Genetic Engineering & Biotechnology News

JUL 2017

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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32 | JULY 2017 | | Genetic Engineering & Biotechnology News a patient. The patient, who suffered a genetic disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID), only experienced a temporary benefit. Sub- sequent clinical interventions, in the late 1990s and early 2000s, highlighted the risks of gene therapy. One patient in the United States died as a result of a gene therapy experi- ment meant to cure a form of liver disease, and several pa- tients in France developed leukemia after they received gene therapy to resolve an immune deficiency. After these setbacks, clinical trials testing gene therapies started to decrease. At the same time, gene therapy was re- tooled. For example, it began to incorporate more effective vec- tors. Eventually, clinical trials picked up, and in 2012 the Euro- pean Union issued its first gene therapy approval. Today, more than 2300 clinical trials using gene therapies are in progress, with over half of trials in Phase I. Finally, after decades of halt- ing progress, gene therapy seems poised to realize its promise. What's the Big Idea? Gene therapy involves replacing or compensating for a defective or mutant gene in one's body by introducing "cor- rect" DNA, effectively treating, and in many cases, curing disease. It promises to treat a wide range of diseases such as cystic fibrosis, heart disease (and its complications), diabetes, hemophilia, AIDS, and an ever-growing list of cancers. Several gene-therapy methods are being investigated. A mutated gene can be replaced so that it expresses a normal phenotype. It can also be "fixed"; that is, it can be turned off so that it no longer promotes disease. Conversely, a healthy gene can be turned on so that it may inhibit disease. In other cases, gene therapy allows the diseased cells to be more evident to the immune system, allowing for normal immune response. It should be noted that gene therapy is still in a very ex- perimental phase. Although it may seem promising, it still poses serious risks. It is constantly being studied to instill confidence in its safety and efficacy. It is studied most inten- sively for its potential against diseases that aren't treatable via traditional methods. The Heart of the Matter Congestive heart failure (CHF) is the condition in which the heart's blood pumping function is compromised and be- comes inadequate to meet the body's needs. Many factors can contribute to CHF, one of which is a downregulation of the adenylyl cyclase type 6 (AC6) protein found in heart muscle, causing a low ejection fraction in the left ventricle. CHF may be treated by a candidate therapy being devel- Gene Therapy: A New Twist on an Old Helix See Gene Therapy on page 34 Renova Therapeutics has several gene and peptide therapies in development for sufferers of cardiovascular and metabolic diseases. Farthest along the development pathway are RT- 100, which delivers a gene to treat congestive heart failure, and RT- 400, which is a peptide infusion therapy for the treatment of acute decompensated heart failure. NAFLD (non-alcoholic fatty liver disease). Translational Medicine Feature Continued from page 1 Insights Molecular Diagnostics London-based medical research charity MRC Technolo- gy (MRCT) and Belgian molecular diagnostic developer Biocartis announced a partnership that will see the two entities work to develop a range of diagnostic tests on the Biocartis Idylla platform. The first project will be a new liquid biopsy test for the monitoring of breast can- cer patients for resistance to hormone therapy. Under the agreement, the financial terms of which were not disclosed, the two companies will codevelop molecular diagnostic tests on the automated PCR- based Idylla platform, with MRCT acting as a develop- ment contractor while Biocartis will handle the com- mercialization of any developed tests. "Building partnerships with third parties to ac- celerate the expansion of our menu of molecular diagnostic tests is a key element in our strategy," said Erwin Sablon, head of R&D and alliance manage- ment at Biocartis. "MRCT has an experienced team in place and we are confident that together we can successfully develop a range of high-quality tests for the Idylla platform, beginning with this first test for breast cancer." According to Biocartis, the initial focus on develop- ing a liquid biopsy test for breast cancer fits well with its continued development of a portfolio of tests for breast cancer. Currently, the company has three RUO liquid biopsy tests for sets of actionable mutations in melanoma and colorectal cancer. For MRCT, the partnership with Biocartis furthers its commitment to funding the development of promising biotech and pharma technologies and potential thera- pies. To date, the charity, which generates its revenue via royalties from its codevelopment activities, has helped nearly 20 start-up companies and aided in the development and launch of a variety of approved drugs including Humira and Keytruda (pembrolizumab). Michael A. Dalrymple, Ph.D., director, diagnostics and science foresight at MRCT, noted that the orga- nization sees the Idylla platform as one that could "impact the way molecular diagnostics for oncology is done today." "This resonates well with our focus of translat- ing medical innovation into viable and accessible treatments and diagnostics that patients can benefit from," Dr. Dalrymple continued. "Breast cancer has an enormous impact on many lives every day and we see this assay as an important project to start our partnership with." n Biocartis, MRC Technology to Develop Liquid Biopsy Test for Breast Cancer

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