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MIAMI, Feb. 28, 2017 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ:OPK), today announces that GeneDx, a subsidiary of OPKO Health, is proud to participate as a founding member in Illumina Inc.’s (NASDAQ:ILMN) iHope Network on International Rare Disease Day, which takes place today.  iHope Network is led by a group of clinical laboratory partners committed to providing clinical whole-genome sequencing (cWGS) to children with undiagnosed rare diseases. The iHope program aims to offer this advanced technology to help end diagnostic odysseys that these patients and their families endure. In addition to GeneDx, the iHope Network currently consists of the following institutions: Illumina, the Garvan Institute of Medical Research, and Hudson Alpha. As an iHope Network partner, GeneDx has committed to donating 10 whole-genome sequencing tests per year. The variants identified through testing will be shared via public variant databases including ClinVar. By sharing this variant information, GeneDx continues its long-standing commitment to sharing data for better patient care while also contributing to the rare disorder community through further collaboration and research. “We are thrilled to have GeneDx as a founding member of the iHope Network, which will transform the lives of pediatric patients with limited access to resources and who need a genetic diagnosis quickly. As a leader in the field, GeneDx’s clinical whole-genome testing will prove invaluable to these families,” said Ryan Taft, PhD, Senior Director of the Scientific Research Population and Medical Genomics Department, Illumina. GeneDx was founded in 2000 by two scientists from the National Institutes of Health (NIH) with a mission to provide diagnostic testing for patients with rare and ultra-rare disorders. Today, GeneDx has grown into a global industry leader in genomics, having provided testing to patients and their families in over 55 countries. Led by its world-renowned whole exome sequencing program, and an unparalleled comprehensive genetic testing menu, GeneDx has a continued expertise in rare disorders. Both GeneDx and the iHope program strive to provide answers to those affected by rare diseases and to increase awareness for these disorders. “We are delighted to become a participating partner of Illumina’s iHope Network,” said Jane Juusola, PhD, FACMG, Director of the Clinical Genomics Program, GeneDx. “As a laboratory founded to address the needs of patients diagnosed with rare genetic diseases, the very principle of the iHope program aligns with our founding mission. Through our donation of 10 whole-genome sequencing tests, we hope to bring closure to the diagnostic odysseys for children with undiagnosed rare diseases.” GeneDx is a world leader in genomics with an acknowledged expertise in rare and ultra-rare genetic disorders, as well as one of the broadest menus of sequencing services available among commercial laboratories. GeneDx provides testing to patients and their families in more than 55 countries.  GeneDx is a business unit of BioReference Laboratories, a wholly owned subsidiary of OPKO Health, Inc.  To learn more, please visit www.genedx.com. For GeneDx’s complete list of testing options, please visit www.genedx.com or email genedx@genedx.com.  Follow on Twitter @GeneDx and become a fan on Facebook @GeneDxLab to get real-time updates. OPKO Health is a diversified healthcare company that seeks to establish industry-leading positions in large, rapidly growing markets. Our diagnostics business includes BioReference Laboratories, the nation’s third-largest clinical laboratory with a core genetic testing business and a 400-person sales and marketing team to drive growth and leverage new products, including the 4Kscore® prostate cancer test and the Claros® 1 in-office immunoassay platform. Our pharmaceutical business features RAYALDEE, an FDA-approved treatment for SHPT in stage 3-4 CKD patients with vitamin D insufficiency (launched in November 2016), VARUBI™ for chemotherapy-induced nausea and vomiting (oral formulation launched by partner TESARO and IV formulation pending FDA approval), TT401, a once or twice weekly oxyntomodulin for type 2 diabetes and obesity which is a clinically advanced drug candidate among the new class of GLP-1 glucagon receptor dual agonists, and TT701, an androgen receptor modulator for androgen deficiency indications. Our biologics business includes hGH-CTP, a once weekly human growth hormone injection (in Phase 3 and partnered with Pfizer), a long-acting oxyntomodulin for diabetes and obesity (in Phase 1). We also have production and distribution assets worldwide, multiple strategic investments and an active business development strategy. More information available at www.opko.com. This press release contains "forward-looking statements," as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), and such statements may be identified by words such as "expects," "plans," "projects," "will," "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding expected benefits of the iHope program, that it will benefit patients with rare disease and better patient care, as well as other non-historical statements about our expectations, beliefs or intentions regarding our business, technologies and products, financial condition, strategies or prospects. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These factors include those described in our filings with the Securities and Exchange Commission, as well as the risks inherent in funding, developing and obtaining regulatory approvals of new, commercially-viable and competitive products and treatments. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, among other factors. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward-looking statements. We intend that all forward-looking statements be subject to the safe-harbor provisions of the PSLRA.

News Article | October 26, 2016
Site: www.eurekalert.org

For the first time, National Institutes of Health (NIH) researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1). The gene therapy involved inserting a functional copy of the NPC1 gene into mice with the disease; the treated animals were then found to have less severe NPC1 symptoms. The study, led by researchers at NIH's National Human Genome Research Institute (NHGRI) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development, was published Oct. 26, 2016, in the journal Human Molecular Genetics. Niemann-Pick disease is a rare and fatal disorder of the central nervous system (the brain and spinal cord) that has no cure. The disease occurs when a faulty housekeeping gene fails to remove cell waste, like lipids and cholesterol. The accumulation of waste in the spleen, liver and brain causes progressive deterioration in the intellectual and motor functions. It also shortens patient's lives, as people with Niemann-Pick disease typically die in their teens. There are several types of Niemann-Pick disease; this study focused on mice that had been bred with a faulty NPC1 gene to model Niemann-Pick disease, type C1. The researchers' goal was to correct the faulty NPC1 gene in as many cells and organs as possible, with a strong focus on the brain. To do this, they used a non-disease-causing virus called the adeno-associated virus serotype 9 (AAV9) to transfer functioning NPC1 to the cells. The AAV9 containing a functioning NPC1 gene successfully crossed the blood-brain barrier, reaching cells in the brain and elsewhere. Once inside cells, the normal NPC1 gene was then able to make the functional NPC1 protein to correct the cell defects. With a single injection, mice showed improvements in motor coordination, weight gain and longevity compared to those without this gene therapy. The effect of gene therapy equaled that of a drug called VTS-270, which has been evaluated in preclinical and clinical studies at numerous academic labs. However, to be effective, the VTS-270 compound has to be given for the life of the mouse. The team is now investigating if a combination of the two therapies will improve results. "We're very encouraged by this preliminary work," said William J. Pavan, Ph.D., senior investigator in the NHGRI Genetic Disease Research Branch. "The gene therapy is treating the root of the problem, the defective gene." "Our work in NPC1 mice may help lead to human clinical trials and eventually FDA approval for gene therapy as a treatment for NPC1 disease," said Charles P. Venditti, M.D., Ph.D., senior investigator in the NHGRI Medical Genomics and Metabolic Genetics Branch. "For NPC1 patients, gene therapy could halt progression of the disease, improve the quality of their lives and, hopefully, increase the patient's life span." The researchers work on gene therapy for NPC1 also has the potential to treat genetic disorders with some similar features. These might include mucolipidosis IV, Batten disease and Danon disease, researchers wrote. The advanced open access article can be found at: http://bit. . National Human Genome Research Institute (NHGRI) is one of the 27 institutes and centers at the NIH, an agency of the Department of Health and Human Services. The NHGRI Division of Intramural Research develops and implements technology to understand, diagnose and treat genomic and genetic diseases. Additional information about NHGRI can be found at: http://www. . About the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): NICHD conducts and supports research in the United States and throughout the world on fetal, infant and child development; maternal, child and family health; reproductive biology and population issues; and medical rehabilitation. For more information, visit NICHD's website. National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 institutes and centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www. .

BASEL, Switzerland and WATERTOWN, Mass., Feb. 16, 2017 (GLOBE NEWSWIRE) -- Lonza Houston, Inc., a global leader in viral gene and cell therapy manufacturing, and Selecta Biosciences, Inc. (NASDAQ:SELB), a clinical-stage biopharmaceutical company focused on developing biologic therapies for rare and serious diseases that avoid unwanted immunogenicity, have entered into a strategic manufacturing agreement. Under the terms of the agreement, Lonza will produce an Anc80-AAV-based gene therapy product for Selecta’s proprietary program for the treatment of Methylmalonic Acidemia (MMA), a rare inborn error of metabolism, and may in the future produce other Anc80-based products for which Selecta holds exclusive options. This relationship will leverage Lonza’s expertise in the development of robust and industry-scale manufacturing platforms for viral-based products.  Data shows that Anc80-AAV, an in silico-designed synthetic gene therapy vector, has the potential to provide superior gene expression levels in retina, liver, muscle, cochlea’s outer hair cells and other tissue targets in preclinical studies, as well as reduced cross-reactivity as compared to naturally occurring adeno-associated viral vectors (AAVs) that are currently in clinical development. “This agreement with Selecta Biosciences continues to demonstrate Lonza’s leadership position in the cell and gene therapy space,” said Andreas Weiler, Ph.D., Head of Emerging Technologies Business Unit for Lonza’s Pharma&Biotech segment. “Lonza will utilize our extensive cGMP manufacturing knowledge and world-class quality systems to help Selecta Biosciences develop promising novel therapeutics for patients impacted by MMA and other devastating diseases.” “We at Selecta are focused on combining novel and proprietary viral vectors with our immune tolerance Synthetic Vaccine Particles (SVP™) to enable the first non-immunogenic gene therapies, providing the potential for repeat dosing,” said Werner Cautreels, Ph.D., Selecta’s president, CEO and chairman. “We view Lonza – one of the industry’s largest contract manufacturers of biologics and a leading supplier in gene therapy – as an ideal partner. They already have invested in developing various expression technologies, and they share our excitement about Anc80. We look forward to working with them to bring the first Anc80-based program into the clinic as a potential treatment for patients afflicted with MMA.” MMA is an inborn error of metabolism that, according to the National Institutes of Health (NIH), affects an estimated one in 25,000 to 48,000 individuals globally. MMA patients are unable to process certain proteins and fats, leading to the accumulation of toxic metabolites. Symptoms start to develop in early childhood and, despite strict diet, patients suffer from a wide range of disease-related complications such as pancreatitis, strokes and chronic kidney failure. Selecta exclusively licensed Anc80 for MMA from Massachusetts Eye and Ear® (MEE) in May 2016. Under the license agreement, Selecta also has the exclusive option to develop gene therapies using Anc80 for additional pre-defined lysosomal storage, genetic muscular and genetic metabolic diseases. Selecta intends to combine Anc80 with recently discovered transgenes and Selecta’s SVP-Rapamycin to create a novel gene therapy candidate for MMA. This candidate is intended to a) enable the treatment of patients with and without pre-existing anti-AAV antibodies; b) prevent cellular immune responses that often reduce the expression levels of gene therapies; and c) provide the ability to administer repeat gene therapy doses to achieve sufficient levels of methylmalonyl-CoA mutase (MUT), the enzyme that MMA patients are lacking. To advance the MMA program, Selecta entered into a Collaborative Research and Development Agreement (CRADA) with MEE and the National Human Genome Research Institute, NIH, in 2016. Principal investigators in this CRADA initiative are Charles Venditti, MD, PhD, Senior Investigator and Head, Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch and Luk Vandenberghe, PhD, Director of the Grousbeck Gene Therapy Center at MEE and an Assistant Professor at Harvard Medical School. A physician-scientist specializing in the study of inborn errors of metabolism including MMA, Dr. Venditti and his group have published several studies showing the effectiveness of gene therapy as a treatment for MMA in mice. Dr. Vandenberghe from MEE is the inventor of Anc80. Lonza is one of the world’s leading and most-trusted suppliers to the pharmaceutical, biotech and specialty ingredients markets. It harnesses science and technology to create products that support safer and healthier living and that enhance the overall quality of life. Not only is Lonza a custom manufacturer and developer, the company also offers services and products ranging from active pharmaceutical ingredients to drinking water sanitizers, from nutritional and personal care ingredients to agricultural products, and from industrial preservatives to microbial control solutions that combat dangerous viruses, bacteria and other pathogens. Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with approximately 40 major manufacturing and R&D facilities and more than 10,000 full-time employees worldwide. The company generated sales of CHF 4.13 billion in 2016 and is organized into two market-focused segments: Pharma&Biotech and Specialty Ingredients. Further information can be found at www.lonza.com. Selecta Biosciences, Inc. is a clinical-stage biopharmaceutical company focused on developing biologic therapies for rare and serious diseases that avoid the immune responses that compromise efficacy and lead to life-threatening complications. Selecta is applying its proprietary Synthetic Vaccine Particles (SVP™) to a range of therapeutic areas in which immunogenicity is a key challenge. SEL-212, the company’s lead candidate in Phase 2, is being developed to treat chronic refractory gout patients and reduce their debilitating symptoms, including flares and inflammatory arthritis. Further, Selecta’s two proprietary gene therapy product candidates have the unique potential to enable repeat administration, allowing for dose adjustment in patients and maintenance of therapeutic activity over time. The company is seeking to expand the use of its SVP platform in other areas, such as immuno-oncology, allergies, autoimmune diseases and vaccines. Selecta is based in Watertown, Massachusetts. For more information, please visit http://selectabio.com. Additional Lonza Information and Disclaimer Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited (“SGX-ST”). Lonza Group Ltd is not subject to the SGX-ST’s continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual. Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this presentation due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this release. Selecta Biosciences, Inc. Disclaimer Any statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (“the company”), including without limitation, statements regarding the development of its pipeline, the ability of the company’s SVP platform, including SVP-Rapamycin, to mitigate immune response and create better therapeutic outcomes, the potential treatment applications for products utilizing the SVP platform in areas such as gene therapy, immuno-oncology, allergies, autoimmune diseases and vaccines, whether the company’s proprietary gene therapy product candidates will enable repeat administration, allow for dose adjustment in patients or maintain therapeutic activity over time, the sufficiency of the company’s cash, cash equivalents, investments, and restricted cash and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hypothesize,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including their uncertain outcomes, the unproven approach of the company’s SVP technology, undesirable side effects of the company’s product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the company’s inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, substantial fluctuation in the price of its common stock, a significant portion of the company’s total outstanding shares have recently become eligible to be sold into the market, and other important factors discussed in the “Risk Factors” section of the company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, or SEC, on November 10, 2016, and in other filings that the company makes with the SEC. In addition, any forward-looking statements included in this press release represent the company’s views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any obligation to update any forward-looking statements included in this press release.

Morris T.J.,Medical Genomics | Butcher L.M.,Medical Genomics | Feber A.,Medical Genomics | Teschendorff A.E.,University College London | And 4 more authors.
Bioinformatics | Year: 2014

The Illumina Infinium HumanMethylation450 BeadChip is a new platform for high-throughput DNA methylation analysis. Several methods for normalization and processing of these data have been published recently. Here we present an integrated analysis pipeline offering a choice of the most popular normalization methods while also introducing new methods for calling differentially methylated regions and detecting copy number aberrations. © 2013 The Author 2013.

News Article | October 29, 2016
Site: motherboard.vice.com

When you walk into a dispensary in any one of the 25 states where medicinal and/or recreational marijuana is legal, you'll likely see display cases lined with a dozen or more strains of weed. Many of these strains, like Pineapple Express or Blue Dream, will sound familiar, but is the Granddaddy Purp you buy in Colorado the same stuff your guy in California is selling you? To answer this question, a company called Medicinal Genomics is creating a repository of cannabis genomes which are stored on the Bitcoin blockchain. The company hopes that its efforts will standardize strain nomenclature so that customers always know what they're getting while also defending the intellectual property rights of those who breed new strains of weed. Medicinal Genomics is a lot like any other cannabis testing company, insofar as they run tests on marijuana plants to look for microbial contaminations and determine their cannabinoid content to help marijuana cultivators comply with state regulations. Yet what sets this company apart is that their labs are also offering customers the ability to sequence the genome of their cannabis plants. Hundreds of strains of cannabis exist and cultivators are working on breeding new strains all the time. In many cases, what sets these strains apart can be difficult to tell with a glance and a sniff—to really see the difference between them, you need to look at the DNA of the plant. Long before the rise of industrial cannabis, the name of the strain didn't really matter. If your dealer was growing pot in his mom's basement and told you that your buds were OG Kush, then that's what they were—who cares if you were actually getting Girl Scout Cookies as long as it got you stoned. But now that marijuana is becoming a regulated industry (at least on the state level), the name of a strain of weed is starting to matter: not only have we turned into a generation of pot snobs, but making sure a strain has consistent qualities is also crucial to its effectiveness as a medicine. As a result, large growers are beginning to think about securing intellectual property rights for their strains, which became a possibility in August 2015 when the first patent for a strain of weed was filed at the US Patent Office. In response to this development, Medicinal Genomics saw an opportunity: it could assuage the fallout from the looming legal battle over strain ownership by allowing customers to register their strains on the Bitcoin blockchain. For $600, growers can now buy a DNA purification kit for one of their plants and ship the genetic material to one of Medicinal Genomics partner labs for sequencing. Once the sequencing is done, scientists at Medical Genomics will compare the strain's genome to a reference strain—in their case, this is Purple Kush—and record its genetic deviations from this reference to differentiate it as a unique strain. Once the genotype of a grower's plant has been determined, the scientists create a file documenting the unique properties of that strain and then runs that file through a cryptographic hash algorithm which scrambles the file's information and produces a random string of numbers and letters known as a hashsum, or fingerprint, for the file. This hashsum representing the file which contains the strain genotype is then tacked on to a Bitcoin transaction (the Bitcoin protocol allows for small amounts of information to be added to a transaction). What this does, effectively, is allow the owner of the strain to have an immutable, publicly accessible time-stamped record claiming their ownership of the strain. If another grower were to claim IP rights for the same strain, the original grower can point to the strain's block on the Bitcoin blockchain as proof that they had been growing this strain before. Although many institutions from banks to national governments are developing their own proprietary blockchains as repositories of sensitive information, for Medicinal Genomics it made more sense to integrate the company's work into Bitcoin rather than trying to create a blockchain solely for strains it had sequenced. "The Bitcoin blockchain has been going since 2009 and it's security is in its proof of work," said Kevin McKernan, Medicinal Genomics' Chief Science Officer and a member of the Human Genome Project's R&D team. "If you're dealing with customers' intellectual property and you're putting it in some side chain that you're supporting, if your network goes down and you don't manage that well, then you've let them all down." As might be expected, Medicinal Genomics has sparked a race among growers to sequence their strains and register them on the blockchain. While this is not the same thing as getting a patent from the US Patent Office for that strain and thus having IP rights for that strain, it does protect that grower in the event that someone else files a patent for that particular strain. In that case, the grower with their strain registered on the blockchain would be protected from an IP-violation lawsuit by whoever filed the patent through previous use exemption rights. Medicinal Genomics currently has about 1000 strains registered on the blockchain, approximately 420 of which are publicly accessible through the company's genomic repository, KannaPedia. Yet McKernan's blockchain-powered cannabis genomics project is about more than avoiding lawsuits and enabling effective branding for growers who want you to know that their Green Crack is actually Green Crack. Ultimately it's a biological history project on a grand scale, which seeks to plot out cannabis' evolutionary history while facilitating and mapping the rapid development of new strains. Despite the unprecedented rise of industrial cannabis in the last few years, the plant's future on a global scale is far from certain. By putting this information in a decentralized, public ledger, McKernan and his colleagues are making sure that their dank genomics will never be lost. "If for any reason we ever got shut down, all the people in the community that have the sequence files we gave them and could recreate our database," McKernan told Motherboard. "I think that's important for the cannabis field. If we ever want to figure out the mitochondrial Eve of cannabis, it can't exist in a centralized database under one company's control."

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