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Search Results - Hemophilia
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  • Adimab Licenses Antibody Platform to GSK, Biogen Idec
    with Biogen Idec by licensing to the biotech giant its antibody platform for use in Biogen Idec's targeted therapeutic areas, which include multiple sclerosis and hemophilia. ...
    7-26-2013
  • Drug Candidates on Trial
    ... to assess the safety of ORMD-0801. Hemophilia A > Bayer HealthCare started to enroll ... its investigational compound BAY94-9027 for the treatment of hemophilia A in children. ...
    7-22-2013
  • Biogen Idec, Sobi Cite Promising Phase III Results for Hemophilia...
    factor VIII candidate Eloctate for hemophilia A could potentially reduce the number ... major surgeries in nine patients with hemophilia A, by showing high efficacy during ...
    7-5-2013
  • FDA Approves Baxter's Rixubis for Hemophilia B
    Making it the first new recombinant factor IX approved for hemophilia B in more than 15 years, FDA has approved Baxter International's Rixubis for use in hemophilia B patients 16 and ...
    7-1-2013
  • Gene Editing to Treat Genetic Diseases, Part II
    ... using TALEN technology could help treat other genetic diseases where a few gene mutations are responsive, such as sickle cell diseases, hemophilia, or other muscular dystrophies. ...
    6-20-2013
  • Novo Nordisk Reports Promising Phase III Results for Hemophilia...
    III trial for N9-GP (glycopegylated recombinant factor IX), for hemophilia B patients. ... a paradigm shift in the treatment of hemophilia B." Novo Nordisk said it expects the ...
    5-17-2013
  • Ipsen Enlists PeptiDream in Search for Therapeutic Peptides
    This deal appears to be potential good news for Ipsen, which recently, along with bankrupt company Inspiration Biopharmaceuticals, sold its rights to hemophilia drugs OBI-1 to ...
    4-10-2013
  • Is This Gene Therapy's Breakout Moment?
    to develop and commercialize potential hemophilia B treatments using gene therapy ... an AAV8 vector in up to 16 adults with Hemophilia B. Pharma is attracted to low-lying ...
    4-1-2013
  • A Good Day for Milestones
    Factor VIII (TheraPEG-FVIII) to treat hemophilia A. Celtic subsequently exercised its ... to 72 hours in a preclinical model of naturally occurring hemophilia A, the firms report. ...
    3-7-2013
  • Is This Gene Therapy's Breakout Moment?
    ... to develop and commercialize potential hemophilia B treatments using gene therapy ... an AAV8 vector in up to 16 adults with hemophilia B. Pharma is attracted to low-lying ...
    3-4-2013
  • Genome Editing for R&D and Therapeutics
    ... a range of monogenic diseases such as hemophilia and lysosomal storage disorders like ... An example is our program that targets hemophilia in which the liver fails to produce ...
    3-1-2013
  • Cangene Inks $300+M Deal for Bankrupt Inspiration's IB1001
    prevention of bleeding in patients with hemophilia B, and two preclinical drug candidates ... factor VIII (rpFVIII) for congenital hemophilia A with inhibitors against human FVIII ...
    2-20-2013
  • Alnylam Losses Widened by Tekmira Settlement
    ... data for several other programs, including ALN-AT3 for hemophilia, ALN-TMP for hemoglobinopathies, and ALN-AAT for liver disease associated with alpha-1 antitrypsin deficiency. ...
    2-8-2013
  • Baxter Reaps Benefits from Inspiration Bankruptcy
    porcine factor VIII (rpFVIII) for congenital hemophilia A with inhibitors against human FVIII and acquired hemophilia A from the bankrupt Inspiration Biopharmaceuticals ...
    1-24-2013
  • Glybera's Story Mirrors that of Gene Therapy
    A number of companies tried to demonstrate that AAV2 could be used successfully in patients. Avigen tried AAV2 for hemophilia, using a muscular or intravenous administration. It ...
    1-1-2013
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    Journal Articles

  • Kinetics of Adeno-Associated Virus Serotype 2 (AAV2) and AAV8...
    Yi He, Marc S. Weinberg, Matt Hirsch, Mark C. Johnson, Roland Tisch, R. Jude Samulski, Chengwen Li
    Human Gene Therapy and Part B: Methods
    Adeno-associated virus (AAV) vectors 2 and 8 have been used in clinical trials for patients with hemophilia, and data suggest that the capsid specific CD8+ T cell response has had ...
  • Gut Epithelial Cells as Targets for Gene Therapy of Hemophilia
    Jay N. Lozier, James R. Yankaskas, William Jay Ramsey, Lin Chen, Helen Berschneider, Richard A. Morgan
    Human Gene Therapy and Part B: Methods
    Gut Epithelial Cells as Targets for Gene Therapy of Hemophilia Human Gene Therapy and Part B: Methods
  • Functions of AAV-CMV-F.IX And AAV-EF1α-F.IX in Gene Therapy...
    Viroj Wiwanitkit
    Human Gene Therapy and Part B: Methods
    Functions of AAV-CMV-F.IX And AAV-EF1α-F.IX in Gene Therapy for Hemophilia B Human Gene Therapy and Part B: Methods
  • Efficient Transfection of Primary Cells in a Canine Hemophilia...
    Jay N. Lozier, Arthur R. Thompson, Ping-Chuan Hu, Marjorie Read, Kenneth M. Brinkhous, Katherine A. High, David T. Curiel
    Human Gene Therapy and Part B: Methods
    Efficient Transfection of Primary Cells in a Canine Hemophilia B Model Using Adenovirus-Polylysine-DNA Complexes Human Gene Therapy and Part B: Methods

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