• Drug Combo Can Regenerate Hair Cells, May Restore Hearing Loss

    Each of us has just 15,000 cochlear hair cells, which is a problem because our hearing wanes a little bit every time one of these cells is lost or damaged. Ordinarily, we cannot regenerate these cells, so when we lose large numbers of them due to noise exposure, toxic drugs, or aging, we’re out of luck. But hair cells have been regenerated in the laboratory, raising hopes that treatments could be developed to restore full hearing to those who have suffered hearing loss due to hair cell def
  • Smallest CRISPR So Far Comes from Food Poisoning Bacteria

    Being able to package the genome editing tool CRISPR/Cas9 into a delivery system that can readily target a vast number of tissues in the body has been the goal of molecular biologists since the editing technique as discovered a few years ago. Now, scientists at the Center for Genome Engineering, within the Institute for Basic Science (IBS) in Korea, in collaboration with ToolGen, and Seoul National University have engineered the smallest CRISPR/Cas9 to date, delivered it to the muscle cells and
  • Argos Says Lead Candidate Likely to Fail Phase III Trial

    Argos Therapeutics said today that its lead product candidate rocapuldencel-T is likely to fail a Phase III trial assessing the cancer immunotherapy in combination with Pfizer’s Sutent ®( sunitinib malate) and standard-of-care for metastatic renal cell carcinoma (mRCC).Argos said its disclosure followed a planned interim data review from the Phase III ADAPT trial by the trial’s Independent Data Monitoring Committee. The committee recommended that the study be halted, concluding t
  • New Icahn Stake Causes BMS Stock to Rise

    Carl Icahn is reported to have taken a stake in Bristol-Myers Squibb (BMS) yesterday, the same day the pharma giant announced plans to satisfy another activist investor by adding three directors to its board and buying back $2 billion of its stock.News of Icahn’s reported stake in BMS sent shares higher at the close of trading yesterday, with the stock climbing to $57.21 before sliding to a closing price of $54.78, up 0.35% from $54.59 at the close of the previous trading day Friday.Icahn&
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  • FDA Clears SeptiCyteâ„¢ LAB for Suspected Sepsis Patients

    The FDA granted 510(k) clearance to Immunexpress for the use of its SeptiCyte™ LAB as an aid in differentiating infection-positive (sepsis) from infection-negative (SIRS) systemic inflammation in critically ill patients on their first day of intensive care unit admission.SeptiCyte LAS is the first RNA-based clinical diagnostic tool, direct from whole blood, to aid medical providers in the early identification of infection in suspected sepsis patients, according to the company.“Sepsis
  • Celltrion's Rituximab Biosimilar Truxima Approved in Europe

    The European Commission cleared Celltrion Healthcare’s rituximab biosimilar, Truxima™, for all approved indications of its reference drug, Roche’s blockbuster MabThera/Rituxan, including non-Hodgkin’s lymphoma (NHL), chronic lymphocytic leukemia (CLL), rheumatoid arthritis (RA), granulomatosis with polyangiitis and microscopic polyangiitis.Truxima is the first biosimilar approved for a cancer indication in Europe. “We are excited to offer the first biosimilar m
  • Vaccine Efficiency and Limited Autoimmunity May Be Enhanced by Plasma Cell Discovery

    A research team led by investigators at the University of Pennsylvania School of Veterinary Medicine says it has come to a better understanding of how plasma cells, which are long-lived B cells that reside in the bone marrow and churn out antibodies against previously encountered vaccines or pathogens, are maintained by the body.While plasma cells are vital components of the immune system, they can also be a contributor to disease, as is the case in autoimmune diseases, such as lupus and rheumat
  • Lantheus and GE Healthcare Propose Global Deal for CAD Imaging Agent

    GE Healthcare and Lantheus Medical Imaging signed a term sheet for the continued Phase III development and global commercialization of Lantheus’s flurpiridaz F 18, a positron emission tomography (PET) myocardial perfusion imaging (MPI) agent for diagnosing coronary artery disease.The proposed deal, which the firms announced late yesterday, would see GE Healthcare take over all funding for the program, including a second Phase III study, and global commercialization. Priority territories in
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  • Encouraging Progress toward Reproducibility Reported

    At AAAS 2017, a pair of panel discussions addressed the reproducibility crisis in science, particularly biomedical science, and suggested that it is manageable, provided stakeholders continue to demonstrate a commitment to quality. One panel, led by Leonard P. Freedman, Ph.D. president of Global Biological standards Institute (GBSI), was comprehensive. It prescribed a range of initiatives:drive quality and ensure greater accountability through strengthened journal and funder policies; create hig
  • Scientists Create Active Controllable Electronic DNA Switch

    DNA has unique electrical properties that have for years interested pioneering engineers trying to advance the development of tiny, low-cost electronic devices. Now, a research team led by scientists at Arizona State University (ASU) have developed the first controllable DNA switch to regulate the flow of electricity within a single, atomic-sized molecule—much like flipping your light switch at home, only on a scale 1000 times smaller than a human hair."It has been established that charge
  • Sarepta Sells Priority Review Voucher to Gilead for $125M

    Sarepta Therapeutics said today it will sell its Rare Pediatric Disease Priority Review Voucher to Gilead Sciences for $125 million upfront.Sarepta obtained the voucher when the FDA in September approved the company’s Exondys 51™ (eteplirsen) as a treatment for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.Gilead’s identity as the buyer was disclosed by Sarepta in a regulatory filing. Gilead emerged from “multiple pharmaceutical and biotech companies&rdqu
  • Trevena's Oliceridine Analgesic Meets Primary Endpoints in Two Phase III Studies

    Trevena confirmed that it remains on track to file an NDA with FDA later this year for its intravenous analgesic, oliceridine, on the back of positive topline data from two placebo-controlled Phase III studies. The pivotal APOLLO-1 and APOLLO-2 studies evaluated three doses of oliceridine in the management of moderate-to-severe acute pain following bunionectomy (APOLLO-1), and abdominoplasty, (APOLLO-2).Data reported today confirmed that both trials met their primary responder rate endpoints, an
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  • Marine Snail Venom May Serve as Opioid-alternative for Pain Relief

    Researchers at the University of Utah reportedly have found a compound that blocks pain by targeting a pathway not associated with opioids. Work with rodents indicates that the benefits continue long after the compound have cleared the body, notes the study published online in PNAS.Opioids are highly addictive and, according to the Centers for Disease Control and Prevention, 91 Americans die every day from an opioid overdose. The medical community is in need of alternative therapies that do not
  • Roche/Chugai's Alecensa Wins Conditional EC Approval for ALK-Positive NSCLC

    Roche and Chugai’s oral ALK inhibitor, Alecensa® (alectinib), was granted conditional approval by the European Commission as monotherapy for treating anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC) in adult patients who have previously been treated with crizotinib.Alecensa was originally discovered by Roche’s majority-owned Japanese drugmaker, Chugai. Roche has developed the drug in markets including the U.S. and Europe. Conditional approval
  • BioSense, Neovacs Agree €65M Deal for Therapeutic SLE Vaccine in China

    BioSense negotiated an option to licence Neovacs’ Phase II-stage IFNalpha Kinoid therapeutic vaccine candidate for treating systemic lupus erythematosus (SLE), in China and selected territories. Under terms of the deal Neovacs could receive up to €65 million (approximately $68.5 million) in up front and milestone payments, not including any future sales royalties. The deal is the second signed by Neovacs for the IFNalpha-Kinoid candidate. During late 2015 the firm inked a strategic pa
  • May the Force of Biotechnology Be With You: What We’re Reading, February 2016

    Each month, GMO Answers compiles a few of our favorite news stories into one post on the GMO Answers website.  This post covers a wide variety of topics related to agriculture and biotechnology.
    The latest installment in the Star Wars franchise isn’t the only thing providing a New Hope. Supporters of biotechnology can Strike Back against the myths about GMOs. You don’t have to graduate from the Imperial Academy to know the facts about genetically modified or
  • Cancer Runs Slower When Its Clock Is Ticking

    Most of us work more quickly when we’re “on the clock,” but the same cannot be said of cancer cells, which never proliferate more hastily than after they “punch out,” that is, after they lose any sense of circadian rhythm. Cancer cells spin out of control not only if their internal biological clocks stop entirely, but also if they simply malfunction.Although circadian disruption has long been associated an increased pace of carcinogenesis, scientists had never demon
  • ArQule, Daiichi Sankyo Say Cancer Candidate Tivantinib Fails Phase III Trial

    Daiichi Sankyo and ArQule acknowledged today that ArQule’s lead product, the hepatocellular carcinoma (HCC) candidate tivantinib (ARQ 197), has failed the Phase III METIV-HCC trial.Tivantinib did not meet its primary endpoint in METIC-HCC of improving overall survival, the companies said. Their statement did not include details of the results, saying those would be presented at an unspecified “upcoming scientific forum.”METIV-HCC—which stands for MET-high patients receivi
  • Amygdala Acquires Phase II-Ready Addiction Candidate from Gilead

    Amygdala Neurosciences has acquired the mid-stage behavior and substance addictions candidate GS-6637 from Gilead Sciences for an undisclosed price.“Completion of this transaction launches Amygdala Neurosciences with a Phase-II ready asset that we believe has the potential to become a treatment for addiction,” Peter Strumph, Amygdala's co-founder and CEO, said yesterday in a statement. “In 2017, we look forward to initiating clinical trials for the treatment of both cocaine and
  • Sartorius Joins EMBL Program to Support Academic Training

    Sartorius has joined the European Molecular Biology Laboratory’s (EMBL) Corporate Partnership Program of initiatives, which are designed to offer industrial support to academic training and education. Sartorius will in addition fund travel bursaries for teachers attending courses run by EMBL’s Learning Laboratory for the Life Sciences, or ELLS.The EMBL Corporate Partnership Program includes training courses, conference sponsorship, and workshops for young scientists at the EMBL Inter
  • Eye Study Advances Ability to Improve Clinical Measurement of Retinal Degeneration

    University of Alabama at Birmingham researchers studying rod cell proteins GARP1 and GARP2 to learn how they function in normal phototransduction in the eye have found that GARP2 accelerates retinal degeneration in mice that lack another rod cell protein (cGMP-gated cation channel beta-subunit) that is involved in producing the electrical signal.The knockout genetic background they used allows the potentially detrimental effect of GARP1 and GARP2 to be amplified, and also allows the possible rol
  • Capricorn Drops Cenderitide to Focus on Cell and Exosome Therapies

    Capricor Therapeutics is dropping further development of its Phase II-stage heart failure drug, Cenderitide, and is terminating its licence agreement with the Mayo Clinic for the natriuretic peptide receptor agonist program. "Our decision to return these rights is a strategic move as we prioritize our efforts to advance our core cell and exosome-based therapeutic development programs," said Dr. Linda Marbán, Ph.D., Capricor president and CEO.Capricor said it expects to reach key clinical
  • Capricor Drops Cenderitide to Focus on Cell and Exosome Therapies

    Capricor Therapeutics is dropping further development of its Phase II-stage heart failure drug Cenderitide and is terminating its license agreement with the Mayo Clinic for the natriuretic peptide receptor agonist program. "Our decision to return these rights is a strategic move as we prioritize our efforts to advance our core cell and exosome-based therapeutic development programs," said Linda Marbán, Ph.D., Capricor president and CEO.Capricor said it expects to reach key clinical milest
  • Epithelial Cells Put on the Rack to Extract Turnover Secrets

    It was a stretch, but an investigation into epithelial cell turnover has revealed how cell division keeps pace with cell death. By subjecting epithelial cells to opposing mechanical tensions, scientists based at the Huntsman Cancer Institute (HCI) and at the University of Utah discovered that stretching causes cell division. Also, the scientists learned that crowding causes cell expulsion and death.The rate of cell division must match the rate of cell death if cell populations are to maintain he
  • Major Malaria Vaccine Breakthrough

    The findings from two new studies have just been released describing the efficacy of a malaria vaccine, provided by the biotech company Sanaria. In the small, controlled clinical trials, the vaccine proved to be extremely efficacious and sustained effectiveness over a number of weeks. The new vaccine—called Sanaria ® PfSPZ-CVac—is composed of live, attenuated, and purified malaria sporozoites and the antimalarial drug chloroquine. Results from the two studies were published in Na
  • Lonza to Manufacture Selecta Gene Therapy Candidate for MMA

    Lonza’s viral-based therapeutics unit Lonza Houston has agreed to manufacture an Anc80-AAV–based gene therapy product for Selecta Bioscience’s proprietary program for the treatment of methylmalonic acidemia (MMA) and may produce other Anc80-based products for which Selecta holds exclusive options, the companies said today.The companies said their strategic manufacturing agreement—whose value was not disclosed—will apply to Selecta’s program Lonza’s exper
  • Rare Muscle Disease Treated Successfully with Gene Therapy

    Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease, according to scientists at the University of Washington Medicine Institute for Stem Cell and Regenerative Medicine.The disease arises from a mutation in genes that normally make myotubularin, a protein essential for proper muscle function. Puppies with this naturally occurring mutation exhibit several features of babies with
  • Vedanta Teams with Stanford and Leiden Universities for Microbiome Therapeutics

    Microbiome therapeutics firm Vedanta Biosciences has established separate partnerships with researchers at Stanford University School of Medicine and at Leiden University Medical Center.The collaboration with Stanford University’s Kari Nadeau, M.D., Ph.D., will analyze connections between the gut microbiome and responses to oral immunotherapies in children with food allergies. Professor Nadeau is director of the Sean N. Parker Center for Allergy and Asthma Research.Vedanta’s partners
  • Valeant Psoriasis Drug Gets FDA Nod with Boxed Warning

    FDA approved Valeant Pharmaceuticals’s injectable plaque psoriasis therapy, Siliq (brodalumab), but with a black box warning of suicidal ideation and behaviour. Siliq availability will also be restricted through a Risk Evaluation and Mitigation Strategy (REMS) program. "Patients and their health care providers should discuss the benefits and risks of Siliq before considering treatment,” said Julie Beitz, M.D., director of FDA’s Office of Drug Evaluation III, Center for Drug Eva
  • Judges Side with Broad Institute in CRISPR Patent Dispute

    A three-judge panel of the Patent Trial and Appeal Board (PTAB) has ruled in favor of the Broad Institute of MIT and Harvard in the bitter legal battle royal with University of California (UC), Berkeley, over who invented CRISPR (clustered regularly interspaced short palindromic repeats) gene-editing technology.The PTAB panel found “no interference in fact” between 12 patents related to CRISPR technology that list as inventor Feng Zhang, Ph.D., of the Broad Institute, and a patent ap
  • Designer Babies Yes, Superbabies No

    If King Solomon had a science advisory group, it might have floated the idea of a baby-splitting policy, helping him anticipate which action he might take should he ever have to decide which of two women was a baby’s true mother. As things turned out, Solomon delivered a famously wise ruling. But today we’re less inclined to rely on seat-of-the-finery policy deliberations. Instead, we ask bodies such as the National Academy of Sciences (NAS) and the National Academy of Medicine (NAM)
  • One Step Closer to Predicting Baldness

    Will the future of genomics lead to the abolishment of the male combover, spray on hair, or adverse side effects of preventative medicine for hair loss? Men around the world can only hope. Yet, results from a new study by researchers at The University of Edinburgh could allow some men to let their hair down, as the findings will allow scientists to make better predictions as to who may go bald. While previous work on male pattern baldness had uncovered a handful of genes related to the condition
  • BTG and Society to Study Minimally Invasive Therapies in Immuno-Oncology

    BTG said today it will partner with the Society of Interventional Oncology (SIO) to study the potential of minimally invasive therapies in immuno-oncology, through a collaboration whose value was not disclosed.The specialty drug developer and interventional oncology member organization reason that an expanded number of cancer patients could benefit from combinations of immuno-oncology agents with minimally invasive, loco-regional therapies of interventional oncology.“Our portfolio includes
  • X-Chem, Janssen Expand Inflammatory Disease Collaboration

    X-Chem said today it has expanded its more than 2-year-old collaboration with Johnson & Johnson entity Janssen Biotech focused on discovering new treatments for inflammatory disease.Under the expanded collaboration—whose value was not disclosed—the partners said they will use X-Chem’s DEX™ platform to identify novel modulators for “challenging” disease targets. The multitarget expansion follows the licensing by Janssen last year of multiple series of X-Che
  • Merck Halts Pivotal Alzheimer's Disease Drug Trial on DMC Recommendation

    Merck is halting the pivotal Phase II/III EPOCH trial evaluating its Alzheimer’s disease (AD) candidate verubecestat on the recommendation of the study’s external data monitoring committee (DMC). The DMC said that the trial had “virtually no chance of finding a positive clinical effect.” Following a recent interim safety analysis, the committee did, however, recommend that the second Phase III verubecestat clinical study APECS should continue. APECS is evaluating verubece
  • BI and Weill Cornell Partner to Find New Treatment Approaches for COPD

    Boehringer Ingelheim (BI) and Weill Cornell Medicine are pooling their respective expertise in respiratory diseases, drug discovery, and development to identify new approaches for treating chronic obstructive pulmonary disease (COPD). The 3-year partnership will combine BI’s capabilities in respiratory disease drug discovery and development, with the knowledge and expertise of Weill Cornell Medicine’s Department of Genetic Medicine in chronic airway diseases and evaluating new approa
  • Panel Foresees Changes at FDA, Action on Drug Prices and Obamacare

    NEW YORK—Donald Trump's administration will redirect the FDA, tackle the price of prescription drugs, and repeal, if not immediately replace, the Affordable Care Act, a panel of biopharma leaders, advocates, and observers predicted yesterday.How dramatically the FDA changes direction under Trump will have consequences beyond the agency, affecting biopharma industry growth and investment in early-stage companies, one panelist warned.Jonathan Leff, a partner with Deerfield Management, a heal
  • Improved Analysis of Cancer Gene Copy Number

    Human ovarian cancer cells are highly sensitive to challenge with autophagy-targeting agents. Tumor cells with impacted autophagy are marked by accumulation of the yellow gene marker, which disappears as the cells die. [UCSD]
  • Half Year Result 20171

    thumbnailCSL Limited (ASX:CSL; USOTC:CSLLY) today announced a net profit after tax (NPAT) of $806 million for the six months ended 31 December 2016, up $87 million or 12% on a reported basis when compared to the prior comparable period (PCP).
  • BIO CEO & Investor Conference Succeeds in Bringing Innovators to Investors and Vice Versa

    The 19th Annual BIO CEO & Investor Conference came to a close February 14, having drawn more than 1,225 attendees including nearly 500 investors. During a busy two days, over 2,650 partnering meetings took place arranged through BIO’s One-on-One Partnering™—a proprietary system that enables participants to move efficiently from prospective partner identification to discussion and negotiation. Held at the Waldorf Astoria in New York, the event is one of the largest investor
  • Beyond the IPO: Reverse Mergers to Tap Public Market Capital

    On the opening day of the 2017 BIO CEO & Investor Conference in New York City, conference attendees had a chance to hear a panel discuss an alternative to the traditional IPO approach for emerging biotechnology companies looking to tap into public market capital: the reverse merger.
    IPOs and Beyond: Tapping into the Public Market’s Capital was moderated by Stephen B. Thau, a partner with Morrison & Foerster LLP, and panelists included:
    James Cappuccio, Managing Director, Investmen
  • Good Public Policy Key to Stable Biotech Investment Climate

    It is an important and exciting time for the biotechnology industry. Science is galloping forward and investment continues to flow to promising programs across the country. The one area that can slow this growth and progress is uncertainly surrounding public policy and the potential for short-sighted laws and regulations that could stifle investment and slow the pace of innovation.  In recent months, we have seen the adverse impact of headline pressures on the industry, with social media a
  • Ticks’ Tricks for Skipping Tick-Borne Sicks

    Ticks have attained notoriety for their transmission of Lyme disease, but they have kept secret their ability to survive the dread microbes they carry. Ticks, it turns out, have a distinct immune system, features of which may prove to be subject to manipulation. If the tick immune system were to be strengthened, ticks could become less hospitable to microbes that cause diseases in humans. Also, if the tick immune system were better understood, it might be possible to develop translational approa
  • Natural Antibiotic Synthesis Mechanisms Uncovered

    Understanding the molecular mechanisms that trigger the production of antibiotic compounds, especially through the regulation of gene expression, could help scientists advance the search for new natural antimicrobial agents. Now, a group of investigators, led by researchers at Texas A&M AgriLife Research, has discovered a new protein that could prove invaluable as many researchers continue their hunt for new natural products.    The recent findings deal with how proteins regul
  • Ionis Earns $75M Milestone from Bayer for Progress of Antisense Drug Program

    Ionis Pharmaceuticals will receive a $75 development milestone payment from Bayer, relating to the continued clinical development of the antisense drug, IONIS-FXI Rx , and the start of a clinical program for a second antisense candidate, IONIS-FXI-L Rx . Ionis says it plans to start a Phase IIb study with IONIS-FXI RX in patients with end-stage renal disease who are on hemodialysis. "We recently completed a Phase II study in patients with end-stage renal disease on hemodialysis, in which IONIS-F
  • Novel Technique Developed to Attack Solid Tumors

    Immunotherapies have revolutionized cancer treatment, offering hope to those whose malignancies have stubbornly survived other existing treatments. Yet solid tumor cancers are often resistant to these approaches.New findings from a University of Pennsylvania-led team untangle one of the ways tumors evade immune detection and show how immunotherapies can be modified to tackle even these solid tumors.The focus of the study was the protein type I interferon receptor IFNAR1, which is activated by in
  • Incyte Takes on Global Responsibility for Agenus' GITR and OX40 Antibody Programs

    Incyte and Agenus have amended an existing license, development and commercialization agreement for Agenus’s GITR and OX40 antibody programs, to give Incyte responsibility for funding global development and commercialization of resulting antibody candidates. The amended deal converts the agreements from  development co-funding and profit sharing, to royalty generating, with Incyte paying Agenus 15% royalties on global net sales for each approved product.The firms’ agreement for
  • Vifor, ChemoCentryx Expand Deal for Late-Stage Kidney Disease Therapy

    Vifor Pharma and ChemoCentryx have expanded their existing commercialization agreement for  ChemoCentryx's kidney diseases candidate, avacopan (CCX168), to give Vifor rights to the drug in Asia, including Japan and the Middle East. The updated deal means that Vifor now has global rights to avacopan outside of the U.S. and China.Under terms of the expanded avacopan deal ChemoCentryx retains rights to avacopan in the U.S. and China. Vifor will pay ChemoCentryx $20 million up front, which
  • Aviragen Antiviral Drug Vapendavir Fails Phase IIb Study in Asthma Patients

    Aviragen Therapeutics reported its second Phase II trial disappointment in as many weeks. The firm said that the Phase IIb SPIRITUS study evaluating the oral capsid-binding antiviral drug, vapendavir, in asthma patients with rhinovirus (RV) infection, failed to meet its primary endpoint. At the start of this month Aviragen reported on topline Phase IIa data indicating that a second clinical candidate, BTA585, failed to reduce viral load in adults challenged intranasally with syncytial virus (RSV
  • Fireside Chat with Dr. Kim of Inovio Pharmaceuticals: Immunotherapies and Vaccines

    The first Fireside Chat at the 2017 BIO CEO & Investor Conference was with President and CEO of Inovio Pharmaceuticals, J. Joseph Kim, Ph.D. Moderating the discussion was BIO’s own Phyllis Arthur, Managing Director of Infectious Diseases and Diagnostics Policy.
    Dr. Kim’s impressive background must be noted! It includes an MIT undergraduate degree, a Ph.D. in biochemical engineering from the University of Pennsylvania, and an MBA from The Wharton School. He is an immunologist by

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