Here is some information about Avigen I've distilled from their investor information packet:
Avigen is a leader in the development of gene therapy based on adeno-associated virus (AAV) technology. Gene therapy, designed to treat human disease at a more fundamental level of genetic cause rather than treating the resulting symptoms, is emerging as the beneficiary of genomic research that is identifying genetic causes of disease and offers the promise of treating congenital disorders for which there is currently no effective treatment available.
The AAV vector system used by Avigen is a vehicle used to place genes into cells. It is a re-engineered version of the naturally occurring adeno-associated virus, and is thought to offer the best available gene delivery vector for a number of reasons:
1. AAV vectors are highly compatible with the human immune system. They are the least immunogenic (least likely to produce immune system reaction) of any of the available vectors and may even be effectively undetectable to the immune system.
2. AAV vector-delivered genes show long-term expression, suggesting they could offer long-term therapeutic benefit. Other vectors, such as adenovirus-based vectors, introduce viral protein expression in their host cells that become targeted and destroyed by the immune system, thereby stopping production of the therapeutic protein after one cell cycle.
3. The AAV vector uses site-specific integration based on a spot on human chromosome 19 that makes possible a more predictable and less disruptive gene therapy. It is the basis of Avigen's targeted vector integration (TVI) products.
4. Avigen has a patented high-yield manufacturing process for AAV. This gives the company a unique competitive advantage in its manufacturing efficiency (on the order of 1000 times greater yield per batch). This process produces a purer form of AAV without accompanying adenovirus and promises a much more efficient and potentially safer AAV vector.
Avigen's research program is developing a broad pipeline of products based on the AAV vector and TVI site-based integration. Its leadership position in AAV vectors, increasingly accepted as the most effective, safe and durable form of gene therapy and its proprietary AAV manufacturing process make it likely that the company will have a central position in future collaborations and licensing agreements. As more target genes are discovered through research of genomics companies and the Human Genome Project, corporate collaborations to develop effective gene therapies are expected to increase dramatically. Their need for effective gene delivery systems will likely involve Avigen a major player. Further, the advantages of the company's AAV manufacturing process increase the potential for licensing revenues or even a buyout of the company to get access to that technology.
The company has a healthy amount of cash on hand, and its stock, which has traded as high as $13.25, is currently around $4. I believe this company has enormous potential and will likely appreciate as research developments and corporate partnerships are announced. But I own shares in it, so consider all this accordingly and do your own research.
For more information, you can check the Avigen website:
avigen.com
Baird |
