By Anette Breindl Science Editor Many of the scientists who are attempting to harness stem cells for tissue repair and regeneration do so via a transplantation approach. That pathway consists of finding a source of stem cells and transplanting them. The stem cells themselves might be transplanted, or they might be coaxed into the desired mature cell type or something on the pathway to it first. Either way, it is a transplantation approach. But another possibility is to identify small molecules

By Randy Osborne Staff Writer Think "rare, pediatric diseases" and you tend to think "drug cocktail that changes over time" and "almost impossible to get enough investors, given the risk vs. return, not to mention the long development period." It doesn't have to be that way, said Gene Williams, CEO of Cambridge, Mass-based DART Therapeutics Inc. A biotech firm owned by patient foundations – the Nash Avery Foundation and Charley's Fund – DART hopes to turn Duchenne's muscular dystrophy (DMD) into

By Anette Breindl Science Editor As far as the relationship between Alzheimer's disease and amyloid plaques is concerned, the mystery continues. At a recent talk at the National Institutes of Health, Harvard University's Jie Shen summed up the evidence regarding the so-called amyloid hypothesis, that is, the notion that beta-amyloid is the cause of neurodegeneration and memory loss in Alzheimer's disease. She also gave an overview of her own research, which has led her to conclude that loss-of

By Peter Winter BioWorld Insight Editor There is no doubt that the biotechnology industry is truly global and that emerging markets have become extremely important for pharmaceutical companies. That is not surprising considering the fact that countries such as China, Brazil, Russia and India will represent 30 percent of the projected total global pharmaceutical sales of about $1.2 trillion in 2016, up from 20 percent of the total of $955 billion in 2011. The data from IMS Health reinforces the

By Anette Breindl Science Editor The New York Times profiled the Greek island Ikaria recently as "the island where people forget to die" for its large population (relatively speaking) of healthy individuals who are older than 80. Delos Pharmaceuticals Inc. is named after another Greek Island, and for another reason. But the company, which is a joint project of the Buck Institute for Research on Aging and British firm Biotica Technology Ltd., also hopes that its future customers will, if perhaps

By Anette Breindl Science Editor Intellectual property (IP) is a cornerstone of the drug discovery industry. But as that industry is changing, some are questioning whether that cornerstone is starting to do more harm than good, at least the way it currently is constructed. Though it is not the only type of patent, composition of matter patents are a major kind. Most patented chemicals, though, never see the light of a pharmacy shelf, creating a mismatch between where value is realized, and where

By Nuala Moran Staff Writer LONDON – Kymab Ltd. called up two industry veterans to help with the commercialization of its new platform technology for generating monoclonal antibodies in transgenic mice, as it prepares both to out-license access to the technology and use it to start an internal drug discovery effort. David Chiswell, co-founder and former CEO of Cambridge Antibody Therapeutics plc, and Christian Itin, CEO of Cytos Biotechnology and former CEO of Micromet until its $1.2 billion

By Jennifer Boggs Managing Editor Three years after a new drug application was first submitted for leukemia drug omacetaxine mepesuccinate, during which the product moved twice into different hands via M&A transactions, the FDA granted a long-awaited nod Friday. Branded Synribo, the drug is approved for treating adults with chronic myelogenous leukemia (CML), a blood and bone marrow disease, specifically those whose disease has progressed following treatment from at least two tyrosine kinase

By Anette Breindl Science Editor Researchers reported last week that the retroviral vector that is used for gene delivery in the generation of induced pluripotent stem cells, or iPSCs, does more than just ferry its cargo into cells. It activates the innate immune system – and that activation is critical for efficient reprogramming. Senior author John Cooke said he hopes that by learning more about the process, which he and his team have termed "transflammation," transforming cells via proteins

By Nuala Moran Staff Writer LONDON – Oxford BioTherapeutics Ltd. (OBT) has landed a "transformational" deal with Menarini, in which the Italian pharma company is to invest €800 million (US$1 billion) in a portfolio of five antibody and antibody-conjugate drugs discovered by OBT. Under the collaboration, Menarini will be responsible for the manufacture and clinical development of each product, with OBT providing proprietary cancer targets, antibody generation and drug conjugation technologies

By Catherine Shaffer Staff Writer AstraZeneca plc will be Ironwood Pharmaceuticals Inc.'s development and commercialization partner for linaclotide in the vast China territory. Ironwood filed a clinical trial application with China's State Food and Drug Administration for a Phase III study of linaclotide in irritable bowel with constipation (IBS-C). The companies will share the responsibilities and profits of advancing the drug in China, with London-based AstraZeneca taking a 55 percent share of

By Nuala Moran Staff Writer LONDON – International Biotechnology Trust (IBT) reported strong returns for shareholders, driven by new drug approvals, high levels of merger and acquisition activity and the resilience of leading biotech companies in the current growth-stunting environment. The London-based investment trust saw net asset value (NAV) rise by 41.9 percent to £2.32 (US$3.70) per share in the year ended August 2012. And while – inevitably – the unquoted investments in IBT's portfolio

By Peter Winter BioWorld Insight Editor The disappointing returns on biopharmaceutical research and development, despite the availability of advanced molecular technologies and the investment of billions of dollars, is forcing companies to a re-evaluate their business plans. One idea that appears to be coming back into vogue again is the strategy of giving older drugs a makeover. Reformulating and repurposing older drugs for new uses or improved versions is more attractive than developing a drug

By Catherine Shaffer Staff Writer It was compassion that moved Yutaka Niihara to start Emmaus Medical Inc. more than 10 years ago. The hematologist and University of California Los Angeles (UCLA), professor would have been content to continue with his medical practice and teaching, had it not been his passion for research and his desire to find a treatment for the intense pain crises his sickle cell patients suffered. In the course of their academic research at UCLA, Niihara and Charles Zerez

By Randy Osborne Staff Writer Merck & Co. Inc.'s tie of the licensing knot with AiCuris GmbH & Co. brought €110 million (US$142.2 million) up front and could mean another €332.5 million in milestone payments for AiCuris, with a cytomegalovirus (CMV) candidate impressive in Phase II trials. Wuppertal, Germany-based AiCuris, a spinout from Bayer AG, earlier this year disclosed Phase II data showing that the oral quinazoline letermovir worked and proved tolerable in a trial with 133 bone marrow

By Anette Breindl Science Editor Understanding the structure of a biological molecule, whether it's a protein, DNA or RNA, is often critical for understanding its function. But for a biological molecule, unlike a bridge or a building, its structure is not an unchanging thing. Biological molecules are, in fact, somewhat akin to fidgety children – they are constantly changing their shapes, wiggling around just a bit. For a long time, such wiggling took place out of sight of the prying eyes of

By Peter Winter BioWorld Insight Editor What a difference a year makes. When biotechnology execs and investors converged on the Palace Hotel in San Francisco last year for the two-day annual BIO Investor Forum, the biotech sector had just come through a tough third quarter in terms of fundraising, with public and private financings for the industry down 48 percent compared to 2010. (See BioWorld Today, Oct. 12 and Oct. 25, 2011.) This time around, the environment surrounding the 2012 event

By Anette Breindl Science Editor Freshly minted Nobelist Shinya Yamanaka has a pretty humble explanation for his success: He wasn't any good as a surgeon. "I started my career as a surgeon," he said. "But it turns out I am not talented as a surgeon. So I decided to change my career from the clinic to the laboratory. But I still feel I am a doctor. . . . I really want to help patients. So my goal all my life has been to bring . . . stem cell technologies into the clinic." On Monday, his

By Cormac Sheridan Staff Writer BRUSSELS, Belgium – Continuing the habit of starting the day's proceedings with unexpected musical choices, the audio-visual technician in charge of the Silver Hall meeting room chose Louis Armstrong's and Ella Fitzgerald's inimitable rendition of "Let's call the whole thing off" to kick off Tuesday's sessions at the BioPartnering Future Europe meeting at the Square Brussels Meeting Centre Tuesday. The song was hardly an appropriate scene-setter for a meeting