AVI BioPharma, Inc, a developer of RNA-based drugs, has signed 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' (US 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, 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, 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 US 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."
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.