Volume 15.46 | Nov 24

Issue 15.46 November 24, 2014
Cell Therapy News - Your Industry in an Instant
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Publications | Reviews | Science | Policy | Business | NIH | CBER | Regulatory | Events | Jobs
 
TOP STORY
Gene Therapy Provides Safe, Long-Term Relief for Patients with Severe Hemophilia B
Gene therapy has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle. Participants received one of three doses of gene therapy that used a modified adeno-associated virus 8 as the vector to deliver the genetic material for making Factor IX. [Press release from St. Jude Children’s Research Hospital discussing online prepublication in The New England Journal of Medicine] Press Release | Abstract
FREE Technical Resource: Atlas of Hematopoietic Colonies from Cord Blood. Request your copy.
 
PUBLICATIONS (Ranked by impact factor of the journal)
MicroRNA Silencing for Cancer Therapy Targeted to the Tumor Microenvironment
Scientists introduce a novel antimiR delivery platform that targets the acidic tumor microenvironment, evades systemic clearance by the liver, and facilitates cell entry via a non-endocytic pathway. [Nature]
Abstract | Press Release

Efficient Delivery of RNAi Prodrugs Containing Reversible Charge-Neutralizing Phosphotriester Backbone Modifications
Short interfering ribonucleic neutrals (siRNNs) have favorable drug-like properties, including high synthetic yields, serum stability and absence of innate immune responses. Unlike short interfering RNAs, siRNNs avidly bind serum albumin to positively influence pharmacokinetic properties. Systemic delivery of siRNNs conjugated to a hepatocyte-specific targeting domain induced extended dose-dependent in vivo RNA interference responses in mice. [Nat Biotechnol] Abstract | Press Release

SCNT-Derived ESCs with Mismatched Mitochondria Trigger an Immune Response in Allogeneic Hosts
Researchers demonstrate that allogeneic mitochondria in nuclear-transfer-derived embryonic stem cells (NT-ESCs), which are nucleus-identical to the recipient, may trigger an adaptive alloimmune response that impairs the survival of NT-ESC grafts. [Cell Stem Cell] Abstract | Graphical Abstract | Press Release

First-in-Human Phase I Study of the Liposomal RNA Interference Therapeutic Atu027 in Patients with Advanced Solid Tumors
Atu027 has previously been shown to inhibit local tumor invasion as well as lymph node and pulmonary metastasis in mouse cancer models. Investigators assessed the safety, tolerability, and pharmacokinetics of Atu027 while evaluating therapeutic effects on both primary tumors and metastatic lesions. [J Clin Oncol] Abstract

Young Capillary Vessels Rejuvenate Aged Pancreatic Islets
Scientists demonstrate that aging of pancreatic islets in mice and humans is notably associated with inflammation and fibrosis of islet blood vessels but does not affect glucose sensing and the insulin secretory capacity of islet beta cells. [Proc Natl Acad Sci USA] Abstract | Press Release

Beta-Endorphin Cell Therapy for Cancer Prevention
Scientists show that transplantation of beta-endorphin neurons into the hypothalamus suppressed carcinogens- and hormone-induced cancers in various tissues and prevented growth and metastasis of established tumors via activation of innate immune functions. [Cancer Prev Res] Abstract

AAVrh.10 Immunogenicity in Mice and Humans. Relevance of Antibody Cross-Reactivity in Human Gene Therapy
Researchers compared the humoral immune response with serotypes adeno-associated virus (AAV)rh.10 and AAV9 in mice, and AAVrh.10, AAV9 and AAV2 in 100 healthy humans. Mice, injected-intravenously, raised significantly more anti-AAV9 than anti-AAVrh.10 immunoglobulins, and sera demonstrated greater neutralizing capacity, correspondingly. [Gene Ther] Abstract

Endothelium Trans Differentiated from Wharton’s Jelly Mesenchymal Cells Promote Tissue Regeneration: Potential Role of Soluble Pro-Angiogenic Factors
Investigators evaluated whether administration of endothelial cells derived from mesenchymal stem cells isolated from Wharton’s jelly can accelerate tissue repair in vivo. [PLoS One] Full Article

Free Nature Reviews Immunology Poster: The Immune Response to HIV
 
REVIEWS
Stem Cells and Muscle Diseases: Advances in Cell Therapy Strategies
The authors describe recent advances in different types of human myogenic stem cells, with a particular emphasis on myoblasts but also on other candidate cells described so far. They provide an update of ongoing clinical trials using cell therapy strategies. [Neuropathol Appl Neurobiol] Abstract

Therapeutic Face of RNAi: In Vivo Challenges
This review provides a basic explanation to small interfering RNAs signaling pathways and their therapeutic challenges. The authors provide a comprehensive explanation to failed and successful trials and their in vivo challenges. [Expert Opin Biol Ther] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

Take the "if" out of "diff": Reduce variability when differentiating to hematopoietic cells
 
SCIENCE NEWS
Delivery of Stem Cells into Heart Muscle after Heart Attack May Enhance Cardiac Repair and Reverse Injury
Delivering stem cell factor directly into damaged heart muscle after a heart attack may help repair and regenerate injured tissue. Researchers administered stem cell factor by gene transfer shortly after inducing heart attacks in pre-clinical models directly into damaged heart tissue to test its regenerative repair response. [Press release from Icahn School of Medicine at Mount Sinai discussing research presented at the American Heart Association Scientific Sessions 2014, Chicago] Press Release

Cytori Announces Promising Results in a Feasibility Trial of Cytori Cell Therapy in Conjunction with Anterior Cruciate Ligament Repair
Cytori Therapeutics, Inc. reported positive results from a clinical study of Cytori Cell Therapy™ in anterior cruciate ligament (ACL) reconstruction of patients with ACL tears. [Press release from Cytori Therapeutics, Inc. discussing research presented at the Barcelona Knee Symposium] Press Release

Don't miss ASGCT 18th Annual Meeting!
 
POLICY
Gates Foundation to Require Immediate Free Access for Journal Articles
Breaking new ground for the open-access movement, the Bill & Melinda Gates Foundation, a major funder of global health research, plans to require that the researchers it funds publish only in immediate open-access journals. [ScienceInsider] Editorial

Proposed Rules Will Vastly Expand Trove of Clinical Trial Data Reported in U.S. Database
The amount of clinical data that drug companies must share with the public could soon vastly expand under a U.S. regulation proposed. Trial sponsors would need to report summary results for drugs and devices that are never approved – and not just for products that reach the market – under the proposal. [ScienceInsider] Editorial

From our sponsor: Learn about assays for cord blood. Download the free technical bulletin.
 
BUSINESS
Bristol-Myers Squibb and Five Prime Therapeutics Announce Exclusive Clinical Collaboration to Evaluate the Combination of Investigational Immunotherapies Opdivo (Nivolumab) and FPA008 in Six Tumor Types
Bristol-Myers Squibb Company and Five Prime Therapeutics, Inc. announced that they have entered into an exclusive clinical collaboration agreement to evaluate the safety, tolerability and preliminary efficacy of combining Opdivo (nivolumab), Bristol-Myers Squibb’s investigational programmed death-1 immune checkpoint inhibitor, with FPA008, Five Prime’s monoclonal antibody that inhibits colony stimulating factor-1 receptor. [Bristol-Myers Squibb Company] Press Release

TapImmune Announces Phase II Clinical Trials Collaboration with VGTI Florida to Advance Breast and Ovarian Cancer Vaccines
TapImmune, Inc. announced a new collaborative research agreement with The Vaccine & Gene Therapy Institute of Florida (VGTI Florida), forming a partnership to advance TapImmune’s proprietary, cancer vaccines into Phase II human clinical trials for the treatment of breast and ovarian cancers. [TapImmune, Inc.] Press Release

Transposagen Biopharmaceuticals Enters into Research Collaboration and Worldwide License Agreement with Janssen for Allogeneic Chimeric Antigen Receptor (CAR) T-Cell Therapies
Transposagen Biopharmaceuticals, Inc. announced they have entered into a research collaboration and worldwide license agreement with Janssen Biotech, Inc. to develop allogeneic CAR-T. CAR-T therapies have shown promise in early human clinical trials for the treatment of blood cancers and allogeneic CAR-Ts have the potential for use as off-the-shelf cancer treatments without the need of matching donor with recipient. [Transposagen Biopharmaceuticals, Inc.] Press Release

Theravectys Provides Update on Its Phase I/II Lentiviral Vector-Based Therapeutic Vaccine Trial
Theravectys reports promising preliminary results in the first-ever vaccination trial conducted with lentiviral vectors. The randomized, placebo-controlled trial currently enrolls 38 HIV-positive patients under highly active antiretroviral therapy in 12 clinical sites in France and Belgium and aims at comparing the safety, tolerability and immunogenicity of its therapeutic vaccine candidate at three different doses versus placebo. [Theravectys SA] Press Release

Health Canada Clears Immunovaccine to Conduct Phase II Clinical Trial of DPX-Survivac in Recurrent Lymphoma
Immunovaccine Inc. announced that it has received clearance from Health Canada to conduct a Phase II clinical study of DPX-Survivac in patients with diffuse large B cell lymphoma. [Immunovaccine Inc.] Press Release

Juno T Cell Therapy for Leukemia Granted FDA Breakthrough Therapy Designation
Juno Therapeutics announced that the FDA has granted breakthrough therapy designation to the company’s JCAR015 chimeric antigen receptor product candidate. The designation applies for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia and was filed by Juno’s collaboration partner, Memorial Sloan Kettering Cancer Center, where Phase I clinical trials are currently underway. [Juno Therapeutics Inc.]
Press Release

Cell Therapy Trial Offers New Hope to Liver Disease Patients
Researchers from the University of Edinburgh have received funding to start testing the therapy in patients within the next year. It will be the world’s first clinical trial of a new type of cell therapy to treat liver cirrhosis, a common disease where scar tissue forms in the organ as a result of long-term damage. [MRC Centre for Regenerative Medicine] Press Release

Cord Blood Registry Sponsors 2014 World Stem Cell Summit
Cord Blood Registry® announced that the company is a platinum sponsor of the 10th Annual World Stem Cell Summit, the largest global meeting of stem cell science and regenerative medicine stakeholders. [Cord Blood Registry®] Press Release

Mount Sinai Researchers Awarded Grant to Find New Stem Cell Therapies for Vision Recovery
The National Eye Institute, a division of the National Institutes of Health, has awarded researchers at the Icahn School of Medicine at Mount Sinai a five-year grant totaling $1 million that will support an effort to re-create a patients’ ocular stem cells and restore vision in those blinded by corneal disease. [The Mount Sinai Hospital (EurekAlert)] Press Release
 
NIH
Proposed Collection; 60-Day Comment Request; Application for Collaboration With the Therapeutic Development Branch (TDB), Division of Preclinical Innovation (DPI), National Center for Advancing Translational Sciences (NCATS) (FR Doc. No: 2014-27636)

National Heart, Lung, and Blood Institute; Notice of Closed Meetings (FR Doc. No: 2014-27346)
 
CBER
Changes to an Approved Application: Biological Products: Human Blood and Blood Components Intended for Transfusion or for Further Manufacture; Guidance for Industry
 
REGULATORY
Food and Drug Administration (United States)

Changes to an Approved Application: Biological Products: Human Blood and Blood Components Intended for Transfusion or for Further Manufacture; Guidance for Industry; Availability (FR Doc. No: 2014-27521)

Rare Pediatric Disease Priority Review Vouchers, Draft Guidance for Industry; Availability (FR Doc. No: 2014-27022)
 
EVENTS
NEW World Cord Blood Congress
March 14-16, 2015
Monaco, Monaco

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Mesenchymal Stem Cell (MSC) Laboratory Technician (California Cryobank)

NEW Clinical Laboratory Scientist – Umbilical Cord Blood Processing (California Cryobank)

Research Scientist – Cancer and Inflammatory Diseases (Qu Biologics Inc.)

Research Technician – Cancer and Inflammatory Diseases (Qu Biologics Inc.)

Postdoctoral Researcher – Chromatin and Epigenetic Regulation (Van Andel Research Institute)

Animal Surgery – Regenerative Medicine for Cardiac and Skeletal Muscle Disease (Duke University)

Head of Cellular and Molecular Therapies Laboratory (NHS Blood & Transplant)

Postdoctoral Fellow – Developmental, Stem Cell, and/or Cancer Biology (North Carolina State University)

Postdoctoral Fellow – Collaborative Training Program in Musculoskeletal Health Research (Western University)

Postdoctoral Position – Muscle Cell Therapy (I-Stem)

Graduate Student – Pediatric Cancers (University of Calgary)

Postdoctoral Position – Leukemic Stem Cells (BC Cancer Agency)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)


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