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Editorial Coverage: Several revolutionary gene therapies appear
to be on the verge of delivering remarkable remedies for diseases
that have previously been viewed as untreatable.
- Gene therapy is the next great leap forward in medicine, with
several noteworthy programs underway looking to cure the
incurable
- FDA expects 200 new gene and cell therapy INDs in next two
years
- Genprex — and its initial product candidate Oncoprex — is
pioneering a new approach to treating cancer.
Built on decades of scientific research and innovation, gene
therapy is the next frontier of medicine, determined to provide
remedies for previously untreatable diseases and unmet medical
needs. At the forefront of this gene therapy revolution,
Genprex Inc. (NASDAQ: GNPX) (GNPX
Profile) is pioneering a new paradigm in cancer
therapeutics. The company’s first target is non-small cell lung
cancer, and research indicates that Genprex’s novel technology
might also deliver other cancer-fighting genes to combat a variety
of different cancers. Sangamo Therapeutics Inc. (NASDAQ:
SGMO) is conducting landmark studies to treat hemophilia,
inherited metabolic disorders and other serious diseases at the
genomic level. uniQure N.V. (NASDAQ: QURE) is
leveraging its technology platform to advance a pipeline of
proprietary and partnered gene therapies to treat patients with
hemophilia, Huntington’s disease and other severe genetic diseases.
bluebird bio Inc. (NASDAQ: BLUE) is developing
potentially transformative gene therapies for severe genetic
diseases and T cell-based immunotherapies. And Leap
Therapeutics Inc. (NASDAQ: LPTX) is focused on developing
targeted and immuno-oncology therapeutics.
To view an infographic of this editorial, click here.
Exciting Era
Disease has been a subject of both fear and fascination
throughout history. With each medical discovery, the world has
moved a step closer to understanding not only the complex mysteries
of disease but also the intricacies of curative therapies. Over the
course of centuries, medical science has developed medicines and
treatments that have proved instrumental in saving and improving
the quality of lives.
Now gene therapy, arguably the next great leap forward in
medicine, may hold the promise of curing the incurable. Gene
therapy refers to the therapeutic delivery of nucleic acid (DNA and
RNA) into a patient's cells as a method to treat disease.
Originally conceptualized in 1972, the first gene therapy accepted
as a success in a trial took place in 1990. After 45 years of
global research efforts, the first gene therapy to be approved for
use was in 2012 in Europe.
In the past, gene therapies have been slow to gain government
approval. However, as an indication of the efficacy and potential
of gene therapy, the FDA announced in
January it expects to receive some 200 new gene and cell therapy
INDs in the next two years and plans to expedite the processing of
these requests. Not since the advent of antibiotics in the 1920s
has there been such excitement and promise in medicine.
Killing a Killer
The FDA is not the only indication that is pointing toward a
tsunami of gene therapy innovations. Financial market projections
tell the same tale. For comparison, the global immunotherapy drugs market was valued at
$132.59 billion in 2017 and is expected to achieve growth of
$385.46 billion by 2025 at a CAGR of 14.27% from 2018 to 2025.
These eye-popping numbers portend explosive growth and opportunity
both now and well into the future.
Perhaps no other disease strikes as much dread and fear as
cancer. Many successful treatments are available today that didn't
exist just a couple decades ago; however, cancer cells and how they
grow remain unpredictable and, in some cases, mysterious. Even
after seemingly effective conventional treatments, cancer cells can
hide out in some patients and resurface. Genprex Inc.
(NASDAQ: GNPX) is pioneering a new approach to
treating cancer. Genprex’s initial product candidate, Oncoprex, is an immunogene therapy for non-small cell
lung cancer (NSCLC), one of the most common causes of cancer deaths
in the world. About 85% of lung cancers are non-small cell lung
cancer. Survival rates for late stage non-small cell lung cancer
haven’t improved significantly in over a quarter century. Genprex
is in hot pursuit of life-changing gene technologies for patients
facing this dismal outlook.
Oncoprex encapsulates TUSC2, a powerful, proven tumor suppressor
gene, in cholesterol nanoparticles (nanovesicles) engineered to
target cancer cells. Administered intravenously, these nanovesicles
are attracted to the opposite electrical charge of tumors and pass
through the small blood vessels that form around tumors. As Genprex
CEO Rodney Varner
explained, “It’s a systemic targeted approach. The reason we
say targeted is because the lipid nanoparticles are designed to be
attracted like a magnet to the cancer cells and have an opposite
charge.”
Oncoprex is taken up into cancer cells where the TUSC2 gene is
expressed into a protein capable of restoring certain defective
functions arising in cancer cells. The tumor-killing TUSC2 gene
delivered to normal cells is well tolerated, and the nanovesicles
are taken up by tumor cells after Oncoprex
treatment at up to 25 times the rate at which they are taken up
by normal cells.
Genprex research indicates that Oncoprex, in combination with
targeted or immunotherapies, may be more effective than those drugs
used alone. Many currently approved cancer regimens target only
single mechanisms that cause cancer. Oncoprex has a multimodal
mechanism of action whereby it interrupts cell signaling pathways
that cause replication and proliferation of cancer cells,
re-establishes pathways for apoptosis (or programmed cell death) in
cancer cells and modulates the immune response against cancer
cells. Oncoprex has also been shown to block mechanisms that create
drug resistance. Oncoprex has demonstrated minimal side effects
relative to other lung cancer drugs.
Fast Track
Genprex already has over 30 issued patents for
its platform technologies. The company’s promising pipeline
currently includes a phase I/II clinical trial underway to evaluate
intravenous Oncoprex immunogene therapy in combination with another
cancer drug, Tarceva, and pre-clinical research of the combination
of Oncoprex immunogene therapy with immunotherapies.
The company is also in the process of conducting research to
find biomarkers to identify patients most likely to benefit from
Genprex treatments, as well as other drugs that will be synergistic
with Genprex gene therapies. Given the enormous potential and vast
unmet medical need, Genprex plans to apply for Fast Track,
Breakthrough or RMAT designation from the FDA.
Management on a Mission
Genprex is led by Rodney
Varner, chairman and CEO. An experienced corporate lawyer who
served several biotech start-ups, Varner is a cancer survivor
himself and is on a personal mission to find cures for cancer. In
2009, with the help of several renowned scientists and others, he
identified immense potential in the science of bankrupt biotech
that became the foundation for Genprex’s lead drug candidate,
Oncoprex. He recruited a team of leading scientists to rescue what
they were convinced was an incredibly promising undeveloped
science: the TUSC2 tumor suppressor gene delivered to tumor cells
through a novel nanoparticle delivery system. The science and the
team became the cornerstone of what is today Genprex.
Genprex now boasts some of the world’s best scientific minds as
well as a stable of biotech and operational expertise.
Internationally renowned cancer expert and oncology pioneer Jack A.
Roth, MD, FACS, is chairman of Genprex’s Scientific Advisory Board.
Nobel laureate Dr. James Rothman is a strategic advisor to the
board of directors. Prior to being recruited to Genprex, chief
operating officer Julien L. Pham, MD, MPH, was on the faculty at
Harvard Medical School’s Brigham and Women’s Hospital, then founded
a healthcare IT company. CFO Ryan M. Confer, MS, has more than ten
years of executive experience in planning, launching, developing
and growing emerging technology companies. The management team is
further bolstered by Varner and his thirty-five years of legal
expertise in corporate law, capital formation, technology
transfers, licensing, research agreements, clinical trial
contracts, pharmaceutical and biologics manufacturing and process
development contracts, state and federal grants, plus his extensive
knowledge of the biotech industry.
Gene therapy programs may be on the cusp of curing the
incurable, and the companies that deliver these medical miracles
are in an ideal position to reap a bonanza of recognition and
rewards. Given the promise of Genprex’s pipeline of cancer killers
with global blockbuster potential, the company certainly appears to
be one to watch.
The Quest for Solutions
Sangamo Therapeutics Inc. (NASDAQ: SGMO) is
focused on translating groundbreaking science into genomic
medicines with the potential to transform patients' lives using
gene therapy, ex-vivo gene-edited cell therapy, in vivo genome
editing and gene regulation. Founded in 1995, Sangamo has been a
leader in genomic therapy research and development. With years of
experience in developing gene-editing technologies, the company has
become proficient in gene therapy, cell therapy and gene regulation
technology. This range of approaches allows Sangamo to connect the
underlying biology of the disease to the appropriate
technology.
Netherlands-based uniQure N.V. (NASDAQ:
QURE) has developed a modular technology platform to
rapidly bring new disease-modifying therapies to patients with
severe genetic diseases. The company is advancing a focused
pipeline of innovative gene therapies and has established clinical
proof-of-concept in its lead indication, hemophilia B and
preclinical proof-of-concept in Huntington’s disease. uniQure is
focused on delivering on the promise of gene therapy, single
treatments with potentially curative results.
bluebird bio Inc. (NASDAQ: BLUE) is a
clinical-stage company committed to developing potentially
transformative gene therapies for severe genetic diseases and T
cell-based immunotherapies. The company is developing gene
therapies for severe genetic diseases and cancer. bluebird bio is
working across a spectrum of disorders by researching sickle cell
disease and multiple myeloma using three gene therapy technologies:
gene addition, cell therapy and gene editing.
Leap Therapeutics Inc. (NASDAQ: LPTX) is
focused on developing targeted and immuno-oncology therapeutics.
Leap's most advanced clinical candidate, DKN-01, is a humanized
monoclonal antibody targeting the Dickkopf-1 (DKK1) protein, a Wnt
pathway modulator. DKN-01 is in clinical trials in patients with
esophagogastric, hepatobiliary, gynecologic and prostate
cancers.
For more information on Genprex, visit Genprex Inc.
(NASDAQ: GNPX)
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