Gene Regulation
Immune Regulation
Gene Editing
Gene
Regulation
Most diseases are the consequence of
the expression of foreign genes, or the abnormal or
unregulated expression of the body 's own genes.
In some cases, it is the failure to express a gene that
causes the disease. Recent advances in gene analysis
have provided the information and tools necessary to
develop drugs that intervene in the disease process
at the gene level. For a broad spectrum of diseases,
this approach can be more precise than intervening in
the downstream molecular processes of the disease.
Enzo
's approach to gene regulation involves the introduction
into cellular DNA of a gene that codes for a complementary
RNA molecule that leads to the inactivation of a specific
gene product. A major challenge in designing gene
therapy medicines has been the efficient and safe delivery
of the gene to the appropriate target cell. Gene
delivery is often accomplished using a delivery vehicle
known as a vector. A critical quality of the vector
is its ability to bind to the target cell and effectively
deliver or transduce, the gene into the cell.
It is also critical that the DNA of the vector not produce
proteins or antigens that can trigger an adverse immune
response.
To deliver the gene to the appropriate
target cell, Enzo has developed proprietary vector technology
with the following attributes:
Efficient transduction: A principal
problem to date of most gene therapy programs has been
inefficient transduction, or an unacceptably low rate
of delivery of operating genes to the target cells.
Enzo has achieved transduction rates significantly higher
than those reported by other researchers.
Immunologically 'Quiet ': Transduced
cells often produce non-essential proteins that trigger
an immune response, causing such cells to be cleared
from the body before they can produce a therapeutic
effect. Cells with our StealthVectors® have not
expressed extraneous proteins.
'Smart ' Vectors: We incorporate
into the surface of our vectors proteins with an affinity
for the surface of the cell types intended to be transduced.
By including this targeting mechanism, we create in
essence 'smart ' vectors that preferentially transduce
the intended cell type.
Enzo 's vector technology has broad
applicability in the field of gene medicine.
Immune
Regulation
One of
the most exciting developments in recent years in the field of
immunology is the use of Enzo 's proprietary immune regulation
therapeutic strategy.
This strategy is based on the recent finding that
immune responses can be regulated by the oral presentation of
antigens.
Specifically, the oral presentation of specific
antigens in certain ways has the effect both of suppressing
the immune response toward those particular antigens and
apparently initiating other complementary immune
responses. The
effect can be achieved when the oral presentation occurs
either before or after the body has mounted a specific immune
response to the antigen.
Furthermore, in certain animal model systems it has
been shown that this effect can be transferred through the
transfer of the immune cells. This finding is
immensely important and could have wide ranging implications
for the treatments of a variety of
disorders.
Immune regulation could lead to highly
useful advances in several aspects of medicine.
Several avenues are currently the subject of highly
vigorous research programs pursued under the direction
and leadership of Enzo Therapeutics. These fields
include the treatment of diseases caused by infectious
agents such as HBV and HCV (both chronic active hepatitis
and hepatocellular carcinoma), the treatment of autoimmune
diseases, transplantation medicine and gene therapy.
Enzo 's immune regulation technology
seeks to control an individual 's immune response to
a specific antigen, a substance that the body perceives
as foreign and typically mounts an immune response against
it. Enzo 's proprietary technology utilizes specific
formulations and oral administration of known proteins
to regulate that immune response against the antigen.
We are applying our expertise in immune regulation to develop
proprietary therapeutics for the treatment of a variety
of diseases, including HIV-1 infection, chronic active
hepatitis, and inflammatory bowel diseases, including
Crohn 's Disease.
Gene Editing
Gene editing is the process by which genetic material is
added to cells for the purpose of correcting genetic
defects. Enzo Therapeutics holds the rights to
the highly promising process of gene editing in two
patents (US patents 5,958,681 and 6,358,685) that describe
the various processes that are used for gene editing
and repair. Gene editing differs from gene therapy
in that in gene therapy the desired new genetic material
is added to a cell for the purpose of integrating into
the genome of the cell. For gene editing, the
genetic material added to the cell is generally very
small and is used to repair specific defects in the
genome. This can be extremely useful for cases
where the genetic diseases are caused specifically by
point mutations that can be corrected by gene editing
and repair.
