• Tiny CRISPR Protein from Tiny Archaea May Have Big Diagnostic Impact

    If you could listen closely enough to Archaea, you might hear them busily shredding single-stranded DNA, most likely infectious material from a virus. And if you could look closely enough, you might see that the shredding tool used by the tiny organisms was a tiny Cas protein, not the big, bulky Cas9 protein so famous for its part in the CRISPR/Cas9 gene editing system, but another Cas9, a protein called Cas14.Although researchers have been on the lookout for additional Cas proteins that could b
  • Drug Uptake Gets a Boost in Hard-to-Reach Pancreatic Cells

    Antisense oligonucleotides (ASOs) hold incredible potential to be used as effective therapies for a wide range of diseases. One challenge in moving them from bench to bedside has been their limited ability to be taken up by target cells. A collaboration by AstraZeneca and Ionis Pharmaceuticals has made a huge advance in this area by exploiting a receptor and providing a new approach to delivering ASOs into pancreatic β-cells—cells that are notoriously recalcitrant to ASO uptake. The w
  • Merrimack Reviews Pipeline after Phase II Failure of MM-121 in Lung Cancer

    Merrimack Pharmaceuticals said today it will review its entire development pipeline, including its lead candidate MM-121 (seribantumab), after the monoclonal antibody failed a Phase II trial in patients with non-small cell lung cancer—the company’s second clinical failure in four months.MM-121 missed its primary endpoint in the Phase II SHERLOC trial ( NCT02387216 ) by failing to improve progression-free survival (PFS) after being added to docetaxel in patients with heregulin positiv
  • Gut Bacteria Link Dietary Fiber with Liver Cancer

    We are all told that dietary fiber is good for us, but are all sources and forms of fiber equally beneficial to health? Studies by researchers at the University of Toledo (UT) have surprisingly found that mice developed liver cancer (hepatocellular carcinoma; HCC) when fed on diets fortified with refined soluble fibers such as inulin, a probiotic that earlier this year was FDA approved to be marketed as health-promoting.The research, headed by Matam Vijay-Kumar, Ph.D., director of the UT Microbi
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  • Watchdog Report Reaffirms Concerns With Fail First

    A new report by the Office of Inspector General at the U.S. Department of Health and Human Services (HHS) reaffirms concerns with a regulatory decision that undermines the health and well-being of America’s seniors.
    In an abrupt reversal of long-standing policy, the Centers for Medicare & Medicaid Services (CMS) announced that Medicare Advantage health plans can impose “step therapy” requirements on patients who need drugs covered under Medicare Part B.
    The new directive &m
  • 2018 BIO Investor Forum Wraps Up

    As the stock market continued its volatile swings this week, investors at the BIO Investor Forum—which wrapped up today in San Francisco—seemed to take it all in stride and remain focused on the long-term promise of advancements and breakthroughs in the life sciences. 
    Attendance was up over previous years and included attendees from 34 countries, giving the event a distinctly international flavor. Many were drawn to the conference’s expertly led panel sessions and the opp
  • BIO Investor Forum Explores “Digiceuticals” as a New Class of FDA-Approved Therapeutics

    Within the past year, the FDA has launched a provider pre-certification program as a pilot to manage the expansion of “digiceuticals” or digital therapeutics that produce clinical benefits for patients via interaction with a software application or mobile app. This innovative regulatory approach seeks to provide discipline around clinical utility with the iterative improvements enabled by software versus standard manufactured medicines. Given the near-instant worldwide distribution p
  • A Patient-Centric Approach to Investing & Partnering

    Thursday’s Plenary Luncheon Fireside Chat at the 2018 BIO Investor Forum (BIF) featured two industry leaders: Rowan Chapman, PhD., head of Johnson & Johnson Innovation, California, and moderator Deepa Pakianathan, PhD., managing partner of Delphi Ventures.
    Dispensing with the typical question and answer style fireside chat, Chapman and Pakianathan instead engaged in a dynamic, frank and wide-ranging conversation that drew on their diverse experiences. Both began their careers doin
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  • Lessons from “View from the Board: Responding to an Acquisition Offer”

    The “View from the Board: Responding to an Acquisition Offer” panel at the 2018 BIO Investor Forum focused on real world scenarios and practical advice. The moderator, Michael J. O’Donnell, a Partner at Morrison & Foerster LLP, presented a series of developing scenarios and asked for the panelists to respond. The all-star panel included:Corey Goodman, PhD, Managing Partner, venBio Partners
    Linda Grais, MD, former Chief Executive Officer, Ocera Therapeutics
    Nina Kjellson, Ge
  • Fungal RNAs Targeted by Small Molecule Drugs

    Bulging, looping, and twisting, RNA molecules present distinctive three-dimensional shapes, which presumably contain nooks and crannies into which small molecules can nestle, raising expectations that RNA molecules could be “druggable.” Yet few RNA-targeting drugs have been approved to fight human disease. In hopes of developing drugs that could settle into highly structured RNA molecules, scientists based at Yale have devised a new screening technique. This technique, which combines
  • Novartis to Acquire Endocyte for $2.1B, Expanding RLT Pipeline in Prostate Cancer

    Novartis has agreed to acquire Endocyte for $2.1 billion, the companies said today, in a deal that expands the buyer’s radioligand therapy (RLT) pipeline of targeted oncology treatments with a Phase III candidate and several early-stage candidates.Endocyte is a developer of targeted therapies that combine the company’s small molecule drug conjugate (SMDC) technology with companion imaging agents designed to assist in therapy selection.Endocyte’s lead candidate is 177 Lu-PSMA-61
  • Methylation Markers Distinguish Aggressive from Manageable Prostate Cancer

    Scientists in the U.K. and Canada have identified a DNA methylation signature that they claim can distinguish aggressive prostate cancer from more manageable disease, with up to 92% accuracy. The researchers, headed by Norman Maitland, Ph.D., at the University of York’s department of biology, hope that the pattern of 17 epigenetic markers could be used to assess each case of prostate cancer before deciding on whether the patient will need to undergo surgery or radiotherapy, or whether they
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  • Drug-Resistant Bacteria Zapped by Probiotic-Antibiotic Combination

    Scientists at MIT report that by delivering a combination of antibiotic drugs and probiotics, they could eradicate two strains of drug-resistant bacteria that often infect wounds. To achieve this, they encapsulated the probiotic bacteria in a protective shell of alginate, a biocompatible material that prevents the probiotics from being killed by the antibiotic.The team’s study (“ Biofilm-Inspired Encapsulation of Probiotics for the Treatment of Complex Infections ”) is pub
  • On Track for a Record Breaking 2018 – #BIF18 Opening Plenary

    Despite some recent market volatility, the industry is well positioned for long-term growth and success.
    That was the underlying theme of the opening Plenary Luncheon and Fireside Chat at the 2018 BIO Investor Forum (BIF).
    The fireside chat featured an informative and wide-ranging conversation with industry investing veteran Ed Hurwitz, managing director at MPM Capital. The discussion was moderated by Susanne Mulligan, managing director, life sciences banking, at BMO Capital Markets.
    BIO Preside
  • How Companies are Using Microbiome-Based Therapies to Improve Patient Outcomes

    On Wednesday morning at the BIO Investor Forum, leaders from six companies at the forefront of microbiome innovation assembled to discuss the latest developments in microbiome-based approaches to improve patient outcomes, including microbiome transplants, bioengineered probiotics, next-generation antibiotics, and microbiome-based drug discovery platforms. Investments in microbiome-related companies have accelerated across the past three years, demonstrating high investor expectations for results
  • The 2019 Breakthrough Prize Winners Announced

    The winners of the Breakthrough Prize, known as the world’s most generous science prize, were announced on Wednesday morning. Each recipient will receive $3 million for their “transformative” advances toward understanding living systems and extending human life. In its seventh year, the prizes are awarded in the life sciences, physics, and mathematics. “The winners of the Breakthrough Prize in Life Science show us all how it’s done,” said Cori Bargmann, P
  • RNA-Based Gene Therapy Can Be Turned ON or OFF via Synbio Switches

    DNA-based gene therapy, the usual kind of gene therapy, is hard to deliver compared to RNA-based gene therapy. New DNA must enter the nucleus after being transported by nanoparticles or viral vectors, which can be inefficient or immunogenic. But once new DNA is in place, it has the advantage of being controllable. Gene expression may be turned up or down by any of several well-established methods. This kind of control has been lacking for RNA-based gene therapy, even though RNA promises safer, e
  • AXIM Plans Trials for Flagship Candidate after Receiving GMP License

    AXIM Biotechnologies says it plans to proceed with clinical trials for its flagship drug candidate, the cannabinoid-containing chewing gum MedChew Rx ® , after receiving a GMP manufacturing license from the Netherlands’ Ministry of Health, Welfare and Sport.The license, which took effect October 1, will enable AXIM to produce its cannabinoid-based pharmaceutical drug candidates for human clinical trials for multiple indications.“It’s a major milestone for the developme
  • Get To Know GMOs Month: Spooky GMO Myths – Debunked

    Haunted houses, spooky costumes, and trick-or-treating are all scary delights of the Halloween season. But if you want to give yourself a good fright, you need to look no further than a Google image search for GMOs.
    As part of GMO Answers annual Get to Know GMOs Month, we invite you to come with us for an eerie stroll through some of the Internet’s most outrageous GMO myths and images. And then get the real story behind each of these myths.Myth #1: Evil GMO scientists are injecting GMO cro
  • Fish Oils May Slow Breast Cancer Growth via Immune, Inflammatory Mechanisms

    Past epidemiological studies have suggested that women who consume high levels of long-chain omega-3 fatty acids (LC-ω-3FA) have a lower risk of developing breast cancer (BC) than women who consume low levels of omega-3. New research in mice suggests that a diet rich in long-chain omega-3 fatty acids can suppresses the growth and metastasis of breast cancer, and prolong survival, by suppressing tumor-promoting inflammation and boosting anticancer T-cell responses. The scientists, head
  • Cancer Can Change Neutrophils' Behavior Influencing Them to Support Tumor Growth

    Scientists at the Huntsman Cancer Institute (HCI) at the University of Utah (U of U) say they have gained new insights into the microenvironment surrounding different types of lung tumors and on how these complex cell ecosystems may in turn ultimately affect response to treatment. Their study (“ The Lineage-Defining Transcription Factors SOX2 and NKX2-1 Determine Lung Cancer Cell Fate and Shape the Tumor Immune Microenvironment ”) appears in the journal Immunity. “The 
  • #WorldFoodDay | Biotechnology to Feed the World

    Every October 16th the world celebrates World Food Day, honoring the founding of the Food and Agriculture Organization of the United Nations.
    Established in 1945, the UN’s Food and Agricultural Organization is focused on leading international efforts to defeat hunger. And in today’s world, hunger is an increasingly critical issue that the UN is closely watching.
    In fact, the UN has been sounding the alarm that we must double food production to feed the world’s growing populatio
  • Innovation Intersecting with Investing – Welcome to the BIO Investor Forum

    Today marks the 17th opening of the BIO Investor Forum—a place where emerging biotech companies, researchers and investors mull over opportunities to advance innovations in medicines and treatments with their sights trained on bringing products to the market. The conference has attracted over 300 qualified private equity and public investors from around the world, particularly Asia. Their presence here reflects the enthusiasm around the rapidly evolving areas of scientific and medical disc
  • Paul G. Allen, 65, Remembered as Pioneer Philanthropist in Science and Technology

    Paul G. Allen, the philanthropist, investor, and Microsoft co-founder who died yesterday at age 65 in Seattle of complications of non-Hodgkin’s lymphoma, is being remembered for his interest in, and passion for, advancing science, as well as technology, education, conservation, the arts, and community improvement.During his lifetime, Allen gave more than $2 billion to various causes—including the Seattle bioscience institute bearing his name that pursues research through its three di
  • Birth Method Impacts on Brain Development and Behavior

    New research in mice suggests that whether pups are delivered vaginally or by caesarean section (C-section) has measurable effects on the developmental process of neuronal cell death, and alters neonatal brain development and behavior. Results of the studies, by Nancy Forger, Ph.D., and colleagues at the Neuroscience Institute, Georgia State University, are published in the Proceedings of the National Academy of Sciences ( PNAS ), in a paper titled, “ Birth delivery mode alters perinatal c
  • Immunotherapeutic Antibody Helps Prevent Neurodegeneration

    Scientists from the Gladstone Institutes say they developed an antibody that blocks the inflammatory and oxidative activity of fibrin, which contributes to neurodegeneration in the brain, without compromising the protein's clotting function. To come up with a precise and highly effective antibody, the researchers focused on targeting only a small region of the fibrin protein that is involved in activating the immune system in the brain. This way, they avoided interfering with the part of th
  • Crohn’s Subtype Accurately Predicted through miRNA Molecule

    Investigators at Cornell University and the University of North Carolina have just identified that the levels of a single molecule—microRNA-31 (miR-31)—can predict whether a patient has subtype 1 or subtype 2 of Crohn’s disease (CD). This is a critical discovery as patients with subtype 1, unlike subtype 2, often do not respond well to medications and develop strictures—extreme narrowing of the gut tube, requiring surgery once it develops. Findings from the new study were
  • Organic Farming with Gene Editing: Oxymoron or Tool for Sustainable Agriculture?

    A University of California, Berkeley professor stands at the front of the room, delivering her invited talk about the potential of genetic engineering. Her audience, full of organic farming advocates, listens uneasily. She notices a man get up from his seat and move toward the front of the room. Confused, the speaker pauses mid-sentence as she watches him bend over, reach for the power cord, and unplug the projector. The room darkens and silence falls. So much for listening to the ideas of other
  • Sarepta Acquires U.S., ex-European Rights to Lysogene MPS III Gene Therapy Candidate LYS-SAF302

    Sarepta Therapeutics has acquired U.S. and other ex-European rights to Lysogene’s Phase II/III-bound Mucopolysaccharidosis type IIIA (MPS IIIA) gene therapy candidate LYS-SAF302, through a licensing deal that could generate more than $125 million for Lysogene, the companies said.Sarepta will also retain option rights to an additional CNS-targeted gene therapy candidate under the companies’ agreement. Should both that candidate and LYS-SAF302 be developed, Sarepta’s portfolio wo
  • Cancer-Initiating Cells Stay “Stemmy” via CDK1/Sox2 Interaction

    Extreme normality in superficial or seemingly inconsequential matters can be a sign that something is deeply wrong. This thought may have occurred to scientists at the University of Colorado (CU) Cancer Center when they began looking at a population of cancer cells that, unlike most cancer cells, avoids downregulating MHC Class I molecules. Cancer cells tend to deemphasize these molecules because they can display abnormal proteins and attract the immune system’s attention. Cancer cells tha
  • Alzheimer’s Progression Tracked through Antioxidant Levels

    The idea that antioxidants play a large role in the development and progression of Alzheimer’s disease (AD) is not new. However, researchers have met with difficulties in efficiently monitoring the free-radical scavenging molecules in the brain. Yet now, new evidence from investigators at the National Brain Research Centre in Gurgaon, India shows that the antioxidant, glutathione (GSH), which protects the brain from stress, has been found to be significantly depleted in Alzheimer's patient
  • Microsoft, Eagle Genomics Partner on Microbiome Research

    Microsoft and Eagle Genomics will partner on microbiomic research into immune disorders and antibiotic resistance, Eagle said, through a data collaboration that will be the tech giant’s first partnership focused on microbiome genomics.The collaboration—whose value was not disclosed—will combine its Azure cloud technology and Cognitive Services application program interfaces (APIs) with the artificial intelligence (AI)-based microbiome data management platform of Eagle.Eagle&rsq
  • Dementia Linked with Spontaneous Mutations During Embryonic Development

    Research by scientists in the U.K. suggests that many cases of common neurodegenerative diseases that develop later in life, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD), may be caused by spontaneous gene mutations that occur during embryonic development. Based on research that harnessed a combination of ultra-high deep sequencing and mathematical modeling techniques, a team of scientists in the U.K. reported evidence that somatic (i.e., spontaneous, rather than inhe
  • Fat Tissue Found to Be a Surprising Source of Diabetes

    Scientists in Australia say their research points to fat tissue as a source of disease, and widens our understanding beyond the traditional focus on liver and pancreas as the main culprits. The study (“ Protein Kinase C Epsilon Deletion in Adipose Tissue, but Not in Liver, Improves Glucose Tolerance ”) appears in Cell Metabolism . “ Protein kinase C epsilon (PKCε) activation in the liver is proposed to inhibit insulin action through phosphorylation of the
  • World AMR Congress Keynote Panel to Explore Making “Pull” Incentives a Reality

    Here’s a statistic you might not have known: an estimated 700,000 people die each year due to the growing number of infections resistant to treatment. Known as antimicrobial resistance (AMR), this “silent killer” and looming public health threat has severe social and economic consequences that could have a lasting impact on families, individuals and communities across the globe.
    How best to tackle that problem will be the subject of the World Antimicrobial Resistance Congress l
  • Mouse Pups Have Two (Biological) Mommies

    Using embryonic stem cells and gene editing, healthy mice have been bred from two mothers for the first time. The bimaternal mice not only lived into adulthood, they successfully reproduced with male mice to produce healthy offspring. Mice were also produced from two fathers, but, did not live past a few days.The work was published recently in the journal  CellStem Cell in a paper titled, “ Generation of Bimaternal and Bipaternal Mice from Hypomethylated Haploid ESCs with Imprinting R
  • Immune Design Halts Phase III Trial of Lead Candidate CMB305, Ramps Up G100 Development

    Immunotherapy developer Immune Design said it will halt a Phase III trial for its lead pipeline candidate, the cancer vaccine CMB305, following disappointing Phase II results in synovial sarcoma, and will instead accelerate and expand development of its next-furthest along candidate, the intratumoral TLR4 agonist G100.Immune Design said it will “deprioritize” development of CMB305, a prime-boost cancer vaccine designed to target tumors that express the cellular protein NY-ESO-1, whic
  • Dopaminergic Neurons Generated by Unexpected Conversion of Mature Nerve Cells

    In a serendipitous bit of cell reprogramming sleight of hand, scientists have unwittingly managed to convert mature inhibitory neurons in the brains of live adult mice directly into neurons that resemble the dopamine-producing nerve cells that are lost in Parkinson’s disease.The University of Texas (UT) Southwestern researchers had originally set out to prompt supporting glial cells to develop into dopamine-producing neurons, but their approach instead triggered a type of GABA-producing st
  • Novel Parkinson’s Therapies Possible with New Mouse Model

    Parkinson's disease (PD) is a neurodegenerative disorder that is marked by the accumulation of the protein, α-synuclein (αS), into clumps known as Lewy bodies, which diminish neural health. Now, researchers from Brigham and Women's Hospital (BWH) report the development of a mouse model to induce PD-like αS aggregation, leading to resting tremor and abnormal movement control. The mouse responds to L-DOPA, similarly to patients with PD. The team's study (“ Abrogat
  • Gout Risk Factor Less about Diet, More about Genes

    Despite accumulating evidence that gout isn’t all about diet, the condition retains its reputation as the “rich man’s disease”—or the disease that afflicts those who eat richly, regardless of their fortunes. Back in 2008, a study appeared suggesting that about 12% of gout cases could be attributed to dietary causes. A little later, additional studies identified genes that were associated with gout or gout’s chief risk factor, hyperuricemia, or elevated uric ac
  • Max Planck Partners with Themis Bioscience to Develop Oncolytic Virotherapies

    Themis Bioscience said it will expand its measles vector immunomodulation portfolio into immuno-oncology through an exclusive global license agreement with the technology transfer agency of the Max Planck Society to develop and commercialize oncolytic virotherapies.The licensing deal between Themis and Max-Planck-Innovation—whose value was not disclosed—covers therapies based on an oncolytic measles virus platform jointly developed by the Max Planck Institute for Biochemistry and Ebe
  • Probiotic Good Bacteria Use Fengycins to Eliminate Bad Bacteria

    Infections by pathogenic Staphylococcus aureus bacteria cause many tens of thousands of deaths every year, and the threat of antibiotic-resistant strains, including methicillin-resistant S. aureus (MRSA) is escalating. New research by National Institutes of Health (NIH) scientists now suggests how probiotic Bacillus bacteria can eliminate the reservoir of these potentially harmful bacteria that we can harbor in our noses and gut systems, by secreting a compound that blocks a key S. aureus s
  • Drug Importation: Dangerous for Patients, Consumers, and American Communities

    Writing for The Hill, Steve Forbes, Forbes Media Chairman and Editor in Chief, delivered a simple message to the American people about the implications of drug importation: “Your health could be put in unnecessary danger soon.”
    Let’s rewind to July, when Health and Human Services Secretary Alex Azar directed Food and Drug Administration (FDA) Commissioner Scott Gottlieb to establish a drug importation working group. Azar stated that he was open to a variety of solutions, as lon
  • Tracking Molecular Development of Sepsis May Lead to Improved Therapies

    Scientists at the University of California Santa Barbara (UCSB), Sanford Prebys Medical Discovery Institute (SBP), and UC San Diego say they have developed a method for tracking the development of sepsis on a molecular basis. They believe their discoveries could, in turn, lead to more advanced therapies for sepsis that reduce its mortality, minimize the life-long effects for survivors or even prevent the cascade of life-threatening effects before it begins, while reducing the billions of dollars
  • Brent Erickson in Biofuels Digest: It’s Past Midnight in America

    In 1984, President Ronald Reagan used the phrase “It’s Morning in America” as a now famous campaign message to reassure American’s that his economic policies were working. Fast forward nearly 25 years later and it is now past midnight in America – at least for those who contribute to the U.S. biobased economy and rely on key Farm Bill energy titles.
    Brent Erickson, BIO’s Executive Vice President, Industrial and Environmental
    As Brent Erickson, BIO&rs
  • Antipsychotic Drug Blocks Triple-Negative Breast Cancer Growth

    The results of studies by a team of scientists in the U.K. and Spain suggest that the commonly used antipsychotic drug Pimozide could offer a new treatment approach to the most hard to treat form of breast cancer, and potentially for lung cancer. The research, headed by Mohamed El-Tanani, Ph.D., professor of molecular pathology and cancer therapeutics at the University of Bradford, found that Pimozide significantly reduced the size and number of human triple-negative breast cancer tumors in
  • VC Investment Jumps 51% Year-over-Year During Q3

    Biopharma venture capital activity maintained its red-hot pace during the third quarter of 2018, driven by a handful of deals exceeding $200 million, according to a report released this week.Investors raised $4.002 billion in 130 biopharma VC deals during Q3 2018, according to the most recent quarterly PwC/CB Insights MoneyTree™ Report. That’s 51% above the $2.643 billion in 122 deals recorded for July–September 2017.Those figures do not include “stealth mode” inves
  • Myopia Comes Into Gene Expression Focus

    Myopia, or nearsightedness, develops through a molecular pathway distinct from the pathway that leads to hyperopia, or farsightedness. Resolving the molecular blur behind blurred vision, say scientists based at Columbia University, could sharpen the vision of drug developers interested in pharmacological treatments for myopia, which is not only the most common eye disorder, but a vision problem that is becoming increasingly prevalent. In fact, myopia is becoming one of the leading causes of visi
  • USMCA Paves the Way for Biotech Innovation

    After more than a year of high-stakes drama, the U.S. has inked an updated trade agreement with Mexico and Canada.
    The U.S.-Mexico-Canada Trade Agreement, otherwise known as the USMCA, has achieved an important step in bringing our neighboring countries closer to high U.S. intellectual property standards that have made us the world leader in biotechnology innovation.
    To read more about how the updated trade agreement benefits patients, farmers, and consumers around the globe, read BIO’s pr
  • Micropeptide Discovery May Lead to New Gene Therapy for Heart Failure

    Scientists have discovered a micropeptide molecule that can restore normal heart function in mice, according to a study (“ The DWORF micropeptide enhances contractility and prevents heart failure in a mouse model of dilated cardiomyopathy ”) in  eLife . The micropeptide works by preventing calcium dysregulation and remodelling of the heart and could be a promising new gene therapy target to treat heart failure, say the researchers.“ Calcium (Ca 2+ ) dysregulation

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