5
ing by nanoparticle design will be
extremely useful. Some current at-
tempts at gene therapy use viral vec-
tors to direct therapy toward a par-
ticular cell type, and, once there, to
the appropriate location within the
cell in order for the gene therapy to
have its desired effect. To date,
however, the effectiveness of using
viral vectors to introduce DNA into
specific cell types or tissues is quite
variable. Nanoparticles may be able
to deliver nucleic acids to specific
cell types and even to the desired
compartment within those cells.
One biotech company that
BSR
sees to benefit from advances in
nanoparticles aimed at drug delivery
is
Vical. Vical holds broad rights to
most DNA-based drug technologies,
which includes their naked DNA
technology. Vical has significantly
improved the design of DNA mole-
cules to provide increases in effi-
ciency of gene delivery and expres-
sion. In addition, the company is
developing other synthetic technolo-
gies to deliver DNA directly into
some nonmuscle tissues, including
the use of lipid molecules and syn-
thetic polymers that facilitate direct
absorption of DNA into cells.
Thoughts for investors
Given the significant advances
observed during the last decade in
electronics, chemistry and the bio-
logical sciences, there is strong be-
lief that nanotechnology can and
will deliver new technology to fur-
ther advance biotechnology and
medicine. But for the investor look-
ing to profit from new developments
brought to bear from nanotechnol-
ogy, the choices are quite slim at the
moment. In the past, those biotechs
that delivered drugs and therapies to
market performed better than those
companies with tools and instru-
mentation, and this is unlikely to
change in the future. However, for
those investors wanting to capitalize
on nanotechnology in the short term
(3-5 years), then you may want too
seriously consider companies bring-
ing instrumentation and technology
to the market. Those companies that
were highlighted for detection and
susceptibility, and drug discovery
and development should generate
profits, especially with applications
of the human genome project.
For the medium term (5-10
years), look for companies that an-
nounce advances in nanostructure
and nanoparticles, particularly ap-
plying to improvements in biocom-
patible implants and drug delivery.
Few companies were highlighted
here, but we believe that Vical will
benefit from the creation of nano-
particles for drug delivery.
In the long term (10-20 years),
we look for technology that takes
one or more decades of work to
achieve general effectiveness. Com-
panies that can exploit nanostruc-
tures to create chips for drug deliv-
ery, or nanoparticles for cell ther-
apy, would be a good bet.
What is clear from the
nanotechnology boom is that it will
take time to deliver. However, given
the potential applications that ac-
company miniaturization (e.g. com-
puter processors) then the question
should only be when, and not if.
When these anticipated advances do
arrive,
BSR will be there to help
identify the leaders in the field.
#####
BioTech Sage Report, May 2001
Please note: Due to the infancy of
nanotechnology, those companies
mentioned in this report are likely to
be the few key players developing this
technology. As a result, there is no
need to publish the BioStocks Pool.
