• Early Data for bluebird bio Gene-Therapy Candidate Show Some Promise

    bluebird bio today disclosed its first data, including some promising results, from a pair of clinical studies assessing its LentiGlobin™ gene therapy candidate—including a Phase III study that followed a change in its manufacturing process.In the Phase III Northstar-2 (HGB-207) study of LentiGlobin in patients with transfusion-dependent β-thalassemia (TDT) and non-β 0 /β 0 genotypes, early interim data showed that the first 3 of 15 patients treated to date achieved hi
  • Biofilm-Busting Enzymes May Rout Hospital-Acquired Infections

       
  • Repligen Merging with Spectrum Labs in $359M Deal

    Repligen said today it plans to merge with privately-held Spectrum Laboratories, in an approximately $359 million deal that Repligen said will accelerate the growth of its filtration business and enhance its position as a leading developer of single-use and continuous-manufacturing technologies for bioprocessing.Based in Rancho Dominguez, CA, Spectrum specializes in developing bioprocess-filtration technologies; its offerings include hollow fiber (HF) tangential flow filtration (TFF) products an
  • FDA Approves CSL Behring's Self-Administered C1 Esterase Inhibitor HAE Prophylactic

    FDA approved CSL Behring’s subcutaneously administered C1 esterase inhibitor (human), Haegarda, for routine use in preventing hereditary angioedema (HAE) attacks in adolescent and adult patients. Approval marks the first for a subcutaneously C1 esterase inhibitor, which gives patients the option to self-inject at home."The approval of Haegarda provides a new treatment option for adolescents and adults with hereditary angioedema," said Peter Marks, M.D., Ph.D., director of FDA's Center for
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  • Early Data for bluebird bio Gene-Therapy Candidate Shows Some Promise

    bluebird bio today disclosed its first data, including some promising results, from a pair of clinical studies assessing its LentiGlobin™ gene therapy candidate—including a Phase III study that followed a change in its manufacturing process.In the Phase III Northstar-2 (HGB-207) study of LentiGlobin in patients with transfusion-dependent β-thalassemia (TDT) and non-β 0 /β 0 genotypes, early interim data showed that the first 3 of 15 patients treated to date achieved hi
  • FDA Clears Thermo Fisher NGS Companion Diagnostic for Three NSCLC Treatments

    FDA approved Thermo Fisher Scientific’s Oncomine™ Dx Target Test as the first next-generation sequencing (NGS)-based companion diagnostic that screens tumor samples against panels of biomarkers to identify patients who may respond to one of three different treatments for non-small cell lung cancer (NSCLC).The Oncomine Dx Target Test exploits high-throughput, parallel-sequencing technology to screen tumor samples for 23 NSCLC genes, to identify patients who may be eligible for therapy
  • Novartis' Tafinlar + Mekinist Combo Wins FDA Okay for BRAF V600E-Mutant NSCLC

    The FDA approved Novartis’ BRAF inhibitor Tafinlar ® (dabrafenib) and MEK1/2 inhibitor Mekinist ® (trametinib) as combination therapy for patients with BRAF V600E mutated metastatic non-small cell lung cancer (NSCLC). Novartis said the clearance marks the first in the U.S. for a targeted therapy specifically for metastatic NSCLC with BRAF V600E mutations.The drug combination was cleared by the EC in March for the treating patients with BRAF V600 advanced mutation-positive NSCLC.&nb
  • Whole-Genome Study Shows IGF1R Inhibitors May Help Some Osteosarcoma Patients

    Researchers report in Nature Communications (“ Recurrent mutation of IGF signalling genes and distinct patterns of genomic rearrangement in osteosarcoma ”) that a genetic sequencing study has revealed that some patients with osteosarcoma could be helped by an existing drug.The team, from the Wellcome Trust Sanger Institute, University College London Cancer Institute, and the Royal National Orthopedic Hospital NHS Trust, that 10% of patients with a genetic mutation in particular growt
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  • HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]) FDA Approval

    thumbnailCSL today announced that the U.S. Food and Drug Administration (FDA) has approved CSL Behring’s HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]), the only subcutaneous therapy indicated for routine prophylaxis to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients. 
  • Shaking Up Genome Regulation by Considering Oil/Water-Like Interactions

    In the mixed-up world of gene silencing, it’s not exactly clear why some genomic regions are hard to access. These regions, it has been suggested, may simply be too tightly packed to permit the passage of regulatory proteins needed for gene expression. Tightly packed DNA, however, doesn’t always behave as expected. For example, heterochromatin has been known to exclude small proteins while admitting large ones.Such anomalous behavior naturally attracts the attention of scientists. Ea
  • Crohn’s Disease Severity Amplified by Fungus

    Inflammatory bowel disease (IBD) affects millions worldwide and can often lead to serious intestinal disorders if left unchecked. While there has been a wave of IBD drugs that have recently come to market, the underlying molecular mechanisms for many of these disorders remains a mystery. Now, a team of researchers at Case Western Reserve University (CWRU) School of Medicine recently presented new data at the Digestive Disease Weekconference in Chicago showing that the fungus Candida tropicalis t
  • Lilly Completes $90M Expansion of San Diego Biotechnology Center

    Eli Lilly has added a new state-of-the-art laboratory and 180,000 square feet of additional working space to its Biotechnology Center in San Diego, CA. Reporting completion of the $90-million expansion, Lilly said the 145% increase in working space will help to foster collaborations with external partners, and allow closer partnerships between its biotechnology, discovery chemistry, and research technology teams. The aim is to accelerate drug discovery within therapeutic areas including immunolo
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  • Brain Protein Finding May Offer Insights into Neuro-Disease Development

    Scientists at the University of Texas Medical Branch at Galveston say they have gained a better understanding of how the developing brain ensures that connections between brain cells reach their intended destination while also being maintained during life-span. The team reported its findings (“The glycosylation pathway is required for the secretion of Slit and for the maintenance of the Slit receptor Robo on axons”) in Science Signalling.The brain contains regions that serve specific
  • Repare Raises $68M in Series A to Develop CRISPR-Based Synthetic Lethality Platform

    Versant Venture and MPM Capital co-led a $68 million Series A fundraising round to establish Repare Therapeutics, a Canada-based cancer drug company exploiting a high-throughput, CRISPR -based screening platform to identify synthetic lethality cancer targets and develop precision anticancer therapies.For the last 18 months Repare has been working in stealth mode under Versant incubation to develop its synthetic lethality platform and identify initial oncology targets. The firm says a number of p
  • CRUK Calls for New Research Proposals in Second Phase of Grand Challenge

    Cancer Research UK (CRUK) has launched the second phase of its Grand Challenge program, which awards funding to interdisciplinary research teams from around the world to help address some of the key issues in fundamental cancer research, diagnosis, drug discovery and development. In February CRUK announced the four teams, and their projects, which were selected to share in the first, £71 million tranche of funding . The charity has now thrown down the gauntlet to researchers worldwide to g
  • Cell-Based Flu Vaccine Hits Commercial Scale

    Rather than rely on poultry and the eggs they produce for the manufacture of yearly flu vaccines, the World Health Organization (WHO) and FDA have encouraged vaccine manufacturers to look to other manufacturing technologies, such as use of a cell-based production model. Seqirus announced on June 21, 2017 that it has successfully used such a model for the large-scale production of the flu vaccine using a cell-derived H3N2 candidate vaccine virus that was originally procured from the National Infl
  • Sweden Launches Initiative to Establish Center for Cell and Gene Therapy Research

    Sweden aims to establish a new Centre for Advanced Medical Products (CAMP) as part of a SEK 320 million ($36.6 million), 8-year Swedish governmental initiative to position the country as a leading biologics developer.Swedish regenerative medicine firm Xintela, has been appointed a partner in the 6-year project to establish the CAMP cell and gene therapy research centre, with SEK 48 million ($5.5 million) in funding from the country’s innovation agency and research council, Vinnova and Vete
  • AUS – COLLABORATION, COMMUNICATION KEY TO AG SCIENCE FUTURE

    Better collaboration, communication the key to Australia’s agricultural science future
    20 June 2017. Source: ABC News
    Australia’s agricultural science sector needs to get better at collaboration and communication, if it is going to help farmers meet the big challenges of the next decade and beyond.
    That message is at the heart of the Australian Academy of Science’s strategic plan for the next decade of agricultural science…
    As well, agricultural science developments arou
  • USA – GENE TECH BOOSTS PLANT IMMUNITY

    Genetic engineering boosts immunity against crop disease
    12 June 2017. Source: Daily Nation
    The chemicals that farmers spray on their crops in form of pesticides to kill pests and prevent diseases have always been a bone of contention, with researchers trying to find safer alternatives. A new variety of rice that fights multiple pathogens with no effect on the yield of the crop, is thus a welcome relief for both farmers and scientists.
    The discovery is based on a study of the plant’s immun
  • CHINA – TWO NEW GM CROPS APPROVED FOR IMPORT

    China approves two new GMO crop varieties for import, renews 14 – ag ministry
    14 June 2017. Source: UK Reuters
    China approved two new varieties of genetically modified (GMO) crops for import from June 12, after the world’s top buyer of GMO soybeans pledged to speed up a review of biotech products as part of a recent trade deal with the United States.
    The approvals of new GMO imports follow an agreement on protocols for shipments of U.S. beef to China that was also promised under the
  • BRAZIL – COMMERCIAL APPROVAL FOR GM SUGARCANE

    New GMO crop: Brazil approves insect resistant sugarcane for commercial use
    09 June 2017 – Source: Genetic Literacy Project
    Brazilian biosecurity agency CTNBio on Thursday [June 8] approved commercial use of a genetically modified sugarcane, setting a milestone for Brazil’s highly competitive sugar industry which accounts for about 50 percent of the global trade.
    This is the first time in the world that genetically modified sugarcane was approved for commercial use.
  • USA – HARMONISE CONFLICTING REGULATIONS

    Harmonise conflicting regulations for genetically engineered plants and animals
    09 June 2017. Source: Nature
    In January this year, two US agencies proposed the first substantial overhaul in 30 years of how they regulate genetically altered crops and livestock. Some plant scientists expressed relief. Some animal researchers used more colourful language.
    The proposals — one to govern plants, the other to govern animals — came to wildly different conclusions. The US Department of Agricu
  • UK – NEW REPORT SHOWS GM BENEFITS

    Economic and environmental benefits of GM crops
    06 June 2017. Source: Get Farming A new report released in the UK has found that crop biotechnology has significantly reduced agriculture’s environmental impact and stimulated economic growth in Australia and 25 other countries around the world.
    The independent UK-based PG Economics report has found that over the last 20 years innovative agriculture has contributed to preserving the earth’s natural resources while allowing farmers
  • Panel Takes Look at Trump’s Trade Policy

    Day three of the BIO International Convention brought some heavy hitters to the stage. From Ann Romney’s remarkable and courageous advocacy for patients to many of our industry’s leading innovators discussing the complex ecosystem surrounding drug pricing.
    Another remarkable panel took a deep dive on the effect of Trump’s America First rhetoric and actions and how it’s affecting the global biotechnology industry.
    Not surprisingly, the North American Free Trade A
  • #BIO2017 Fireside Chat: Unlocking the Power of the Genome

    To Francis deSouza, genomics is a transformational force. deSouza, President and CEO of Illumina, joined the 2017 BIO International Convention for a Fireside Chat today for a discussion on the role genomics is playing in treating disease.
    Illumina, based in San Diego, is the largest genomics company in the world. deSouza has served as President and CEO since 2016 and has been with the company since 2013. He was previously with Symantec Corporation, where he was responsible for driving the vision
  • Invest in Diversity to Drive Innovation

    “We’re just looking for someone with the right ‘fit.’”  How many times have you heard that sentence in a hiring conversation?  Probably plenty, but the term is rarely defined – so it ends up functioning as a justification for hiring someone who makes the existing team comfortable.  So who is really surprised when the majority of your employees, especially at the senior level, are cisgender, straight, white men who all went to the same colleges a
  • U.S. Department of Defense Officials Introduce New Strategies to Stimulate Medical Countermeasures Development

    For the past several years, BIO has had the opportunity to partner with the U.S. Department of Defense (DoD) during the annual BIO International Convention to provide an open forum to discuss the challenges associated with developing medical countermeasures.
    When we say medical countermeasures, or MCMs, we are referring to vaccines, therapeutics, and diagnostics designed to protect and treat against chemical, biological, radiological, and nuclear (CBRN) threats such as anthrax and smallpox. 
  • Start-Up CEO: Making the Choices to Develop a Novel Medicine

    The choices surrounding research investment and drug development are daunting for even the most experienced and successful biopharma executives. CEOs and other company leaders must consider a number of complex factors and market data to decide which therapeutic assets warrant investments, and their choices can drastically alter the future of a company.
    An interactive panel at BIO 2017 provided attendees with a first-person perspective into how biopharma CEOs weight these factors. The panel of in
  • On Drug Costs, “We All have Accountability and Responsibility”

    The BIO 2017 convention is well underway, highlighting the innovative breakthroughs taking place across the biotechnology industry. This annual gathering of the world’s biotech leaders is also taking a close look at how they can work to ensure breakthrough treatments are accessible and affordable to patients.
    This was the subject of an engaging dialogue that took place during a session, entitled, “Our Common Goal: Ensuring Access and Affordability of Innovative Medicines.” Mode
  • Parkinson’s and Autoimmunity Strongly Linked in New Study

    Much of the etiology of Parkinson’s disease (PD) was thought to be well established, with the noted exception of the hypothesis that has been kicked around for almost a century that PD is partly an autoimmune disorder. Unfortunately, previous studies were unable to directly link the immune system to the neurodegenerative disease.However, a group of scientists led by investigators at Columbia University Medical Center (CUMC) and the La Jolla Institute for Allergy and Immunology has found di
  • New Evidence Linking Parkinson’s and Autoimmunity

    Much of the etiology of Parkinson’s disease (PD) was thought to be well established, with the noted exception of the hypothesis that has been kicked around for almost a century that PD is partly an autoimmune disorder. Unfortunately, previous studies were unable to directly link the immune system to the neurodegenerative disease.However, a group of scientists led by investigators at Columbia University Medical Center (CUMC) and the La Jolla Institute for Allergy and Immunology has found di
  • Making the Case for Ag Innovation

    Modern ag is multi-faceted, innovative, and above all, critical for feeding a growing population. At BIO’s 2017 International Convention, the Modern Ag Innovation track on June 20 featured industry leaders and academia who delved into the innovative tactics being used to improve agriculture both in the United States and around the world.
    Paul Wenger, president of the California Farm Bureau Federation, kicked off the program with a keynote presentation that touched on some of the current is
  • Skin Microbiome Can Be Negatively Altered by Topical Antibiotics

    Scientists at the Perelman School of Medicine at the University of Pennsylvania say the use of topical antibiotics can dramatically alter communities of bacteria that live on the skin, while the use of antiseptics has a much smaller, less durable impact. The study ("Topical antimicrobial treatments can elicit shifts to resident skin bacterial communities and reduce colonization by  Staphylococcus aureus  competitors"), published in  Antimicrobial Agents and Chemothe
  • Omnigenics Says All Genes Involved in Complex Traits

    The one gene-one trait idea was succeeded long ago by the polygenic model, the idea that many traits, such as height, are complex and controlled by many genes. Now, as bewildering findings accumulate from genome-wide association studies, the polygenic model is starting to show the strain.For complex traits, scientists at Stanford University have noticed, association signals tend to be spread across most of the genome. For example, signals associated with the propensity for a particular disease m
  • Anti-Alzheimer’s Compounds Found in Medicinal Plant Via New Drug Discovery Strategy

    Traditional medicinal plants have been valued over the years for providing insight into an array of diseases. However isolating specific efficacious molecules from the milieu of compounds that constitute most plant species can be a daunting task. Now, researchers at the University of Toyama, Japan have developed a method to isolate and identify active compounds in plant medicines.New data—published recently in Frontiers in Pharmacology in an article entitled, “ A Systematic Stra
  • Rubius Therapeutics Closes $120M Financing, Plans Trials Next Year

    Rubius Therapeutics said today it has raised $120 million in a “highly” oversubscribed private financing to support its development of red-cell therapeutics for multiple therapeutic areas, with plans to launch clinical trials in humans next year.Rubius said it will use proceeds of the financing toward accelerating its Red-Cell Therapeutics™ (RCT™) product portfolio, expanding its management team, and advancing its red-cell therapeutics into the clinic. The company has dev
  • Janssen, Adaptive to Develop clonoSEQ for Measuring MRD in Darzalex-Treated MM

    Janssen Biotech and Adaptive Biotechnologies established a collaboration to develop the latter’s next-generation sequencing (NGS)-based clonoSEQ® Assay for measuring minimal residual disease (MRD) in multiple myeloma (MM) patients treated using Janssen’s Darzalex (daratumumab). Darzalex is a CD38-targeting antibody approved for treating relapsed or refractory MM. Janssen will pay Adaptive an undisclosed upfront technology access payment, plus development funding and potentia
  • Avitide Eyes 2019 Profitability with Series E Financing

    Avitide, a manufacturer and supplier of molecule-specific biopharmaceutical affinity purification solutions, said today it has raised an undisclosed amount in Series E financing.Avitide said it will use the proceeds to fund ongoing operations, with the goal of becoming profitable by 2019.The company focuses on delivering high-yield, high-purity, molecule-specific affinity resins on demand in three months or less. Avitide’s proprietary affinity separation technology is designed to enable it
  • BIO2017: Tuesday’s Exhibit Show Floor Finds

     
    With over 1,800 exhibitors at BIO2017, there is truly something for everyone.  From a puppy, to pie to paracite-free mobile labs!
    First, and most fluffy, is Baxter, the cloned puppy.  Baxter is an 18 week old toy poodle.  The original toy poodle is still alive.  Baxter is a nearly identical clone born at a later time and in a different place.  Baxter was cloned by Viagen Pets.Also on the exhibit show floor was the cleverly named “Boston Cream Pie Networking&
  • Glenmark Licenses APC's Antigen Presenting Cell-Based Anticancer Candidate

    APC Therapeutics granted Glenmark Pharmaceuticals an exclusive license to a small-molecule anticancer compound based on APC's antigen-presenting cell technology. Glenmark said the compound could potentially be used as either monotherapy or in combination with approved treatments. The firm will shoulder all clinical development responsibilities, along with regulatory and commercialization activities worldwide. Financial details of the deal were not disclosed."Glenmark is an ideal partner to advan
  • Topical Antibiotics Can Alter Skin Microbiome to Allow Growth of Pathogenic <i>S. aureus</i>

    Scientists at the Perelman School of Medicine at the University of Pennsylvania say the use of topical antibiotics can dramatically alter communities of bacteria that live on the skin, while the use of antiseptics has a much smaller, less durable impact. The study ("Topical antimicrobial treatments can elicit shifts to resident skin bacterial communities and reduce colonization by  Staphylococcus aureus  competitors"), published in  Antimicrobial Agents and Chemothe
  • Debiopharm Acquires Wee-1 Kinase Inhibitor Program from Almac Discovery

    Swiss biopharma Debiopharm International negotiated global rights to Almac Discovery’s Wee-1 kinase inhibitor program. The deal gives Debiopharm exclusive rights to Almac Discovery’s Wee-1 patent portfolio and associated IP, in return for an undisclosed upfront payment, plus future development and commercial milestone payments, and sales royalties.Almac Discovery has developed and optimized a series of oral, selective inhibitors of Wee-1 kinase, a regulator of the G2 checkpoint. Comm
  • “High Tech Enables High Touch.” Stanford’s Dean of Medicine on How Technology is Changing the Face of Healthcare

    Dean Lloyd Minor says technology, and specifically, smartphones are making healthcare deliverable in real-time. By the year 2020, tech crunch predicts smartphones are expected to reach 80% of the world’s population. He’s encouraged and yet pragmatic about this statistic. He says, “Technology will never replace one on one human interaction between patient and physician, but it will allow us to make our information available, for example, training midwives in India.” He add
  • What’s Your Bio Strategy?

    John Cumbers, founder of SynBioBeta, introduced today’s biofuels and renewable chemicals track at the 2017 BIO International Convention with the question, What’s Your Bio Strategy, which is also the title of his forthcoming book. His point is that industrial biotechnology, synthetic biology and new techniques such as CRISPR will disrupt industries in the very near future. A strategy for adopting and adapting to biotechnology is as necessary for the future as a strategy for informatio
  • Heart Disease ‘Vaccine’ Candidate Clears Way for Cholesterol Clearance

    It gives rise to antibodies that could prevent disease, but it’s not quite a vaccine. It qualifies as an immunotherapy because it targets one of the body’s own proteins, not a protein associated with a pathogen. The “it” in this case is AT04A, a peptide-based formulation that induces an immune response against a protein that interferes with cholesterol clearance.According to a recent study, AT04A lowered cholesterol in a mouse model of atherosclerosis. This result, detail
  • eFFECTOR, Pfizer, Merck KGaA Partner to Combine Avelumab with MNK1/2 Inhibitor

    eFFECTOR Therapeutics, Pfizer, and Merck KGaA established a partnership to carry out Phase II study evaluating the safety, tolerability, and efficacy of eFFECTOR’s small-molecule investigational MNK1/2 (MAP kinase-interacting kinase 1 and 2) inhibitor eFT508 combined with Merck KGaA’s and Pfizer’s programmed death-ligand 1 (PD-L1) antibody avelumab (Bavencio ® ) in patients with microsatellite-stable relapsed or refractory colorectal cancer (CRC). eFFECTOR will conduct the
  • Pamplona Capital to Acquire Parexel for $5B

    Parexel has agreed to be acquired for approximately $5 billion by Pamplona Capital Management, which plans to take the CRO private, the prospective buyer and acquisition target said today.The deal ends a turbulent two years in which Parexel has sought to contain costs following disappointing results.On May 4, Parexel revealed plans in a regulatory filing to eliminate about 6% of its workforce, “approximately 1100 to 1200 jobs,” as part of an expansion of a restructuring disclosed in
  • New Immune System Finding May Open Door to Novel Cancer Therapies

    Scientists at the Massachusetts Institute of Technology say they have identified a mechanism that the immune system uses to eliminate genetically imbalanced cells from the body. Almost immediately after gaining or losing chromosomes, cells send out signals that recruit natural killer cells, which destroy the abnormal cells.The findings raise the possibility of harnessing this system to kill cancer cells, which nearly always have too many or too few chromosomes, according to the investigators."If
  • Cell Medica Acquires Catapult Therapy for Lead WT1-TCR T Cell Therapy

    Cell Medica is buying Catapult Therapy TCR and the firm's gene-modified WT1-TCR T cell therapy candidate. The treatment is currently in Phase I/II development for the potential treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).Catapult Therapy TCR is a special purpose vehicle established by The Cell and Gene Therapy Catapult (CGT Catapult), UCL Business (UCLB) and Imperial lnnovations, to develop the WT1-TCR T cell therapy, which was originally discovered by researcher
  • Infusion, OncoSynergy Partner on Direct Delivery of mAb Therapy for Glioblastoma

    Infuseon Therapeutics and OncoSynergy are teaming up to evaluate the latter’s monoclonal pan-CD29 antibody OS2966, delivered using Infuseon’s Cleveland Multiport Catheter™ (CMC), for the potential treatment of glioblastoma. The firms’ strategic alliance aims to establish proof of concept that OS2966 can be delivered directly to the brain using the CMC, prior to considering a Phase I study for the glioblastoma indication.OncoSynergy is a University of California, San Franc

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