleotideresearch that have led to exon skipping drug candidates that retainsequence-specific splice-skipping activity, while showing little or noprotein binding or associated off-target effects. In addition, recentdevelopments in chemistry have improved intracellular delivery and otherfeatures important for drug-like character, suggesting that the Company andothers may have overcome many of the hurdles which were significantbarriers 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 theworld. Approximately one in every 3,500 boys worldwide is afflicted withDuchenne muscular dystrophy with 20,000 new cases reported each year. Itis a devastating and incurable muscle-wasting disease associated withspecific inborn errors in the gene that codes for dystrophin, a proteinthat plays a key structural role in muscle fiber function. Symptomsusually appear in male children before age 6. Progressive muscle weaknessof the legs and pelvis eventually spreads to the arms, neck, and otherareas. By age 10, braces may be required for walking, and most patients areconfined to a wheelchair by age 12. Eventually, this progresses tocomplete paralysis and increasing difficulty in breathing. The condition isterminal and death usually occurs before the age of 30. The outpatient costof 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-baseddrugs using the company's expanded portfolio of proprietary antisensecompounds (PMOs). The company's technology applications leverage distinctmechanisms of action in a range of genetic diseases, genetic disorders andthe genetic code of disease-causing organisms. The emerging field ofdirected alternative RNA splicing represents AVI's newest and most excitingapplication based on the company's core antisense technology. Functionalattributes of this approach may include correcting genetic defects (RNAmutations; which AVI believes could produce promising treatments forDuchenne muscular dystrophy), coding for novel soluble receptors (anexciting and novel approach which could have application in the treatmentof inflammatory diseases such as rheumatoid arthritis), and the reductionin activity of immune modulators in disease states (currently being appliedto IL-10). AVI's RNA-based drug programs also include blocking mRNAtranslation. In AVI's biodefense program, this application has beensuccessful against the single-stranded RNA viruses Ebola Zaire and MarburgMusoke in non-human primates and may have value against other viral targetssuch as HCV, Dengue, Junin, influenza and RSV viruses. This applicationalso will be evaluated in the clinic for the treatment of cardiovascularrestenosis by our partner Cook Medical. More information about AVI isavailable at www.avibio.com.
"Safe Harbor" Statement under the Private Securities Litigation Reform Actof 1995: The statements that are not historical facts contained in thisrelease are forward-looking statements that involve risks anduncertainties, including, but not limited to, the results of research anddevelopment efforts, the results of preclinical and clinical testing, theeffect of regulation by the FDA and other agencies, the impact ofcompetitive products, product development, commercialization andtechnological difficulties, and other risks detailed in the company'sSecurities and Exchange Commission filings.
AVI Press and Investor Contact:Michael HubbardDirector of Corporate Communications(503) 227-0554Email Contact
For more information, go to www.marketwire.com
Recent Press Release
Advertisement
POS Magnetic Card Readers
Online distributor for point of sale equipment, TYSSO and Pegasus.








