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Potential Blockbuster Gene Therapy Programs to Watch in 2019

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NEW YORK, April 10, 2019 /PRNewswire/ -- 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 about Genprex, please visit Genprex Inc. (NASDAQ:GNPX).

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