Monday, December 12, 2011

Gene therapy breakthrough treats hemophilia B

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December 12, 2011
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  Today's Top Story 
  • Gene therapy breakthrough treats hemophilia B
    Researchers have treated hemophilia B with gene therapy, a first for a well-known disease, according to this article. The scientists used a virus called adeno-associated virus-8 to deliver a good version of the gene for clotting agent Factor IX to six patients. A single injection allowed four of the patients to stop the usual therapy, injections of concentrated Factor IX. Researchers in Memphis, Tenn., London and Philadelphia collaborated on the project, and they plan to treat 20 more patients to determine the best dosage. The New York Times (tiered subscription model) (12/10) LinkedInFacebookTwitterEmail this Story
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  Health Care & Policy 
 
  • Study: Blood cancer drug Mylotarg has benefits for AML
    Patients with newly diagnosed acute myeloid leukemia survived for 25 months after being treated with Pfizer's blood cancer drug Mylotarg combined with chemotherapy, while those who received chemotherapy survived only 15 months, according to findings reported at the American Society of Hematology meeting. The drug, pulled from the market last year for being linked to deaths from liver complications, was previously used in patients with cancer recurrence. Pfizer said it is planning to talk with regulators about returning of the drug to the market. Bloomberg Businessweek (12/10) LinkedInFacebookTwitterEmail this Story
  • Blood platelets made from adult stem cells
    Japanese researchers have reprogrammed adult stem cells to create blood platelet cells. The platelets had the same life span as normal human platelets, researchers reported at a meeting of the American Society of Hematology. The study "has the potential of marching us forward to a day when we might be able to eliminate blood platelet shortages," said Dr. Charles Abrams of the University of Pennsylvania. Reuters (12/11) LinkedInFacebookTwitterEmail this Story
  • Partnership aims to develop molecular tumor testing protocol
    GE Healthcare company Clarient and Acorn Research will work together to develop a standardized protocol for molecular tests of tumor samples during a patient's initial cancer diagnosis. The protocol will help health care providers decide on the most effective treatment plan, according to the companies. The method also may allow biomarker data to be matched with results from clinical trials and clinical practice. MolecularImaging.net (12/9) LinkedInFacebookTwitterEmail this Story
  • Vertex, Alios start joint research on hepatitis C compounds
    Vertex Pharmaceuticals and Alios BioPharma have begun two clinical trials of Alios compounds ALS-2200 and ALS-2158 for people with hepatitis C. Under a licensing agreement signed in June, Alios will receive $60 million upfront as well as research and development dues in exchange for giving Vertex worldwide development and commercialization rights to the drug candidates. Mass High Tech (Boston) (12/9) LinkedInFacebookTwitterEmail this Story
  • Pancreas study boosts hope for diabetes stem cell treatment
    A study of pancreatic agenesis offered insights on how stem cells could be turned into insulin-producing pancreatic beta cells. In 15 out of 27 subjects with pancreatic agenesis, a defective form of the GATA6 gene was found. Researchers hope their study "will help the crucial work to try and make beta cells for patients with type 1 diabetes," one of the scientists said. Google/The Press Association (U.K.) (12/11) LinkedInFacebookTwitterEmail this Story
  • GSN works on new prenatal test for unassisted pregnancies
    Gene Security Network is developing a noninvasive prenatal test for unassisted pregnancies that promises to be almost as accurate as amniocentesis for detecting inherited genetic disorders such as Down syndrome, cystic fibrosis and Tay-Sachs disease. The test can be used in the first trimester of pregnancy, giving it a key advantage over other screening methods. "GSN's approach ... holds immense promise to achieve clinically useful results," said Dr. Ronald Wapner, the lead investigator of a clinical study of the test. GSN markets a test for babies conceived through in vitro fertilization. Bloomberg (12/9) LinkedInFacebookTwitterEmail this Story
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  Company & Financial News 
 
  • Inovio raises $4 million in public offering
    Vaccine-maker Inovio Pharmaceuticals raised $4 million in a public offering and said it would use the money for clinical research and development. The company has midstage treatment candidates for malaria, HIV and cervical cancer. MedCityNews.com (12/7) LinkedInFacebookTwitterEmail this Story
  • Novartis restructures neuroscience research
    Novartis is establishing a new group at its main research facility in Cambridge, Mass., to investigate the genetic causes of brain diseases as it restructures its neuroscience research unit. "We believe if we understand the circuitry we may have a different direction to go in, some of the more traditional directions we worked in aren't as robust for the next wave of discoveries, and there are new opportunities," said Mark Fishman, who heads the company's drug research. The firm is closing a research center in Basel, Switzerland, and suspending some brain-research projects. The Wall Street Journal (12/8) LinkedInFacebookTwitterEmail this Story
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  Food & Agriculture 
 
  • WestBred donates $25,000 for pest control
    WestBred, a unit of Monsanto, has presented the Montana Grains Foundation with a $25,000 check to help finance the group's efforts to seek better means of managing and controlling the wheat stem sawfly. WestBred developed WB Gunnison and WB Quake, two wheat varieties with sawfly-resistant properties. Ag Weekly (12/9) LinkedInFacebookTwitterEmail this Story
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  Hot Topics 

Top five news stories selected by BIO SmartBrief readers in the past week.

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  Industrial & Environmental 
  • Valero to provide funding for cellulosic-ethanol project in Mich.
    Valero Energy has agreed to supply most of the funds needed by Mascoma to develop its wood-based ethanol plant in Kinross, Mich. Mascoma aims to begin construction on the $232 million project within the next six months, with completion due before 2013, the company said. The "project is appealing to Valero because it will be the first, or one of the first, commercial-scale cellulosic ethanol plants," said spokesman Bill Day. Bloomberg (12/9) LinkedInFacebookTwitterEmail this Story
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