AVI BioPharma Receives Key U.S. Patents Covering Preservation and Expansion of Stem Cells Using Third-Generation NeuGene Antisense
Broad Patent Coverage Secures AVI’s Central Role in Stem Cell Applications
PORTLAND, Ore. — Jan. 19, 2005 — AVI BioPharma, Inc. (Nasdaq: AVII), today announced the issuance of United States Patent 6,841,542 B2, and notice of allowance on another patent, covering the use of antisense to modulate stem cell maturation. AVI’s third-generation NeuGene® antisense agents, which target transforming growth factor beta (TGF-beta) and other gene targets, have been shown to program stem cells in culture for long-term survival after implantation.
“AVI’s NeuGene technology represents a powerful tool for stem cell therapy,” said Denis R. Burger, Ph.D., chief executive officer at AVI. “The paucity of stem cells recovered from bone marrow or other sources makes robust therapeutic applications very difficult to achieve. When harvested stem cells are cultured to increase their numbers, they most often differentiate and lose their stem cell character.
“AVI’s NeuGene technology has been used successfully to inhibit differentiation in culture, resulting in stem cell preservation and expansion, and ultimately leading to long-term survival and functionality in recipients. This gene-targeted modulation of stem cells with antisense may be the practical key to allowing stem cell therapy to reach its tremendous potential.”
The issued patent, titled “Transforming Growth Factor Beta (TGF-beta) Blocking Agent-Treated Stem Cell Composition and Method,” includes NeuGene antisense inhibition of TGF-beta, which prevents stem cell differentiation while maintaining stem cell survival and the characteristics necessary for therapeutic use. Bone marrow transplant studies involved incubation of mouse donor cells with TGF-beta 1 NeuGene antisense compounds, and resulted in rapid stem cell engraftment and long-term stem cell survival in the recipient mice.
The allowed patent, titled “Antisense Compositions and Cancer-Treatment Methods,” includes methods for treating stem cells with antisense to increase stem cell numbers and therapeutic benefit, as well as to slow the growth of cancer cells.
NeuGene antisense compounds targeting a stem cell adhesion factor known as very late antigen-4 (VLA-4) have been shown to induce the release of stem cells from bone marrow into circulation. This strategy may reduce the need for costly and possibly damaging stem cell manipulation in stem cell therapy procedures. In addition, NeuGene compounds targeting ecotropic viral integration site 1 (EVI-1) have been designed to manipulate stem cells to enhance their therapeutic capacity.
“Stem cell therapy applications are broad and include treatment of autoimmune diseases, degenerative diseases, specific injuries and cancer,” said Patrick L. Iversen, Ph.D., senior vice president of research and development at AVI. “These studies have used NeuGene antisense approaches to regulate and enhance stem cell utility in order to facilitate the development of novel stem cell therapeutics. This positions AVI as a significant resource for all stem cell applications. As part of our broader program in immunology, stem cell applications represent a third nearer-term focus area for the company, along with cardiovascular applications and infectious diseases.”
AVI believes its NeuGene antisense therapeutics have a significant role to play in the future of stem cell research and clinical development. NeuGenes are synthetic compounds that mirror a critical portion of a target gene and bind to specific portions of the target genetic sequence. Like a key in a lock, NeuGene antisense compounds are designed to match up perfectly with a specific gene sequence, blocking the function of the target gene. Unlike other antisense technologies, AVI’s NeuGene drug candidates have exhibited an excellent safety profile with no drug-related adverse events in 11 clinical trials to date.
About AVI BioPharma
AVI BioPharma develops therapeutic products for the treatment of life-threatening diseases using two technology platforms: third-generation NeuGene antisense drugs and cancer immunotherapy. AVI’s lead NeuGene antisense compound is designed to target cell proliferation disorders, including cardiovascular restenosis, cancer and polycystic kidney disease. In addition to targeting specific genes in the body, AVI’s antiviral program uses NeuGene antisense compounds to target single-stranded RNA viruses, including West Nile virus, SARS coronavirus, hepatitis C virus and dengue virus. AVI’s second technology, Avicine®, is a therapeutic cancer vaccine with late-stage trials planned for the treatment of pancreatic cancer. More information about AVI is available on the company’s Web site at http://www.avibio.com.
“Safe Harbor” Statement under the Private Securities Litigation Reform Act of 1995: The statements that are not historical facts contained in this release are forward-looking statements that involve risks and uncertainties, including, but not limited to, the results of research and development efforts, the results of preclinical and clinical testing, the effect of regulation by the FDA and other agencies, the impact of competitive products, product development, commercialization and technological difficulties, and other risks detailed in the company’s Securities and Exchange Commission filings.