Profile: AVI BioPharma, Inc. (AVII.O)

avid BioPharma, Inc. (AVI), incorporated on July 22, 1980, is a biopharmaceutical company specializing in the discovery and development of ribonucleic acid (RNA)-based drugs targeting a range of diseases. The Company has developed and optimized derivatives of its antisense chemistry (phosphorodiamidate morpholino oligomers (PMOs)) that can be designed to target disease mechanisms through mechanisms of action. The applications of AVI’s RNA technology platform include genetic diseases (Duchenne muscular dystrophy (DMD)), infectious diseases (Ebola and Marburg viruses), cardiovascular disease (restenosis) and other early discovery targets. Several of its antiviral programs, including Ebola, Marburg, Junin and Dengue, have been or are funded by the United States government, and other governmental and non-governmental funding. On March 20, 2008, the Company acquired Ercole Biotechnology, Inc. (Ercole).

Duchenne Muscular Dystrophy programs

The Company is developing a series of drugs for the potential treatment of DMD. Boys with DMD have a mutation in the genetic information that codes for the production of a critical muscle protein, dystrophin, which is localized to the sarcolemmal membrane of muscle cells. The absence of dystrophin in muscle cells leads to cell damage, an abnormally permeable cell membrane and causes cell death and fibrotic replacement. As of December 31, 2008, it had two programs underway with drugs that skip exon 51 (AVI-4658) and exon 50 (AVI-5038).

The Company’s Splice Switching Oligomers’ (SSO) drug, AVI-4658, targets the mutations resulting in DMD, forces the genetic machinery to skip over an adjacent piece (one or more codons) of RNA, thus restoring the ability of the cell to process genetic instructions, thereby, allowing for production of a new, albeit truncated, dystrophin protein. It is conducting preclinical evaluations with its SSO drug, AVI-5038, which is designed to skip exon 50 and restore a range of associated mutations.

AVI-5126 Prevention of Restenosis

AVI has partnered with Global Therapeutics, a Cook Medical Company, to evaluate AVI’s cardiovascular restenosis drug for use on a chromium cobalt drug-eluting stent (DES) for the treatment of cardiovascular restenosis. The application of AVI-5126 to the Global Therapeutics Silencer System is designed to offer rapid drug release and reversion to a bare metal stent within hours of implantation.

Infectious Disease Programs

The Company’s infectious disease program is focusing on single-stranded RNA viruses using its Translation Suppressing Oligomers (TSO) technology with its PMOplus chemistry backbone to target the fatal diseases caused by Dengue, Ebola, Marburg and Junin viruses, as well as many of the items included on the Department of Homeland Security’s list of bioterrorism agents, including anthrax and ricin. In November 2008, the Company filed an Investigational New Drug application (IND) with the United States Food and Drug Administration (FDA) for AVI-6002 and AVI-6003.

The Company competes with Prosensa, Santaris, Merck, and ISIS Pharmaceuticals.