NetworkNewsWire
Editorial Coverage: After thirty years of development, gene
therapy is seeing a surge of progress and profitability.
- Gene therapies tackle diseases by altering the genetic makeup
of patients’ cells.
- Recent mergers and research developments make this a promising
time to invest in the sector.
- Gene therapy companies are developing treatments for everything
from cancer and HIV to skin diseases.
Genprex Inc. (NASDAQ: GNPX) (GNPX
Profile) is developing treatments to
tackle lung cancer. Spark Therapeutics Inc. (NASDAQ:
ONCE), which recently entered a merger agreement with the
Roche Group, targets ailments such as retinal and neurodegenerative
diseases. Dyadic International Inc. (OTCQX: DYAI)
is focused on finding uses for a fungal-based technology that could
bring treatments to market faster. TrovaGene Inc. (NASDAQ:
TROV) uses cell-level treatments to tackle cancer through
inhibiting a specific enzyme. The technology developed by
Novartis AG (NYSE: NVS) uses specific viruses to
deliver treatment for a range of illnesses.
To view an infographic of this editorial, click here.
Saving Lives by Changing Genes
Genes are the fundamental building blocks of life, the lines of
code that tell our bodies how to grow and behave. Since their
discovery, our understanding of how we work has been transformed.
But it’s only since the 1980s and the discovery of gene therapy
that we’ve been able to work directly with genes toward the goal of
saving lives.
Gene therapy involves the introduction of modified genetic
material into a patient’s cells. Though gene therapy was first
attempted on human cells in 1980, it took until 1989 for the first
successful transfer of genes in a person. Early tests were dogged
by challenges and controversy, but researchers persevered. More
than 2,300 clinical trials of gene therapy were carried out between
1989 and 2016, providing a wealth of information about the workings
of gene therapy, as well as the potential. Now, as the technology
surges towards a wave of practical cures for patients, gene therapy
has become a significant sector within the bioscience industry.
There’s never been a better time to buy into gene therapy.
Why Now?
Gene therapy isn’t exactly new. For years, companies such as
Genprex
Inc. (NASDAQ: GNPX) have been working on improvements
to the technology and new ways to apply it in improving and saving
lives. The highest profile work has been done in tackling cancer,
an area in which Genprex specializes. The difficulty of treating
cancer, combined with its origin in cell growth gone bad, makes
gene therapy a natural fit for this area. But plenty of other uses
have also been explored over the past three decades.
If gene therapy has been around for this long, why is now such
an ideal time to invest?
One reason is that the industry has seen a series of mergers and
acquisitions. Small-cap biotech company Spark Therapeutics, which
specializes in gene therapy, was recently bought up by Roche for
$4.8 billion, offering Spark investors a likely gain of 190 percent
for this year. MeiraGTx Holdings, another small-cap gene therapy
company, achieved an $80 million private placement, with Johnson
& Johnson as its lead investor, pushing its shares up 67
percent so far this year. These and other moves show the interest
of big medical companies in gene therapy.
This evidence of the faith of the markets has helped boost stock
values for a range of gene therapy companies, with some seeing triple digit growth. But these companies
don’t need to be the targets of mergers to benefit from this. A
rising tide lifts all boats, and a shift in perspective that sees
gene therapy as a mature and profitable endeavor increases
confidence in companies such as Genprex, which are still working
away in their own corners.
Another reason for the rising confidence in gene therapy
companies — and a driver for these mergers — has been the
successful development of new therapies. Recent research has seen developments in the use of
gene therapy for a wide range of diseases, including such high
profile and solidly funded areas as cancer and HIV.
This research has led to the development of new and exciting
treatments. BioMarin is testing
a gene therapy for hemophilia, which holds potential to more
efficiently treat a difficult and expensive-to-tackle disease that
can currently cost hundreds of thousands of dollars a year in
treatment. Genprex is working on Oncoprex to tackle
non-small cell lung cancer. News of promising results for a
muscular dystrophy drug, alongside the purchase of a smaller gene
therapy company, rallied shares in
Sarepta Therapeutics and sent the stock up 34 percent in the
first two months of 2019.
New therapies boost investor confidence and provide companies
with more options, just like any tech innovation. And because of
the special nature of their work, these companies do much more than
that.
Saving Lives
Simply put, gene therapy can save lives. Like sustainable
energy, gene therapy is one of those investments that makes the
world a better place even as it makes the investor a profit. Money
going into this sector is literally changing the world.
Gene therapy saves lives by altering the genetic makeup of a
cell. Diseases can be caused by faulty copies of genes that don’t
create the proteins they should within a cell. The insertion of
better genes can restore function to the cell, building a healthier
body from the most basic building blocks up. That’s why the work of
companies such as Genprex can be so transformative.
Delivery systems for the genes are as important as the genes
themselves. Carriers called vectors deliver the genetic material
into the cells. These are often viruses, turning a life form that
is hostile to humanity into one that benefits us. In the case of
Genprex’s Oncoprex treatment, the genes are carried in an
intravenously delivered nanovesicle, or a tiny compartment like
those found in cells. The Oncoprex nanovesicle is small enough to
cross barriers into the lungs, where it can deliver its genetic
material to the cancer site.
Once in the lungs, the drug targets the negatively charged
cancer cells. It’s taken into the cells, where it produces a
protein that can restore failing functions caused by the cancer.
The treatment interrupts the pathways that cause cancer cells to
reproduce and rebuilds the pathways that lead these cells to die.
It also blocks mechanisms that cause resistance to anti-cancer
drugs.
This uses one of two forms of gene therapy: gene replacement
therapy. Currently, gene replacement
therapies are the only ones approved by the FDA. The
alternative, gene editing, has been the subject of various clinical
trials but has not yet led to an approved treatment. For now, gene
replacement therapy, or tackling unhealthy cells through the
addition of healthy genes, is the proven way forward to gene
therapy, and the approach appears to be serving Genprex well.
The Gene Therapy Industry
Advancements in gene therapy over the past thirty years have led
to a range of companies specializing in this area.
Spark Therapeutics Inc. (NASDAQ: ONCE) is a
commercial gene therapy company that specializes in treatment for
inherited retinal diseases, neurodegenerative diseases and diseases
that can be treated through the liver. The company tackles rare
inherited diseases at their root by treating mutated genes,
augmenting, suppressing or replacing those genes, depending upon
how they negatively affect the body. The company’s expertise and
technology has drawn the attention of the Roche Group, and the two
entered into a merger agreement in February.
Spark will continue to operate as an independent company within the
Roche Group but with the benefit of that group’s substantial
financial backing.
Based in Florida and the Netherlands, Dyadic
International Inc. (OTCQX: DYAI) is a biotech company that
focuses on finding applications for a fungus-based technology that
the company has nicknamed C1. This can be used to increase the
speed and efficiency with which drugs are created. The technology
is used in a wide range of treatments, including vaccines,
antibodies and enzyme therapies. The company placed
at No. 36 on the 2019 OTCQX Best 50 list, a list of the top 50
companies listed on the OTCQX market.
TrovaGene Inc. (NASDAQ: TROV) is an oncology
therapeutics company developing drugs that will affect cell
division to tackle the growth of cancers. The company’s treatments
work by using a Polo-like Kinase 1 (PLK1) Inhibitor, which reduces
the overexpression of an enzyme common in cancer cells. By changing
cell division and the repair of damage within the cell, these
treatments are designed to save the lives of cancer patients.
TrovaGene currently has two ongoing clinical trials and will be
reporting on its progress at the American Association for Cancer
Research Annual Meeting in April.
Novartis AG (NYSE: NVS) is working on gene
therapies using adeno-associated viruses (AAVs) to deliver the
treatment. The company uses innovative science and technology to
address some of society’s most challenging healthcare issues.
Recent research by Novartis has shown successful
results in treating patients with the skin condition psoriasis,
with nine out of ten patients achieving clear or almost-clear skin
during the first 16 weeks of treatment. The company is also working
on issues such as cardiovascular
disease.
Gene therapy is opening up new treatments for a huge range of
diseases and appears to hold out promise for a healthier world.
For more information on Genprex, visit Genprex Inc.
(NASDAQ: GNPX)
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