Exon Skipping Conference for Duchenne Muscular Dystrophy
The Foundation to Eradicate Duchenne (FED), the CureDuchenne Foundation, AVI BioPharma and Prosensa will host an Exon Skipping Conference for Duchenne Muscular Dystrophy from October 14 -17, 2008 at the Banbury Center of Cold Spring Harbor Laboratory, Cold Spring Harbor, NY. Participants are being drawn from a wide area of research, from chemistry to regulatory authorities, to critically review progress on oligonucleotides as therapeutic agents for Duchenne Muscular Dystrophy.
Over the past years, there have been advances in nucleotide chemistry that have led to oligonucleotides (exon skipping compounds) that retain sequence-specific anti-sense activity, while showing little or no protein binding or associated off-target effects. Additional developments in chemistry have improved intracellular delivery, allowing more potent small molecules showing biochemical efficacy in a larger variety of tissues and cells. Recent studies suggest that many hurdles previously inhibiting movement of anti-sense drugs into clinics and standard of care have now been overcome.
The goal is to end the meeting with a more clear understanding of the hurdles that remain in using oligonucleotides as therapeutic agents and to highlight research strategies that are likely to produce positive outcomes.
This conference is being organized by Adrian Krainer, Ph.D., Professor, Banbury Center of Cold Harbor Laboratory; Eric Hoffman,Ph.D, Center Director, Center for Genetic Medicine Research, Children’s National Medical Center; and Terence A. Partridge, Ph.D., Principal Investigator, Children’s Research Institute, Center for Genetic Medicine Research, Children’s National Medical Center
Participation is by invitation only.
About Duchenne Muscular Dystrophy (DMD)
One in every 3,500 boys worldwide is afflicted with Duchenne muscular dystrophy. They will be in wheel chairs by 12 and, historically, most have not survived their teenage years. Duchenne knows no boundaries and crosses all cultures and races. It can happen in any family. There currently is no cure, but for the first time, there are promising therapies on the horizon.
About The Foundation to Eradicate Duchenne (FED)
The Foundation to Eradicate Duchenne was established by Dana and Joel Wood of Alexandria, Virginia. Their son James Wood was diagnosed in May 2000 with Duchenne Muscular Dystrophy. The Woods are both lobbyists in Washington, D.C., and have devoted much of their time and energies to this cause, working with others to achieve millions of dollars in federal earmarks for Duchenne research and a significant increase in the attention devoted to Duchenne at the National Institutes of Health. For more information on FED, please visit www.duchennemd.org.
CureDuchenne is a non-profit organization founded in 2003 by Debra and Paul Miller, parents of a Duchenne boy. CureDuchenne’s Vision is its name…to cure Duchenne muscular dystrophy. CureDuchenne aggressively seeks the most promising, leading edge research breakthroughs and expedites them to the clinical trial process. The ultimate goal…give the boys that have Duchenne now a chance to live a normal life by expediting the availability of treatments/therapies and or a cure that can give quality of life to Duchenne boys, much like insulin does for diabetics. For more information about CureDuchenne, please visit www.cureduchenne.org email firstname.lastname@example.org.
Prosensa is a Dutch biopharmaceutical company dedicated to the development of RNA based therapeutics targeting diseases with unmet medical needs, in particular neuromuscular disorders. Prosensa’s drug development is based upon a unique and proprietary technology platform involving ‘exon skipping’, enabling correction of mutated RNA. This ability to modulate genes selectively through RNA-based therapeutics could provide a new way to treat a wide range of human diseases. The company has, together with its partner LUMC, a leadership position in fundamental patents, technology, and know-how relating to RNA-based approaches and exon skipping. Prosensa’s lead compound for DMD is currently in clinical phase I/II development and the company has received orphan drug designation both in Europe and the U.S.A. Prosensa is elected the European Venture Company of 2007. For more information on Prosensa, please visit www.prosensa.nl.
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. More information about AVI is available at www.avibio.com.