NetworkNewsWire
Editorial Coverage: Gene therapies are gaining growing
attention within the medical sector.
- Gene therapies help restore or replace the body’s damaged or
missing genes.
- Groundbreaking treatments can stop the spread of cancer cells,
cause cancer cells to die.
- Gene therapies are also being developed for ailments such as
migraines, skin conditions and hemophilia.
Genprex Inc. (NASDAQ: GNPX) (GNPX
Profile) is developing gene therapies for the
treatment of cancer, including its initial drug candidate,
Oncoprex(TM) immunogene therapy. Spark Therapeutics Inc.
(NASDAQ: ONCE) is tackling hereditary diseases through
treatments that augment, suppress or replace mutated genes.
Dyadic International Inc. (OTCQX: DYAI) uses a
fungus-based technology to deliver treatments, initially to humans,
and now to animals. TrovaGene Inc. (NASDAQ: TROV)
limits the growth of cancer through treatments that limit cell
division. Novartis AG (NYSE: NVS) uses
adeno-associated viruses as carriers for a range of treatments
which are proving safe and effective in clinical trials.
To view an infographic of this editorial, click here.
Potentially Life-Saving Therapy
A generation ago, gene therapy might have been considered
science fiction. Digging into the fundamental building blocks of
life and manipulating them to repair damaged bodies seemed nearly
impossible. But much progress has been made over the past few
decades and has shown that this approach is possible not just in
theory but as a practical solution to one of the biggest killers on
the planet — cancer.
The battle against cancer is a difficult and heartbreaking one.
As the disease causes people’s own bodies to attack themselves,
conventional drugs and surgery struggle to conquer the disease. But
gene therapy takes a different approach, digging into the
fundamentals of how bodies work. As more companies experiment with
how gene therapy treatments might work, promising cures appear to
be emerging.
Damaged Genes
Gene therapies are being developed by companies such as
U.S.-based Genprex Inc. (NASDAQ: GNPX). With a
strong focus on clinical research combined with the latest in
medical science, Genprex has a passion for developing therapies
that could transform the way the medical world treats cancer.
Gene therapy is designed to tackle the root cause of cancer:
missing or defective genes in human cells. These genes have faults,
sometimes referred to as mutations, that cause those genes to
behave differently from the way the body needs them to. This can
lead to significant problems with three different types of
genes:
- Genes that encourage the cell to multiply.
- Genes that stop the cell from multiplying.
- Genes that encourage repair to damaged genes and cells.
The damage can take place during cell division or the natural
life process of the cell. Mutations can also be caused by outside
influences, such as carcinogenic chemicals. As mutant cells
multiply, they can spread through the body. And because these
harmful cells are a fundamental part of the body, the body has a
hard time recognizing the life-threatening danger these cells are
creating.
A large part of the research about how gene therapy works has
been focused on the root causes of this cell mutation. The more the
medical world understands how cancer starts and spreads, the better
it is able to counter it. And now, thanks to recent developments
and discoveries, scientists are moving from simply understanding
the problem to actually finding new and potentially more powerful
real-life applications and treatments.
Gene Therapies
Gene therapies, the promising treatments pursued by companies
such as Genprex, are based on turning this problem on its head.
These therapies take genetic code, tiny snippets of biological
information that tell the human body how to build itself, and
insert them into cells. Once in place, successful gene therapies
replace damaged or missing code and help the body cure itself.
To get into the cells, therapies rely on carriers called vectors
to carry new genetic material into cells. The most common vectors
are viruses, as these tiny life forms can already get into cells.
The serious harmful effects of the viruses are removed and replaced
with beneficial genetic code. Dangers to the human body are
actually transformed into defenders. Genprex uses a non-viral
vector, encapsulating the new genetic code in a cholesterol
nano-vesicle, which targets cancer cells.
The inserted genes can do a range of different things. Some
boost the immune system so that it can better fight off attacks.
Some actually interact with existing drugs and other cancer
treatments, often resulting in a better outcome. Some block the
processes that preserve cancer cells, allowing the body to destroy
the mutant cells.
Creating these treatments is challenging work, and the companies
that excel are innovators with the ability to try new approaches.
Their work has won acclaim and awards, such as Genprex’s place as a finalist in the technology innovation
category of the Fierce Innovation Awards.
Turning Death on Its Head
The particular type of treatment being pursued by Genprex
involves turning normal logic on its head and using the process of
normal cell death to save lives.
The cells in a human body don’t have an infinite lifetime. In
common with most multicellular organisms, the body’s cells are
programmed from the beginning to die at a certain point. This
process, called apoptosis, ensures the constant renewal of the
body, nature’s own way of keeping the body healthy. An average
human loses 50 to 70 billion cells per day in this way.
A lack of apoptosis is one of the factors behind cancer. If the
cells don’t die off because of internal stresses or outside
signals, they can grow out of control, with diseased cells taking
over space from healthy ones.
Genprex’s proprietary platform technology delivers tumor
suppressor genes to the site of the cancer. These genes function in
several ways, including restoring the process of apoptosis, or
normal cell death, in cancer cells.
Oncoprex, Genprex’s leading product candidate, interrupts the
signaling pathways that cause cancer cells to replicate and
proliferate; and restores the pathways that lead to apoptosis. The
growth of cancer cells is slowed down, and their natural death
cycle is switched back on, potentially saving the life of the body
they inhabit.
Drawing Attention
Given the proliferation of cancer and the dramatic nature of
these treatments, it’s hardly surprising that anti-cancer companies
have drawn favorable attention. For example, Noble Capital Markets
has started covering Genprex, giving the company
an outperform rating, which could bode well for the future of
the company.
This sort of positive recognition acknowledges not only the
effectiveness of gene therapy as a treatment but the effective
business practices driving many gene therapy firms. It’s only
through well-developed product development lines that therapies can
be established and brought to market.
To this end, Genprex has identified a series of milestones it
plans to pursue. Those milestones include steps such as optimizing
product development and manufacturing, exploring faster approval
paths with the FDA, and working to ensure that successful clinical
trials will see treatments move quickly toward market.
The future of life-saving treatment lies within our cells.
Using Genes for Different Diseases
The impact of gene therapy isn’t limited to cancer. Commercial
gene therapy company Spark Therapeutics Inc. (NASDAQ:
ONCE) specializes in remedies for inherited retinal
diseases, neurodegenerative diseases and ailments that can be
targeted through the liver. The company’s work includes taking on
rare inherited diseases that other treatments may not be able to
tackle, addressing them at their root by augmenting, suppressing or
replacing mutated genes. It has recently announced its sponsorship of a science fair for the hemophiliac
community, as the company raises its profile among those
dealing with rare and debilitating ailments.
Dutch-American biotech company Dyadic International Inc
(OTCQX: DYAI) has centered its work on a fungus-based
technology the company has nicknamed C1. Like similar cutting-edge
treatments, this approach adapts existing biological material to
new ends. C1 may increase the speed and efficiency of creating
drugs, helping to get supplies to market for a wide range of
treatments, including vaccines, antibodies and enzyme therapies.
The company recently announced a sublicensing
agreement with Australian drug company Luina Bio, which will
see this technology applied to the development and
commercialization of C1 medicines for animals.
Among the companies tackling cancer is TrovaGene Inc.
(NASDAQ: TROV), a therapeutics company whose drugs work by
affecting cell division to halt the growth of cancerous cells. A
Polo-like Kinase 1 (PLK1) inhibitor reduces the overexpression of
an enzyme common in cancer cells, changing cell division and the
repair of damage. Recent trials of a new drug indicate potential effectiveness in combination with other
treatments, which would allow oncologists to target more
challenging forms of cancer.
Novartis AG (NYSE: NVS) is using gene therapies
to treat a variety of illnesses. By using adeno-associated viruses
as carriers, the company is working to deliver new treatments to
patients. Its drugs are being developed to tackle the skin condition psoriasis, cardiovascular disease, and migraines, for which recent tests have shown
Novartis’s drugs to be both safe and effective.
Gene therapy treatment appears to offer solutions for a range of
diseases. With its ability to tackle the monster that is cancer,
gene therapy seems likely to continue growing in influence and
importance within the world of medicine.
For more information on Genprex, visit Genprex Inc.
(NASDAQ: GNPX)
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