Volume 13.05 | Feb 13

Issue 13.05, February 13, 2012
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TOP STORY
Researchers Develop Gene Therapy to Boost Brain Repair for Demyelinating Diseases
Scientists believe they have found a way to help the brain replace damaged oligodendrocytes and myelin. According to the researchers, leukemia inhibitory factor enables remyelination by stimulating oligodendrocyte progenitor cells to proliferate and make new oligodendrocytes. [Press release from the California Institute of Technology discussing online prepublication in the Journal of Neuroscience] Press Release | Abstract

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PUBLICATIONS (Ranked by Impact Factor of the Journal)

Infection-Responsive Expansion of the Hematopoietic Stem and Progenitor Cell Compartment in Zebrafish Is Dependent upon Inducible Nitric Oxide
Using a zebrafish model of demand-driven granulopoiesis to explore the hematopoietic stem and progenitor cell (HSPC) response to infection, investigators present data supporting a model where the zebrafish ortholog of the cytokine-inducible form of nitric oxide synthase (iNOS/NOS2) Nos2a acts downstream of the transcription factor C/ebpß to control expansion of HSPCs following infection. [Cell Stem Cell] Abstract | Press Release

Genetic Correction of β-Thalassemia Patient-Specific iPS Cells and Its Use in Improving Hemoglobin Production in Irradiated SCID Mice
In the present study, researchers successfully generated induced pluripotent stem cells (iPSCs) from fibroblasts collected from a two-year-old patient who was diagnosed with a homozygous 41/42 deletion in his β-globin gene. The researchers successfully corrected this genetic mutation in the β-thalassemia iPSCs by homologous recombination. [Cell Res] Abstract

A Rat Decellularized Small Bowel Scaffold that Preserves Villus-Crypt Architecture for Intestinal Regeneration
In this study, using a detergent-enzymatic treatment, scientists optimize in rats a new protocol that creates a natural intestinal scaffold, as a base for developing functional intestinal tissue. [Biomaterials]
Abstract | Press Release

Tumor Tropism of Intravenously Injected Human Induced Pluripotent Stem Cell-Derived Neural Stem Cells and Their Gene Therapy Application in a Metastatic Breast Cancer Model
Using a dual-color whole body imaging technology, researchers demonstrated that after tail vein injection human neural stem cells displayed a robust migratory capacity outside the central nervous system in both immunodeficient and immunocompetent mice and homed in on established orthotopic 4T1 mouse mammary tumors. [Stem Cells] Abstract

Enhancing Muscle Membrane Repair by Gene Delivery of MG53 Ameliorates Muscular Dystrophy and Heart Failure in δ-Sarcoglycan-Deficient Hamsters
Here, researchers show that systemic delivery and muscle-specific overexpression of human MG53 gene by recombinant adeno-associated virus vectors enhanced membrane repair, ameliorated pathology, and improved muscle and heart functions in δ-sarcoglycan-deficient TO-2 hamsters, an animal model of muscular dystrophy and congestive heart failure. [Mol Ther] Abstract

Multiple Intravenous Administrations of Human Umbilical Cord Blood Cells Benefit in a Mouse Model of Amyotrophic Lateral Sclerosis
The aim of the present pre-clinical translation study was to evaluate the effects of multiple low dose systemic injections of mononuclear cells from human umbilical cord blood into G93A SOD1 mice at different disease stages. [PLoS One] Abstract | Press Release

Transplantation of Telencephalic Neural Progenitors Induced from Embryonic Stem Cells into Subacute Phase of Focal Cerebral Ischemia
Researchers aim to establish cell therapy for cerebral ischemia using embryonic stem cells. [Lab Invest] Abstract

Gene Silencing of IL-12 in Dendritic Cells Inhibits Autoimmune Arthritis
In this study, researchers demonstrate the therapeutic utilization of shRNA-modified dendritic cells as an antigen-specific tolerogenic vaccine strategy for autoimmune arthritis. [J Transl Med] Abstract | Press Release

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
Demonstration of safe and stable reversal of blindness after a single unilateral subretinal injection of a recombinant adeno-associated virus (AAV) carrying the RPE65 gene prompted researchers to determine whether it was possible to obtain additional benefit through a second administration of the AAV vector to the contralateral eye. [Sci Transl Med] Abstract | Press Release

Skin-Derived Fibroblasts for the Treatment of Refractory Achilles Tendinosis: Preliminary Short-Term Results
This study aimed to assess the safety and efficacy of the use of autologous skin-derived collagen-producing cells in the treatment of refractory Achilles tendinosis. [J Bone Joint Surg Am] Abstract

Parkin Controls Dopamine Utilization in Human Midbrain Dopaminergic Neurons Derived from Induced Pluripotent Stem Cells
Here investigators generate induced pluripotent stem cells from normal subjects and Parkinson’s disease patients with parkin mutations. [Nat Commun] Abstract | Press Release

Small Molecule Mesengenic Induction of Human Induced Pluripotent Stem Cells to Generate Mesenchymal Stem/Stromal Cells
Researchers devised a single-step method to direct mesengenic differentiation of human embryonic stem cells and induced pluripotent stem cells using a small molecule inhibitor. [Stem Cells Transl Med]
Abstract | Press Release

Increased Potency of Cardiac Stem Cells Compared with Bone Marrow Mesenchymal Stem Cells in Cardiac Repair
Researchers compared intramyocardial injection of human fetal cardiac-derived c-kit+ stem cells with two doses of adult mesenchymal stem cells or control (phosphate buffered saline) in nonobese diabetic/severe combined immune deficiency mice after coronary artery ligation. [Stem Cells Transl Med] Abstract | Press Release

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SCIENCE NEWS

Alnylam Scientists Discover Novel Method for Monitoring RNAi Activity in Blood Samples
Alnylam Pharmaceuticals, Inc. announced that its scientists have discovered a novel method for monitoring RNAi activity in blood samples, representing a potential major advance for future development of RNAi therapeutics, other therapeutic modalities, and diagnostics. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the Keystone Symposia on Molecular and Cellular Biology: Gene Silencing by Small RNAs, Vancouver] Press Release

Results from Phase I Clinical Trials Demonstrate Positive Safety Data of AVI BioPharma’s RNA-Based Therapeutics for Treatment of Ebola and Marburg Viruses
AVI BioPharma, Inc. announced positive safety results from all six dose cohorts in the single ascending dose studies of AVI-6002 and AVI-6003, AVI’s lead drug candidates being evaluated for the treatment of Ebola virus and Marburg virus, respectively. [AVI BioPharma, Inc.] Press Release

Bone Marrow Cell Therapy for Heart Attack: Phase II Study Starting
Doctors at Emory University School of Medicine are testing whether bone marrow cells can help heal patients’ hearts after a heart attack. The trial is the next phase of a clinical study whose first phase began in 2006. [Emory University] Press Release

TiGenix Rheumatoid Arthritis Phase IIa Study Opens Third and Last Cohort
TiGenix announced that upon review of the safety data of the first three patients of the second cohort of the company’s Phase IIa clinical trial in rheumatoid arthritis it has received the go-ahead from the independent Safety Monitoring Board to recruit and dose the remaining 20 patients of this cohort and to open the third and final cohort. [TiGenix] Press Release

Tekmira Announces Initiation of TKM-Ebola Phase I Clinical Trial
Tekmira Pharmaceuticals Corporation announced the commencement of patient enrollment in its Phase I clinical trial for TKM-Ebola. [Tekmira Pharmaceuticals Corp.] Press Release

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POLICY

Administration Announces New Steps to Fight Alzheimer’s Disease
The Obama Administration announced new efforts to fight Alzheimer’s disease, including immediately making an additional $50 million available for cutting-edge Alzheimer’s research. [United States Department of Health and Human Services, United States] Press Release

New Governance Structure Approved by AMP Membership
The members of the Association for Molecular Pathology (AMP) voted in favor of adopting significant changes to the structure of its governing body. [Association for Molecular Pathology, United States] Press Release

Lawmakers Reintroduce Public Access Bill
Lawmakers introduced a proposal to make scientific papers funded with taxpayer money available for free on the Internet. [The Federal Research Public Access Act, United States] Press Release

Duplicate-Grant Case Puts Funders Under Pressure
Critics call for tighter checks to stop researchers being funded twice for the same work. [National Science Foundation, United States] Press Release

Europe Experiments with Joined-Up Research
In an unprecedented effort to break through the geopolitical barriers to collaboration, European researchers launched a joint strategy to prioritize and coordinate research on neurodegenerative diseases such as Alzheimer’s and Parkinson’s. [Joint Programme in Neurodegenerative Disease Research, European Union]
Press Release

World Vaccine Cell Culture 2012



BUSINESS

Sistemic to Work with TiGenix on Landmark €5.9 Million, FP7 European Project Aiming at Developing an Innovative Rheumatoid Arthritis Cell Therapy Treatment Product
Sistemic announced that its proprietary technology (SistemQC™) has been chosen to form a key part of the research and production process for a landmark multinational collaborative project led by TiGenix, Belgium and including partners from Spain, France, the Netherlands as well as the UK and Belgium. The project, REGENER-AR, will bring the first stem cell therapy treatment product for rheumatoid arthritis, Cx611, through final research and clinical development. [Sistemic Scotland Limited] Press Release

The Gamida Cell-Teva Joint Venture Concludes Enrollment for the Phase III Study of StemEx®, a Cord Blood Stem Cell Product, for Leukemia and Lymphoma
Gamida Cell announced that the Gamida Cell-Teva Joint Venture, equally held by Gamida Cell and Teva Pharmaceutical Industries, has enrolled the last of 100 patients in the international, multi-center, pivotal registration, Phase III clinical trial of StemEx, a cell therapy product in development as an alternative therapeutic treatment for adolescents and adults, with blood cancers such as leukemia and lymphoma, who cannot find a family related, matched bone marrow donor. [Gamida Cell] Press Release

Zimmer and ISTO Technologies Announce Phase III Clinical Study to Evaluate the Effectiveness of Engineered Juvenile Cartilage to Repair Damaged Knees
Zimmer Holdings, Inc. and ISTO Technologies, Inc. announced a Phase III clinical study to evaluate DeNovo® ET Engineered Tissue Graft, an engineered cartilage implant intended to repair cartilage defects in the knee. [Zimmer Holdings, Inc.] Press Release

Cytomedix Expands Commitment to Regenerative Medicine with Acquisition of Aldagen
Cytomedix, Inc. announces the completion of the acquisition of Aldagen, Inc., a privately held biopharmaceutical company developing regenerative cell therapies based on its proprietary ALDH bright cell technology. [Cytomedix, Inc.] Press Release

Harvard Bioscience, Inc. Acquires AHN
Harvard Bioscience, Inc. announced that it has acquired the business of AHN Biotechnologie GmbH through its wholly owned Biochrom Ltd. subsidiary. [Harvard Bioscience, Inc.] Press Release

Community Tissue Services’ Capital Campaign Ends with 1.3 Million Raised
Community Tissue Services announced the end to the ‘Exceeding Expectations Saving Lives’ capital campaign for the Center for Tissue, Innovation and Research.  The campaign raised $1.3 million in unrestricted charitable contributions to support the continuing growth of the organization. [Community Tissue Services] Press Release

St. Michael’s and King Saud Receive First Joint Patent
St. Michael’s Hospital and King Saud University have received their first joint U.S. patent to use the BRCA1 gene as a therapy for cardiovascular disease. [St. Michael’s Hospital] Press Release

Tekmira Announces USPTO Issuance of Key Patents Broadly Covering LNP Formulations and Mitigation of Immune Stimulation
Tekmira Pharmaceuticals Corporation announced that the United States Patent & Trademark Office (USPTO) has recently issued two key patents expanding its intellectual property portfolio: one covers its lipid nanoparticle (LNP) delivery technology, and the other covers the chemical modification of siRNA to mitigate immune stimulation. [Tekmira Pharmaceuticals Corp.] Press Release

Intellect Neurosciences Files New Patent Applications for Immunotherapy Methods to Target Abnormal Tau Protein in Treatment of Alzheimer’s Disease
Intellect Neurosciences, Inc. announced that it has filed two United States and one international patent application regarding passive and active immunotherapy approaches targeting abnormal forms of tau protein that lead to death of nerve cells in Alzheimer’s disease. [Intellect Neuroscience, Inc.] Press Release

Silence Therapeutics Announces Board Changes and Appointment of New Chief Executive Officer
Silence Therapeutics plc announced the promotion of Tony Sedgwick PhD, FRCPath to the role of Chief Executive Officer from Chief Business Officer with immediate effect and the departure of Thomas Christély who has resigned as a Director of the Company for personal reasons but will ensure a smooth transition. [Silence Therapeutics plc] Press Release

American CryoStem Joins Alliance for Regenerative Medicine
American CryoStem Corporation announced its association with the Alliance for Regenerative Medicine. [American CryoStem] Press Release

Pluristem to Expand Research & Development of Radiation Exposure Treatment
Pluristem Therapeutics, Inc. announced that following preliminary discussions with several governmental authorities, it will expand its research and development efforts on an acute radiation exposure treatment. [Pluristem Therapeutics, Inc.] Press Release

Cell Culture World 2012



NIH

National Institute of General Medical Sciences; Notice of Closed Meeting (FR Doc No: 2012-3035)

Center For Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-3030)

National Cancer Institute; Notice of Closed Meeting (FR Doc No: 2012-2771)

National Cancer Institute; Notice of Closed Meeting (FR Doc No: 2012-2775)

Proposed Collection; Comment Request: Information Program on Clinical Trials; Maintaining a Registry and Results Databank (FR Doc No: 2012-3048)

Salary Limitation on FY2012 AHRQ Grants, Cooperative Agreements, and Contracts (NOT-HS-12-007)

Notice of Corrections for PAR-12-085 NIBIB Research Education Programs for Residents and Clinical Fellows (R25) (NOT-EB-12-002)

Request for Information (RFI): Input into the Deliberations of the Council of Councils Working Group on the Use of Chimpanzees in NIH-Supported Research (NOT-OD-12-052)

Notice of Change to Non-Allowable Costs for PA-10-071: NIH Support for Conferences and Scientific Meetings (Parent R13/U13) (NOT-OD-12-048)

Stem Cell Approaches to Developing New Therapies for Ocular Diseases (R01) (RFA-EY-12-001)



CBER

Roster of the Cellular, Tissue and Gene Therapies Advisory Committee

February 10, 2012: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Roster

February 28-29, 2012: Vaccines and Related Biological Products Advisory Committee Meeting: Meeting Materials and Briefing Documents



REGULATORY

Food and Drug Administration (United States)

FDA Issues Draft Guidance on Biosimilar Product Development
The U.S. Food and Drug Administration issued three draft guidance documents on biosimilar product development to assist industry in developing such products in the United States. Press Release

Medicines and Healthcare Regulatory Agency (United Kingdom)

MHRA Welcomes a New Director of Medical Devices
John Wilkinson OBE joins the Medicines and Healthcare products Regulatory Agency (MHRA) as Director of Medical Devices this week. Press Release



EVENTS

NEW World Vaccine Cell Culture Congress 2012
April 11-12, 2012
Washington, United States

NEW International Society for Cellular Therapy (ISCT) 2012 Annual Meeting
June 5-8, 2012
Seattle, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.



JOB OPPORTUNITIES

Research and Development Technologist, hPSC Media (STEMCELL Technologies)

Scientist – Pluripotent Stem Cells (STEMCELL Technologies)

Research Technologist, Manufacturing Sciences (STEMCELL Technologies)

Business Analyst – Product Management (STEMSOFT Software)

Senior Development Associate (Genzyme Corporation)

Scientist II, Innovation – Cell Processing Technologies (Lonza Walkersville, Inc.)

Senior Manager, Innovation – Cell Processing Technologies (Lonza Walkersville, Inc.)

Postdoctoral Fellow – Program in Children’s Regenerative Medicine (The University of Texas Medical School at Houston)

Postdoctoral Fellow and Technician Positions (North Carolina Central University)

Quality Assurance Manager, GMP (Cellular Dynamics International)


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