NEW YORK, February 28, 2018 /PRNewswire/ --
NetworkNewsWire Editorial Coverage:
Since itsfirst 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
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.
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