Here’s Why I’m Watching These Six Biotech Stocks


For investors, the end of ASCO in no way means the end

of intriguing opportunities in biotech. Starting today, the 6th
Annual Meeting of the American Society of Gene Therapy begins in Washington, DC,
and with it begins more potential trading opportunities. This meeting, although
not as popular as ASCO, is sexier than ASCO by way of its content. It brings
together scientists and companies to discuss the latest cutting-edge research in
gene therapy for a variety of diseases. The presentations at this meeting are
pushing the envelope in applying gene therapy to the treatment of disease. 

I not only like this meeting
from a physician standpoint, I also like it from a short-term investor
standpoint. Companies presenting at this meeting have come in under the ASCO
radar screen and will not get noticed until the data is presented over the next
few days. Most of the presentations involve preclinical data on gene therapy and
some clinical studies. However, given the current post-ASCO euphoria over
biotechs, I suspect some of these companies will move over the next two days
even if the preclinical data exhibits potential. These are the companies with a
presence at this meeting.


Sangamo BioSciences

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has a major presence at
this meeting with eleven presentations discussing the company’s proprietary gene
transcription factors called zinc finger DNA-binding proteins or ZFPs. Now this
is a mouthful. However, it is very interesting stuff with solid science behind
it.  Basically, ZFPs are proteins that allow scientists to turn genes on or
off. The hope is to use these highly specific gene modulators to turn designated
genes on or off with the intent to treat disease on a genetic level. For
instance, Sangamo BioSciences will present preclinical data on its ZFP
technology that turns on one of the genes (VEGF A) necessary for the growth of
new blood vessels, a process called angiogenesis. We all heard about anti-angiogenesis
drugs to treat cancer (Genentech’s Avastin) during ASCO. However, in treatment
approaches to other diseases doctors often want to induce the formation of new
blood vessels. 

Potentially, this ZFP-induced upregulation of the VEGF A gene and the formation
of new blood vessels could lead to the treatment of atherosclerotic disease in
arteries of the legs or heart. Imagine using gene therapy to induce new blood
vessel formation in damaged heart tissue after a heart attack. I have always
liked this company ever since its birth. It doesn’t have any revenue-generating
products or drugs in clinical trials. However, it does possess exciting new
gene-regulation technology grounded in proven science and all backed by
Massachusetts Institute of Technology brains. 

Other companies represented at this meeting of gene therapy minds
include
Targeted Genetics
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, Avigen

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,
Ariad

Pharmaceuticals

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,
Introgen Therapeutics
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, and last but not least GenVec

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.

All of these companies are developing novel ways to utilize gene therapy for the
treatment of human diseases. 

Targeted Genetics
specializes in technology that inserts genes into targeted cells to potentially
treat disease. Once a desired gene is inserted into a diseased cell, the gene
can be activated to produce a protein product lacking in that particular
diseased cell. What is interesting about this company is that one of its
presentations discusses the preclinical evaluation of its novel vaccine for HIV
infection. This presentation alone could attract some attention just because it
is in the HIV vaccine space.

Avigen also is
developing a technology to facilitate the entry of desired genes into targeted
cells. This company uses an adeno-associated virus to carry specific genes into
cells. Avigen is working on gene therapy to treat Hemophilia A/B (genetic
blood-clotting disorder) and Parkinson’s disease.

Introgen Therapeutics
recently made a splash at ASCO with its new Advexin gene therapy trials. The
company revealed some very interesting clinical data regarding its p53
suppressor gene delivery system (INGN 241) to treat advanced breast and
esophageal cancer. At this meeting, Introgen’s presence may continue to fuel
investor enthusiasm.
GenVec
also presented some encouraging clinical data at the recent ASCO meeting that
basically got lost in the euphoria generated by the heavyweights Genentech and
ImClone. However, at this gene therapy meeting GenVec is presenting a paper that
may attract some investor attention. The company will discuss preclinical data
on its novel treatment for the disease anthrax. We all know about anthrax. GenVec’s
preclinical data on its new treatment approach for this disease has a lot of
promise and should make some news.

Good
Luck,

Paul
Ruggieri MD, FACS   
                Â