Gene Therapy Holds Out Hope for Diabetics

NEW YORK, February 28, 2018 /PRNewswire/ --

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Since its first successful use nearly 30 years ago, gene therapy has grown into an important if experimental part of the medical sector. Now it is taking off in a big way, offering treatment for chronic ailments such as diabetes and other conditions. PreveCeutical Medical, Inc. (CSE: PREV) (OTC: PRVCF) (FSE: 18H) (PRVCF Profile) is at the forefront of this segment of healthcare, seeking preventive gene treatments to tackle Type 2 diabetes. MannKind Corp. (NASDAQ: MNKD) is offering other innovations for diabetics, with the first inhalation-based drug for managing insulin levels, and Danish multinational Novo Nordisk (NYSE: NVO) offers support for tackling diabetes, with 85% of its business in this area. The potential of gene therapy is being proved with other ailments, for example the work of Sangamo Therapeutics, Inc. (NASDAQ: SGMO) on hemophilia, Huntington's disease, and HIV. Even obscure diseases are becoming treatable through gene therapy thanks to Audentes Therapeutics, Inc. (NASDQ: BOLD), whose virally carried treatments tackle Criger-Najjar syndrome and Pompe disease.

Cures on a Genetic Level

Gene therapy is one of the most cutting-edge and potentially powerful tools available in modern medicine. By introducing foreign genetic material into a patient, doctors can change the way the human body reacts. This could be used to tackle chronic and difficult to treat diseases, including genetic disorders and tough viruses. The main focus of research so far has been on treating cancer.

Though most work is still in the experimental phase, governments and corporations increasingly recognize the potential of such treatments. Grants and investment for work in this area mean that research is still a major part of the revenue stream for gene therapy companies. As exciting new techniques are developed, a growing number of companies are looking to get in on this vital part of the medical market. One of the most promising areas of research is the treatment of diabetes.

Prevention Before Cure

Among the companies exploring the potential of this technology is PreveCeutical (CSE: PREV) (OTCQB: PRVCF) (FSE: 18H). Founded in 2015, PreveCeutical is dedicated to preventive approaches to medicine. Rather than waiting for the disease to manifest and then dealing with the symptoms, PreveCeutical focuses on stopping the problem in its tracks.

Led by a team with decades of combined experience in research and entrepreneurship, PreveCeutical brings a mix of grounded experience and fresh vision to healthcare. While focused primarily on preventive medicine, the company is exploring both preventive and curative treatments using organic and nature identical products. The aim is to provide effective treatments that combine the complex and often under-utilized potential of nature with the rigor of scientific research.

Gene Therapy

The company's research division, headed by Dr. Harry Parekh, is exploring the potential of gene therapy to treat Type 2 diabetes and obesity.

One of the biggest challenges for gene therapy is getting beyond single gene treatments. If a disease is triggered by a single gene, then the therapy only needs to tackle that one gene - a challenging task in itself. If, like diabetes, it is caused by multiple genes, then more complex treatment is needed. Just understanding the causes of a disease is more complex. This is where PreveCeutical's research is focused.

Through five years of multi-disciplinary research, Dr. Parekh and his team have built up a convincing model of Type 2 diabetes and obesity. They have identified a single gene that causes many cases of these diseases, and so can be targeted to prevent them. This paves the way for clinical work on curing these diseases through gene therapy.


"With diabetes, over-production of a particular protein molecule has been identified and purposed to be responsible for the key drivers of diabetes and obesity, starting patient on an inevitable journey of significant co-morbidity and increased rates of mortality. PreveCeutical's gene-silencing technology would effectively "turn off" the genetic signal which leads to the over-production of this key protein molecule, bringing it back down to safe, normalized levels … Thus gene-silencing does not represent a mere management for diabetes and obesity, it represents the potential for a bona fide cure," Dr. Parekh explains (http://nnw.fm/e2NW2).

Tackling Obesity and Diabetes

Obesity and diabetes are linked and are both becoming more prevalent. Tackling diabetes alone cost $827 billion around the world in 2016. With diagnoses of diabetes rising, in part in response to dietary changes, around 300 million people are currently affected by the disease, with this number expected to reach 600 million by 2020. That's a huge market of customers looking for effective treatments such as those offered by PreveCeutical's gene therapy.

Dr. Parekh's research identified a gene that overexpresses itself in the body, leading to excessive production of insulin and an imbalance in body fats.  Smart therapeutics will aim to prevent the overexpression of the gene, stopping the chain of processes that lead to diabetes and obesity. Targeting systems carry the genetic material to the tissues that do most to produce insulin, so that the gene therapy focuses on the areas where it will be most effective. By tackling the shortcomings in many other treatments, the company is looking to find a way to ensure that the genetic material is more effective when it hits its target. Proprietary chemistry unpacks the treatment in the tissue where it is needed, while the packaging biodegrades to provide extra nutrients and avoid leaving toxic carrier chemicals in the body.

Effective Drug Delivery

This safer, less toxic delivery of gene therapy ties in with one of PreveCeutical's wider research concerns: drug delivery. Its gene treatment for diabetes is a step away from the use of viruses to deliver the genetic material. The viral approach is common and, while effective, can leave toxic elements in the body. Instead, a biodegradable carrier becomes part of the treatment without negative side effects.

This concern with effective delivery is also behind the company's Sol-Gel nose-to-brain delivery system. The effectiveness of drugs is often limited by the effectiveness of the way they are delivered. Digestive enzymes can reduce the impact of swallowed drugs. Injection can become difficult or painful as injection sites inflame.

The Sol-Gel system is designed to provide a more effective, less toxic approach. A liquid form of the drug is delivered to the nasal passage, where it forms a gel. Over the course of up to seven days, it slowly releases the compound it contains, for a steady stream of treatment. PreveCeutical is exploring the use of this system for the increasingly important cannabidiol drug group, aiming to provide a delivery system that avoids the negative side-effects these drugs can have.

According to Grand View Research, the preventative healthcare market will be worth around $432 billion by 2024. People are becoming more aware of the benefits of such treatments, including lower costs and better quality of life. For those with chronic diseases, longer lifespans mean an even greater need to prevent diseases from manifesting. With its innovative technologies and a foot in the door of this market, PreveCeutical is taking the steps to become a major player in preventive medicine.

Exploring Therapies

PreveCeutical isn't the only company exploring the potential of gene therapies to address diabetes. New startup Endsulin is researching the use of gene therapy to treat Type 1 diabetes. Its work builds on research which found a single DNA sequence responsible for promoting and activating the human insulin gene. It aims to produce a single one-time injection which will provide millions of diabetics with better control over their glucose levels and so their health.

MannKind (NASDAQ: MNKD) is also looking at new ways to treat diabetes, among other ailments. The company's Afrezza product is the first inhalation-based drug for managing insulin levels in diabetics. Focused on novel delivery systems, the company is finding ways to help diabetics and those suffering from pulmonary arterial hypertension.

On its website, Danish multinational Novo Nordisk (NYSE: NVO) emphasizes the potential of the technology, stating that "Novo Nordisk finds that the contained use of gene technology is an important tool in the identification, development, and production of pharmaceuticals for patients. These pharmaceuticals could not otherwise be provided in sufficient quantity or quality" (http://nnw.fm/eh7uE). Founded over 90 years ago, the company has become a major global player in the pharmaceuticals market, with over 40,000 employees creating products sold in 180 countries. Novo reports that 85% of its business is concerned with treating and managing diabetes. It has branched out into other chronic diseases, looking at the unserved needs of hemophiliacs beyond hospital care.

With 21 years of experience, Sangamo Therapeutics (NASDAQ: SGMO) is an important player in the field of gene therapy. The company is developing treatments for a wide range of single gene diseases, including hemophilia, Huntington's disease, and HIV. By using gene therapy, genome editing, cell therapy, and gene regulation, it brings a range of approaches to bear in using genes to improve human health.

Gene therapy also has an important part to play in tackling diseases which are less well known but can be equally crippling for sufferers. Audentes Therapeutics (NASDAQ: BOLD) caters to this market. It is developing treatments that use the harmless adeno-associated virus to carry genetic material to the cells of patients. By dealing with diseases such as Criger-Najjar syndrome and Pompe disease, the company is serving a corner of the market that other companies are largely unconcerned with. It identifies the treatments most likely to result in meaningful successes and focuses on developing these cures for niche markets.

Gene therapy is increasingly important to the treatment of chronic disease. Now diabetics will also benefit from the preventive medicine this approach can bring.

For more information on PreveCeutical, visit PreveCeutical (CSE: PREV) (OTCQB: PRVCF) (FSE:18H)

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