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Top 10 Pharma Firms of 2015 – GEN

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Big pharma has a big public perception problem among Americans, according to Gallup poll results released September 14. Of more than 1,000 Americans polled, 43% held an unfavorable view of the industry, compared with just 35% who view pharma favorably. Pharma ranks 23rd of 25 industries for which Gallup solicited opinions; the federal government finished dead last.

Investors, however, hold a more mixed view of traditional drug developers. While shares for most biotechs have zoomed, pharma giants are almost as likely to have seen their total values of all outstanding shares—their “market capitalization”—shrink as grow over the past year. Four of 10 companies appearing on GEN’s List of Top 10 Pharma Firms of 2014 (GlaxoSmithKline, Johnson & Johnson, Merck & Co., and Roche) showed smaller market cap totals on this year’s edition of the List compared with last year. Another five companies, however (AbbVie, Bayer, Novartis, Pfizer, Sanofi) showed year-over-year increases in their market caps, which are computed as shares times current market price.

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University vs. industry: Boston gathering takes on biotech’s longstanding divide – Boston Business Journal

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It was like any other contest where startup founders pitch their ideas to a skeptical audience, except the majority of the people doing the pitching as well as in the audience had either gray or no hair.

Most of the 50-plus people gathered in a conference room at Boston’s Harborside Hotel late last week were seasoned biotech executives and consultants, or people with years of experience trying to develop promising academic discoveries. And the “pitch contest” came at the end of a 27-hour session focused on solving a single, looming question that’s haunted the life sciences for decades: How to make sure that the most promising such discoveries make it into the hands of a company with the knowledge and financing to oversee its development.

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A Johns Hopkins team designed an Ebola suit so good, it’s going on the market – The Washington Post

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Youseph Yazdi was surprised by the number of people who jumped in to help design better protective gear for people helping Ebola victims – everyone from freshmen to robotics experts to a wedding-dress maker.

But he was even more surprised when the solutions the team came up with at the hackathon at Johns Hopkins University attracted the notice of leading producers of protective clothing. A version of the suit they designed will be manufactured by DuPont and available early next year, the university announced Monday.

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Expanding the Reach of Precision Medicine – Promising Practices – Management – GovExec.com

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Readers of the NIH Director’s Blog know how excited I am about the potential of precision medicine for revolutionizing efforts to treat disease and improve human health. So, it stands to reason that I’m delighted by the positive reactions of researchers, health professionals, and the public to a much-anticipated report from the Precision Medicine Initiative Working Group of the Advisory Committee to the NIH Director. Topping the report’s list of visionary recommendations? Build a national research cohort of 1 million or more Americans over the next three to four years to expand knowledge and practice of precision medicine.

When the president announced PMI during his 2015 State of the Union address, he envisioned a precise new era in medicine in which every patient receives the right treatment at the right time — an era in which health care professionals have the resources at hand to take into account individual differences in genes, environments, and lifestyles that contribute to disease. To achieve this, PMI’s national research cohort would tap into recent advances in science, technology and research participation policies to build the knowledge base needed to develop individualized care for all diseases and conditions.

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Sisu Global Heath wins $100,000 investment from AOL co-founder Steve Case – Baltimore Sun

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A Baltimore startup that developed a surgical tool to recycle the blood of a patient suffering from internal bleeding landed a $100,000 investment Monday from AOL co-founder Steve Case, winning a “live pitch” competition among eight Baltimore companies.

Sisu Global Health, founded less than two years ago by three women, was singled out for the potential impact its Hemafuse surgical tool could have in emerging markets of sub-Saharan Africa, said Case, CEO of Washington-based investment firm Revolution LLC.

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Roche Drug First to Clear Clinical Trials for Hard-to-Treat Form of Multiple Sclerosis – Scientific American

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Switzerland’s Roche has moved into pole position in the race to launch the world’s first treatment for progressive multiple sclerosis but smaller players are working hard on rival approaches.

While there are a number of treatments for relapsing remitting MS, the most common form of the disease, there are no approved drugs for progressive MS, which is marked by steadily worsening symptoms.

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The beginning of Precision Medicine marks the beginning of the end of common diseases…

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The use of a patient’s or tumor’s DNA sequence to diagnose/characterize a disease and select or discover drugs that have the highest likelihood of response with least toxicity is one way of broadly defining Precision Medicine. Although most doctors would agree that medicine is far from precise, President Obama’s state of the union address in early 2015 made ‘precision medicine’ a household phrase drawing public attention to this burgeoning field. Mayo clinic has adopted the phrase individualized medicine in naming its world class center at Rochester, MN. Some even refer to it as genomic medicine but I like ‘personalized medicine’ (PM) better. Major healthcare organizations in US and around the world are early adopters and are already practicing PM.

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Vtesse, Inc. Initiates Phase 2b/3 Clinical Trial of VTS-270 for Treatment of Niemann-Pick Type C1 (NPC) Disease

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Vtesse, Inc. today announced that the first three patients have been screened for inclusion in its pivotal Phase 2b/3 clinical trial with VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC). This clinical trial follows a Phase 1 study conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

NPC is a progressive, irreversible, chronically debilitating – and ultimately lethal – genetic disease. It is caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen. Vtesse has worked extensively with regulators in the United States and Europe with the goal of conducting its pivotal study under one global protocol that will evaluate safety and efficacy of VTS-270 to support approval of the drug by regulatory agencies in both regions.

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