New Gene Therapies Hold Out Promise for Patients

BioMedWire 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.

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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