Cell Therapy News Volume 11.44 | Nov 15 2010

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    Cell Therapy News 11.44, November 15, 2010.
    In this issue:  Science  |  Policy  |  Business  |  NIH  |  CBER  |  Regulatory  |  Events Subscribe  

    TOP STORY  

    Specialized Blood Vessels Jumpstart and Sustain Organ Regeneration 
    Researchers showed that sinusoidal endothelial cells in the liver can be manipulated so that they prompt hepatocytes to expand and regenerate lost tissue. [Press release from Howard Hughes Medical Institute discussing online prepublication in Nature]

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    SPECIAL FEATURE

    Launching ISCT's Inaugural Commercial Development Focus Group on Peripheral Vascular Disease
    Read more on how ISCT is pursuing a greater strategic alignment with its industry members in the free ISCT Industry White Paper. [International Society for Cellular Therapy]

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    SCIENCE NEWS

    Rare Syndrome Aided by Stem Cell Gene Therapy
    Stem cell gene therapy resulted in marked clinical improvement for two patients with the rare Wiskott-Aldrich syndrome, researchers reported. [Press release from MedPage Today discussing online prepublication in the New England Journal of Medicine]
     
    Researchers Discover Key Mutation in Acute Myeloid Leukemia
    Researchers have discovered mutations in a particular gene that affects the treatment prognosis for some patients with acute myeloid leukemia. [Press release from the National Institutes of Health discussing online prepublication in the New England Journal of Medicine]
     
    Researchers Identify New Aspect of Cell Reprogramming
    An interdisciplinary group of leading geneticists and stem cell researchers has found a new genetic aspect of cell reprogramming that may ultimately help in the fine-tuning of induced pluripotent stem cells into specific cell types. [Press release from Harvard University discussing online prepublication in Nature Genetics]
     
    Modeling Autism in a Dish
    Researchers successfully used human induced pluripotent stem cells derived from patients with Rett syndrome to replicate autism in the lab and study the molecular pathogenesis of the disease. [Press release from the Salk Institute for Biological Studies discussing online prepublication in Cell]
     
    Gene Identified for Spread of Deadly Melanoma
    Researchers have identified a gene linked to the spread of eye melanoma. [Press release from Washington University School of Medicine in St. Louis discussing online prepublication in Science]
     
    Researcher Discovers Potential New Target for Therapy for Aggressive Prostate Cancer
    A monoclonal antibody targeting a well-known cell surface protein inhibited prostate cancer growth and metastasis in an aggressive form of the disease that did not respond to hormone therapy. [Press release from the University of California, Los Angeles Jonsson Comprehensive Cancer Center discussing online prepublication in Nature Medicine]
     
    Scientists Find Nerve Cell Activity Drains Stem Cell Pool in Developing Brain
    Researchers have shown that as newly formed nerve cells start firing electrical signals, this activity slows down stem cell division, emptying out the stem cell pool in favor of nerve cell formation. [Press release from The Scripps Research Institute discussing online prepublication in Neuron]
     
    Rogue Gene Hijacks Stem Cells to Jumpstart Human Cancer
    Researchers have shown that a gene called FOXM1 exploits the inherent self-renewal property of stem cells causing excessive cell proliferation. [Press release from Queen Mary, University of London discussing online prepublication in Cancer Research]

    Geron Announces Publications Demonstrating That Its Telomerase Inhibitor Drug Targets Cancer Stem Cells
    Researchers demonstrate the inhibitory effect of imetelstat on cancer stem cells from breast and pancreatic tumor cell lines in vitro and in animal models of the human disease. [Press release from Geron Corporation discussing online prepublication in Cancer Research]
     
    Scientists Uncover a Genetic Switch That Turns Immune Responses On and Off
    Scientists have identified a “cellular switch,” called eye transformer, that controls the flow of information from chemical signals outside of the cell to genes in the cell nucleus. [Press release from EurekAlert! discussing online prepublication in the FASEB Journal]
     
    Growth Factor Regenerates Tooth Supporting Structures: Results of a Large Randomized Clinical Trial
    Researchers found that the topical application of fibroblast growth factor-2 can be efficacious in the regeneration of human periodontal tissue that has been destroyed by periodontitis. [Press release from EurekAlert! discussing online prepublication in the Journal of Dental Research]
     
    Stem Cell Transplants in Mice Produce Lifelong Enhancement of Muscle Mass
    A study shows that specific types of stem cells transplanted into the leg muscles of mice prevented the loss of muscle function and mass that normally occurs with aging, a finding with potential uses in treating humans with chronic, degenerative muscle diseases. [Press release from the University of Colorado at Boulder discussing online prepublication in Science Translational Medicine]
     
    Shire Presents Positive New Data at the 60th Annual American Society of Human Genetics for Patients with Type 1 Gaucher Disease
    Shire plc presented positive new data from a Phase III clinical trial (study 039) designed to evaluate the efficacy of VPRIV® (velaglucerase alfa for injection) compared with imiglucerase in patients with type 1 Gaucher disease. [Press release from Shire plc discussing research presented at the 2010 Annual American Society of Human Genetics]

    CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

    Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
    Researchers treated two patients who had Wiskott-Aldrich (WAS) syndrome with a transfusion of autologous, genetically modified hematopoietic stem cells (HSC). They found sustained expression of WAS protein expression in HSC, lymphoid and myeloid cells, and platelets after gene therapy. [N Engl J Med]
     
    DNMT3A Mutations in Acute Myeloid Leukemia
    DNMT3A mutations are highly recurrent in patients with de novo acute myeloid leukemia with an intermediate-risk cytogenetic profile and are independently associated with a poor outcome. [N Engl J Med]
     
    Inductive Angiocrine Signals from Sinusoidal Endothelium Are Required for Liver Regeneration
    Researchers demonstrate that liver sinusoidal endothelial cells constitute a unique population of phenotypically and functionally defined VEGFR3+CD34-VEGFR2+VE-cadherin+FactorVIII+CD45- endothelial cells, which through the release of angiocrine trophogens initiate and sustain liver regeneration induced by 70% partial hepatectomy. [Nature]
     
    Large Intergenic Non-Coding RNA-RoR Modulates Reprogramming of Human Induced Pluripotent Stem Cells
    Researchers characterize the transcriptional reorganization of large intergenic non-coding RNAs (lincRNAs) that occurs upon derivation of human induced pluripotent stem cells and identify numerous lincRNAs whose expression is linked to pluripotency. [Nat Genet]
     
    A Model for Neural Development and Treatment of Rett Syndrome Using Human Induced Pluripotent Stem Cells
    Using Rett syndrome (RTT) as an autism spectrum disorders genetic model, researchers developed a culture system using induced pluripotent stem cells from RTT patients' fibroblasts. [Cell]
     
    Frequent Mutation of BAP1 in Metastasizing Uveal Melanomas
    Findings implicate loss of BAP1 in uveal melanoma metastasis and suggest that the BAP1 pathway may be a valuable therapeutic target. [Science]
     
    Monoclonal Antibody Targeting of N-Cadherin Inhibits Prostate Cancer Growth, Metastasis and Castration Resistance
    Comparing gene expression in isogenic androgen-dependent and castration-resistant prostate cancer (CRPC) xenografts, researchers found a reproducible increase in N-cadherin expression, which was also elevated in primary and metastatic tumors of individuals with CRPC. [Nat Med]

    Visual Activity Regulates Neural Progenitor Cells in Developing Xenopus CNS Through Musashi1
    While proliferation of neural progenitors predominates during early central nervous system (CNS) development, progenitor cell fate shifts toward differentiation as CNS circuits develop, suggesting that signals from developing circuits may regulate proliferation and differentiation. Researchers tested whether activity regulates neurogenesis in vivo in the developing visual system of Xenopus tadpoles. [Neuron]
     
    Induction of Human Epithelial Stem/Progenitor Expansion by FOXM1
    This study provides the first evidence that FOXM1 participates in an early oncogenic pathway that predisposes cells to tumorigenesis by expanding the stem/progenitor compartment and deregulating subsequent keratinocyte terminal differentiation. [Cancer Res]
     
    Prevention of Muscle Aging by Myofiber-Associated Satellite Cell Transplantation
    Researchers demonstrate that engraftment of myofiber-associated satellite cells, coupled with an induced muscle injury, markedly alters the environment of young adult host muscle, eliciting a near-lifelong enhancement in muscle mass, stem cell number, and force generation. [Sci Transl Med]

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    POLICY

    New Stem Cell Action Coalition Launches Campaign Supporting Embryonic Stem Cell Research
    The Genetics Policy Institute announced the launch of a new advocacy coalition of 30 organizations to foster public awareness and advance the cause of embryonic stem cell research in the United States. [Stem Cell Action Coalition, United States]

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    BUSINESS

    UCB and Plasticell Partner to Identify Compounds for Stem Cell Research and Cell Therapeutics
    UK stem cell technologies firm Plasticell is to provide UCB with services based on its CombiCultâ„¢ screening technology to help identify new indications for compounds in stem cell biology, differentiation, and, potentially, tissue regeneration. [Genetic Engineering and Biotechnology News]

    The Maryland Stem Cell Research Commission Announces Opportunity for Collaborative Research Grants with State of California Researchers
    The Maryland Technology Development Corporation, which administers the Maryland Stem Cell Research Fund, announced a pilot program with the California Institute for Regenerative Medicine (CIRM). This pilot program provides an opportunity for Maryland scientists to conduct collaborative research with the recipients of CIRM Early Translational II Grant Awards. [Maryland Stem Cell Research Commission Press Release]
     
    Clavis Pharma and Clovis Oncology Extend Partnership and Sign New $205 Million Deal for the Development and Commercialization of Anti-Cancer Agent CP-4126 in Asia and Rest of the World
    Clavis Pharma ASA and Clovis Oncology, Inc. announced a new agreement for the further development and commercialization of CP-4126 (also known as CO-101), which is currently in Phase II development in pancreatic cancer. [Clavis Pharma Press Release]
     
    Watson's Eden Biodesign and Crucell Sign Vendor Network Agreement Facilitating Access to cGMP Manufacturing Services for Vaccines and Gene Therapies Using PER.C6® Cell Line Technology
    Eden Biodesign Limited announced the execution of a non-exclusive Vendor Network Agreement with Crucell, under which Eden Biodesign has become a pre-approved authorized provider of process development and cGMP manufacturing services using Crucell’s proprietary PER.C6® cell line technology. [Eden Biodesign Limited Press Release]
     
    Jefferson Awarded Multi-Million Dollar NIH Grant to Create a Center of Excellence for Autoimmune Diseases
    The Department of Neurology at Jefferson Medical College of Thomas Jefferson University has been awarded a multi-million dollar grant from the National Institutes of Health (NIH) for the creation of a center of excellence to study autoimmune diseases from basic science research to its translation into clinical applications. [Thomas Jefferson University Hospital Press Release]
     
    Ceregene Raises $11.5 Million in Series D Financing
    Ceregene, Inc. announced that it has closed its Series D preferred financing in the amount of $11.5 million. The funds will be used to help support Ceregene’s currently enrolling Phase IIb clinical study of CERE-120 for the treatment of Parkinson’s disease. [Ceregene, Inc. Press Release]
     
    Delenex Secures Series A Financing
    Delenex Therapeutics AG has announced the closing of its Series A venture equity investment round securing CHF 13.5 million (USD 14 million, EUR 10 million). [Delenex Therapeutics Press Release]

    Intercytex Reforms as U.S. Department of Defense Funds Vavelta® Scar Therapy Trials for Injured Soldiers
    Intercytex, the regenerative medicine company, announces that it has reformed as a private enterprise and that its lead product, the cell therapy (VAVELTA®) will be evaluated by the U.S. military to help treat injured soldiers. [Intercytex Press Release]

    Boost for Regenerative Medicine Research in Ireland
    In a major boost for Regenerative Medicine research in Ireland, the Minister for Enterprise, Trade and Innovation announced funding of almost EUR10 million for the Regenerative Medicine Institute (REMEDI) at NUI Galway. [Science Foundation Ireland Press Release]
     
    Athersys Announces FDA Authorization of Phase II Clinical Trial for Inflammatory Bowel Disease with Pfizer Regenerative Medicine
    Athersys, Inc. announced that the U.S. Food and Drug Administration (FDA) has authorized commencement of a Phase II clinical trial evaluating the safety and efficacy of administration of MultiStem®, Athersys' allogeneic investigational cell therapy product, for the potential treatment of ulcerative colitis. [Athersys, Inc. Press Release]
     
    Oxford BioMedica Announces U.S. IND Approval for Novel Ocular Product in Wet AMD
    Oxford BioMedica plc announced that the U.S. Food and Drug Administration has approved its Investigational New Drug (IND) application for the Phase I/II clinical development of RetinoStat®, a novel gene-based treatment for neovascular “wet” age-related macular degeneration (AMD). [Oxford BioMedica plc Press Release]
     
    Prostate Cancer Drug Gets Lukewarm Review
    There is only “moderate’’ evidence that the newly approved prostate cancer drug Provenge helps patients, according to an analysis done for Medicare that was made public. [The New York Times]
     
    USC Opens Eli and Edythe Broad CIRM Center
    California Governor Arnold Schwarzenegger joined University of Southern California (USC) administrators, trustees, elected public officials and more than 150 invited guests in the dedication and ribbon-cutting ceremony for the Eli and Edythe Broad California Institute for Regenerative Medicine Center for Regenerative Medicine and Stem Cell Research at USC. [University of Southern California Press Release]



    NIH
     

    Federal Budget Rollback Could Slash NIH Grant Success Rates
    Funding rates for biomedical researchers could drop by half, to a historical low of 10%, if Republicans follow through on their vow to cut overall federal discretionary civilian spending to 2008 levels.
     
    New Format Change Announced for National Institutes of Health, Agency for Healthcare Research and Quality, Centers for Disease Control and Prevention, and Food and Drug Administration Funding Opportunity Announcements (NOT-OD-11-019)

    Notice of Clarification of the Project Period for Translational Scholar Awards in Pharmacogenomics and Personalized Medicine (K23) (PA-11-009) (NOT-RR-11-001)

    Notice to Extend the Expiration Date for PA-07-458, Technology and Methods Development for Genomics, Population Genomics and ELSI (R01), and PA-07-459, Feasibility Studies to Develop Technology and Methods for Genomics, Population Genomics and ELSI (R21) (NOT-HG-11-005) 

    Molecular Genetics of Drug Addiction and Related Co-Morbidities (R01) (PA-11-026)

    Office of the Director, Office of Biotechnology Activities; Notice of Meeting (FR Doc. 2010-28373)
     
    Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-27743)

    Center for Scientific Review; Amended Notice of Meeting (FR Doc. 2010-28467)
     
    National Heart, Lung, and Blood Institute; Notice of Closed Meeting (FR Doc. 2010-28069)

    Government-Owned Inventions; Availability for Licensing (FR Doc. 2010-27912)

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    CBER

    PDA/FDA Adventitious Viruses in Biologics: Detection and Mitigation Strategies Workshop
    The PDA/FDA Adventitious Viruses in Biologics: Detection and Mitigation Strategies Workshop is being organized currently as a result of recent viral contamination events. This workshop is intended to encourage modernization in industry with respect to viral detection and control measures. 
     
    FY 2009 Annual Report: Innovative Technology Advancing Public Health

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    REGULATORY

    FOOD AND DRUG ADMINISTRATION (United States)

    Majority of Drug and Biological Product Makers Meeting Postmarketing Requirements and Commitments
    Most makers of approved drug and biological products are meeting their regulatory obligations and meeting targets for postmarketing studies/clinical trials in a timely manner, according to a study released by the U.S. Food and Drug Administration.
     
    Guidance for Industry: Cellular Therapy for Cardiac Disease; Availability [Docket No. FDA-2009-D-0132]

    EUROPEAN MEDICINES AGENCY (European Union)

    European Medicines Agency and Massachusetts Institute of Technology Launch Joint Project on Regulatory Science
    The European Medicines Agency and the Massachusetts Institute of Technology’s Center for Biomedical Innovation and Center for International Studies are launching a collaborative research project with a focus on enhancing regulatory science in pharmaceuticals.

    THERAPEUTIC GOODS ADMINISTRATION (Australia)

    Drugs Designated as Orphan Drugs
    Added Afamelanotide (SCENESSE), ex vivo cultured adult human mesenchymal stem cells (PROCHYMAL) and Drug: Tobramycin (TOBI).

    Consultation: Proposed Amendments to the Therapeutic Goods Regulations 1990 to Implement the Biologicals Framework


    EVENTS (Listed by Date)

    3rd Annual Workshop on Genome Engineering
    November 16, 2010
    Seattle, United States

    22nd European Organization for Research and Treatment of Cancer (EORTC)-National Cancer Institute (NCI)-American Association for Cancer Research (AACR) Symposium: Molecular Targets and Cancer Therapeutics
    November 16-19, 2010
    Berlin, Germany

    NEW London Regenerative Medicine Network Meeting
    November 17, 2010
    London, United Kingdom

    9th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
    November 17-19, 2010
    Sonoma, United States

    8th Annual Commercial Translation of Regenerative Medicine Conference
    November 18-19, 2010
    London, United Kingdom

    Translational Excellence in Regenerative Medicine (TeRM) – Biophysical Stimuli and Mass Transport for Tissue Development
    November 18-19, 2010
    Lattrop, The Netherlands

    4th Annual Conference of the Korea Society of Gene Therapy (KSGT)
    November 19, 2010
    Seoul, Korea

    Stem Cell Network's 10th Annual Scientific Meeting
    November 22-24, 2010
    Calgary, Canada

    Berlin-Brandenburg School for Regenerative Therapies (BSRT) PhD Symposium: Stem Cells – Hopes, Fears and Realities
    November 24-25, 2010
    Manchester, United Kingdom

    American Association for Cancer Research (AACR) Tumor Immunology – Basic and Clinical Advances
    November 30-December 3, 2010
    Miami, United States

    2nd European Association for Cardio-Thoracic Surgery (EACTS) Meeting on Cardiac and Pulmonary Regeneration
    December 3-4, 2010
    Vienna, Austria

    3rd Annual BioPartnering China
    December 5-7, 2010
    Shanghai, China

    Tissue Engineering & Regenerative Medicine International Society (TERMIS) – North America (TERMIS-NA) 2010 Annual Conference
    December 5-8, 2010
    Orlando, United States

    5th Annual Stem Cell Meeting on the Mesa
    December 7, 2010
    La Jolla, United States

    Genesis 2010
    December 9, 2010
    London, United Kingdom

    The American Society for Cell Biology 50th Annual Meeting
    December 11-15, 2010
    Philadelphia, United States

    NEW Partnering for Cures
    December 14-15, 2010
    New York, United States

    Pharmacology in Stem Cells Research and Regenerative Medicine
    December 17, 2010
    London, United Kingdom

    NEW The 3rd Guangzhou International Conference on Stem Cell Biology and Regenerative Medicine
    December 17-19, 2010
    Guangzhou, China

    Functional Consequences of Structural Variation in the Genome
    January 8-13, 2011
    Steamboat Springs, United States

    NEW 4th Annual OneMedForum
    January 11-13, 2011
    San Francisco, United States

    COGEM Symposium: GM Viruses as Medicine: Panacea or Pandora's Box?
    January 20, 2011
    Amsterdam, The Netherlands

    Extracellular Matrix and Cardiovascular Remodeling
    January 23-28, 2011
    Tahoe City, United States

    5th Annual Stem Cells and Regenerative Medicine World Congress
    January 24-25, 2011
    San Diego, United States

    BIO Asia International Partnering Conference
    January 24-25, 2011
    Tokyo, Japan

    Phacilitate 7th Annual Cell & Gene Therapy Forum 2011
    January 24-26, 2011
    Washington, D.C., United States

    Phacilitate 9th Annual North American Vaccine Forum
    January 24-26, 2011
    Washington, D.C., United States

    IQPC’s 4th Biobanking Conference
    January 24-26, 2011
    Philadelphia, United States

    Keystone Symposia: Stem Cells in Development, Tissue Homeostasis and Disease
    January 30-February 4, 2011
    Santa Fe, United States

    16th Proteomics Symposium
    February 3-6, 2011
    Lorne, Australia

    36th Lorne Conference on Protein Structure and Function
    February 6-10, 2011
    Lorne, Australia

    CMC Workshop: Translating Science into Successful Regulatory Submissions
    February 7-9, 2011
    Washington, D.C., United States

    NEW Stem Cell Research Forum of India 2nd Annual Meeting
    February 8-11, 2011
    Bangalore, India

    23rd Lorne Cancer Conference
    February 10-12, 2011
    Lorne, Australia

    32nd Lorne Genome Conference
    February 13-15, 2011
    Lorne, Australia

    BMT Tandem Meetings (ASBMT/CIBMTR)
    February 17-21, 2011
    Honolulu, Hawaii

    1st Annual Clinical Meeting of the Cell Society
    February 18-19, 2011
    La Jolla, United States

    Molecular Medicine Tri-Conference: Stem Cells – Revolutionizing Regenerative Medicine and Personalized Therapy 2011
    February 23-25, 2011
    San Francisco, United States

    Molecular Medicine Tri-Conference: Inaugural Circulating Tumor Cells for Cancer Detection, Diagnosis, Prognosis and Treatment
    February 23-25, 2011
    San Francisco, United States

    American Association for Cancer Research (AACR) Targeting PI3K/mTOR Signaling in Cancer
    February 24-27, 2011
    San Francisco, United States

    9th Annual BioPartnering North America
    February 27-March 1, 2011
    Vancouver, Canada

    18th Conference on Retroviruses and Opportunistic Infections (CROI 2011)
    February 27-March 2, 2011
    Boston, United States

    6th Annual Stem Cell Summit
    March 1, 2011
    New York, United States

    American Association for Cancer Research (AACR) Stem Cells, Development, and Cancer
    March 3-6, 2011
    Vancouver, Canada

    Keystone Symposia: Stem Cells, Cancer and Metastasis
    March 6-11, 2011
    Dillon, United States

    BIO-Europe Spring 2011
    March 14-16, 2011
    Milan, Italy

    The 6th International Conference on Oncolytic Viruses as Cancer Therapeutics
    March 16-19, 2011
    Las Vegas, United States

    NEW 15th Annual Hilton Head Workshop – Regenerative Medicine, Innovations for Clinical Applications
    March 16-19, 2011
    Hilton Head Island, United States

    HIV Evolution, Genomics and Pathogenesis
    March 20-25, 2011
    Whistler, Canada

    NEW StemCONN 11
    March 22, 2011
    Farmington, United States

    United Kingdom National Stem Cell Network 2011 Annual Scientific Conference
    March 30-April 1, 2011
    York, United Kingdom

    10th Annual New England Science Symposium
    April 1, 2011
    Boston, United States

    American Association for Cancer Research (AACR) 102nd Annual Meeting 2011
    April 2-6, 2011
    Orlando, United States

    5th RNAi & miRNA World Congress
    April 26-27, 2011
    Boston, United States

    3rd Epigenetics World Congress
    April 26-27, 2011
    Boston, United States

    Inaugural Next-Gen Sequencing Congress
    April 26-27, 2011
    Boston, United States

    GTCbio 2nd International Conference on Cancer Immunotherapy & Immunomonitoring
    May 2-5, 2011
    Budapest, Hungary

    Australasian Gene Therapy Society (AGTS) 7th Meeting
    May 4-6, 2011
    Melbourne, Australia

    BioPartnering India
    May 4-6, 2011
    Bangalore, India

    2nd European Biomarkers Summit
    May 18-19, 2011
    London, United Kingdom

    Inaugural Theranostics for Personalised Medicine
    May 18-19, 2011
    London, United Kingdom

    International Society for Cellular Therapy (ISCT) 17th Annual Meeting
    May 18-21, 2011
    Rotterdam, The Netherlands

    American Society of Gene and Cell Therapy (ASGCT) 14th Annual Meeting
    May 18-21, 2011
    Seattle, United States

    13th Annual C21 BioVentures
    May 24-26, 2011
    Napa, United States

    Gordon Research Conference: Viruses & Cells
    May 29-June 3, 2011
    Lucca (Barga), Italy

    Visit our events page to stay up to date with the latest events in the cell, gene and immunotherapy community.


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