Volume 15.28 | Jul 21

Issue 15.28 July 21, 2014
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Transplanting Gene into Injured Hearts Creates Biological Pacemakers
Cardiologists developed a minimally invasive gene transplant procedure that changes unspecialized heart cells into “biological pacemaker” cells that keep the heart steadily beating. [Press release from Cedars-Sinai Heart Institute discussing online prepublication in Science Translational Medicine] Press Release | Abstract
New TeSR™-E8™ is Here For Feeder-Free Culture of Human ES Cells and iPS Cells
PUBLICATIONS (Ranked by impact factor of the journal)
Histone Chaperone ASF1A Is Required for Maintenance of Pluripotency and Cellular Reprogramming
Researchers show that ASF1A, a histone-remodeling chaperone specifically enriched in the metaphase II human oocyte, is necessary for reprogramming of human adult dermal fibroblasts into undifferentiated iPSCs. [Science] Abstract | Press Release

AAV Vectors Expressing LDLR Gain-of-Function Variants Demonstrate Increased Efficacy in Mouse Models of Familial Hypercholesterolemia
Scientists sought to create vectors expressing gain-of-function human low-density lipoprotein receptor (LDLR) variants that are resistant to degradation by human proprotein convertase subtilisin/kexin type 9 and inducible degrader of LDLR and thereby enhance hepatic LDLR protein abundance and plasma LDL cholesterol reduction. [Circ Res] Abstract

Mesenchymal Stromal Cell Infusions Improve Refractory Chronic Graft versus Host Disease through an Increase of CD5+ Regulatory B Cells Producing Interleukin 10
Mesenchymal stromal cells (MSCs) have shown promise for treating refractory chronic graft-versus-host disease (cGvHD), but the favorable effects of MSCs therapy in cGvHD are complex and not fully understood. In this prospective clinical study, 20 of 23 cGvHD patients had a complete response or partial response in a 12-month follow-up. [Leukemia] Abstract

Direct Induction of Hematoendothelial Programs in Human Pluripotent Stem Cells by Transcriptional Regulators
By screening the effect of 27 candidate factors, researchers reveal two groups of transcriptional regulators capable of inducing distinct hematopoietic programs from human pluripotent stem cells: pan-myeloid (ETV2 and GATA2) and erythro-megakaryocytic (GATA2 and TAL1). [Nat Commun] Abstract | Press Release

Disease-Regulated Local IL-10 Gene Therapy Diminishes Synovitis and Cartilage Proteoglycan Depletion in Experimental Arthritis
Investigators expressed the anti-inflammatory interleukin-10 (IL-10) gene under the control of inflammation-dependent promoters in a mouse model of arthritis. [Ann Rheum Dis] Abstract

HSV-sr39TK Positron Emission Tomography and Suicide Gene Elimination of Human Hematopoietic Stem Cells and Their Progeny in Humanized Mice
Scientists demonstrate the in vivo generation of functional effector T-cells from CD34-enriched human peripheral blood stem cells modified with a lentiviral vector designed for clinical use encoding a T-cell-receptor recognizing the cancer/testes antigen NY-ESO-1, co-expressing the PET/suicide gene sr39TK. [Cancer Res] Abstract

Activated Platelet Supernatant Can Augment the Angiogenic Potential of Human Peripheral Blood Stem Cells Mobilized from Bone Marrow by G-CSF
Investigators examined autologous ‘activating platelet supernatant’ as a priming agent for stem cells; thereby enhance their pro-angiogenic potential and efficacy of stem cell-based therapy for ischemic diseases. [J Mol Cell Cardiol] Abstract

Effects of a Novel Archaeal Tetraether-Based Colipid on the In Vivo Gene Transfer Activity of Two Cationic Amphiphiles
An original archaeal tetraether derivative was used as a colipid in combination with one or the other of two monocationic amphiphiles. Gene transfer experiments were conducted in mice with lipid/DNA complexes being administered via systemic or local delivery routes. [Mol Pharm] Abstract

A Dystrophic Muscle Broadens the Contribution and Activation of Immune Cells Reacting to rAAV Gene Transfer
Recombinant adeno-associated viral vectors (rAAVs) are used for therapeutic gene transfer in skeletal muscle, but it is unclear if immune reactivity to gene transfer and persistence of transgene are affected by pathologic conditions such as muscular dystrophy. Scientists compared dystrophic mice devoid of α-sarcoglycan with healthy mice to characterize immune cell activation and cellular populations contributing to the loss of gene-modified myofibers. [Gene Ther] Full Article

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Xenogene Vaccination in the Therapy of Cancer
Several xenogene vaccines have been advanced to early Phase I/II human clinical trials where they have shown to be safe, well tolerated and capable to induce detectable immune responses against human tumor antigens. [Exp Opin Biol Ther] Abstract

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

Directed Differentiation of Pluripotent Stem Cells - A Cell Stem Cell Poster
OncoSec Medical to Present at 2014 DNA Vaccine Conference
OncoSec Medical Inc. announced that its Chief Medical Officer, Dr. Pierce will present “Intralesional DNA Immunotherapy: Converting Anti-PD-1 Non-Responders to Responders.” [Press release from OncoSec Medical Inc. (Business Wire) discussing research to be presented at the International Society of DNA Vaccines (ISDV) 2014 DNA Vaccine Conference, San Diego] Press Release

ACell Initiates Post-Market Study of MatriStem® Pelvic Floor Matrix
ACell, Inc. announced enrollment of the first patient in a U.S. Phase IV clinical trial evaluating the safety and long-term effectiveness of ACell’s MatriStem® pelvic floor matrix device as compared to native tissue repair for the treatment of pelvic organ prolapse. [ACell, Inc.] Press Release

Accelerate Your Cell Therapy to Market - Cell Therapy Bioprocessing 2014
HIV/AIDS Field Shocked by Death of Leading Researcher in MH17 Crash
As thousands of researchers gathered to attend the 20th International AIDS Conference the usual joyous hugs of greeting between far-flung colleagues were replaced by hugs of sorrow at the loss of Dutch HIV scientist Joep Lange, a leading light in the field, and at least five others heading to the meeting who were on the Malaysian Airlines flight shot down over Ukraine. [ScienceInsider] Editorial

Cora Marrett Steps Down as NSF Deputy Director
Cora Marrett has been a jack-of-all-trades administrator at the National Science Foundation (NSF) for a good part of the past two decades, including two stints as acting director of the $7 billion agency. The 72-year-old sociologist announced she will retire next month, before the end of her term as NSF’s deputy director. [ScienceInsider] Editorial

Wellcome Trust Looks to Youth and ‘High-Risk’ Research
The United Kingdom’s largest biomedical charity is to shake up the way it forks out its funding, with an increased emphasis on ‘high-risk’ research and stronger support for less-experienced scientists. [Nature News] Editorial

Lawsuit Claims Stem Cells Unsafe
StemCells, Inc. is being sued by a whistleblower who claims that the company did not follow proper protocol in preparing treatments. Rob Williams, who was once a senior manager at the firm, alleges that the company fired him after he brought the problems to light. [The Scientist] Editorial

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A*STAR Partners with Roche to Develop New Cancer Therapeutics
A*STAR’s Bioprocessing Technology Institute has entered into an agreement with Roche to identify novel drug candidates for the detection and treatment of cancer. [Agency for Science, Technology and Research (A*STAR)] Press Release

Sernova Agrees on Terms to Gain Access to Proprietary Stem Cell Technology for the Treatment of Diabetes
Sernova Corp. announced it has agreed on key terms with the University Health Network of Toronto (UHN) to gain access to worldwide, exclusive rights to certain patent-pending technologies developed by distinguished UHN researchers for the advancement of insulin-producing stem cells for the treatment of patients with insulin-dependent diabetes. [Sernova Corp.] Press Release

Hemispherx Biopharma and Bioclones Join Forces on Novel Therapeutic Cancer Vaccine
Hemispherx Biopharma, Inc. announced a strategic alliance to develop multiple projects with Bioclones (Pty) Ltd. Pre-clinical studies will be directed towards the potential treatment of breast cancer in particular, followed by prostate cancer. [Hemispherx Biopharma, Inc.] Press Release

Imugene Engages Mymetics to Manufacture HER-Vaxx Cancer Immunotherapy
Imugene Limited has engaged Mymetics SA to exclusively manufacture and develop its cancer immunotherapy HER-Vaxx. HER-Vaxx has successfully completed a Phase I study in breast cancer and the next stage of development will be a Phase II study in gastric cancer. [Imugene Limited] Press Release

Lilly and Immunocore Enter Immunotherapy Agreement to Co-Discover and Co-Develop Novel Cancer Therapies
Eli Lilly and Company and Immunocore Limited announced they have entered into a co-discovery and co-development collaboration to research and potentially develop novel T cell-based cancer therapies. [Eli Lilly and Company] Press Release

Osiris Expands Its Grafix® Product Portfolio and Launches Additional Sizes
Osiris Therapeutics, Inc. announced the addition of several new sizes to the Grafix® product line. Grafix maintains the integrity of the extracellular matrix, growth factors, and endogenous fibroblasts, epithelial cells and mesenchymal stem cells of the native tissue. [Osiris Therapeutics, Inc.] Press Release

Silence Therapeutics Ends Completion of Recruitment for Phase IIa Trial
Silence Therapeutics plc, a leader in the discovery, development and delivery of novel RNA interference therapeutics for the treatment of serious diseases, has completed the recruitment of patients for its Phase IIa trial of Atu027 in pancreatic cancer. [Silence Therapeutics plc] Press Release

Genocea Commences Phase II Dose Optimization Trial for GEN-003 HSV-2 Immunotherapy
Genocea Biosciences, Inc. announced the start of a Phase II dose optimization trial for GEN-003, the company’s immunotherapy candidate against herpes simplex-type 2 (HSV-2). [Genocea Biosciences, Inc.] Press Release

Aduro BioTech Receives Breakthrough Therapy Designation from FDA for Innovative Pancreatic Cancer Combination Immunotherapy
Aduro BioTech, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its pancreatic cancer combination treatment that consists of its CRS-207 and GVAX Pancreas immunotherapies. [Aduro BioTech, Inc. (Business Wire)] Press Release

ViaCyte Files Investigational New Drug Application and Device Master File with FDA for Novel Cell Replacement Therapy Product Candidate Designed to Treat Patients with Type 1 Diabetes
ViaCyte, Inc. announced that it has filed an Investigational New Drug application with the United States Food and Drug Administration (FDA) seeking to initiate a Phase I/II clinical trial in patients with type 1 diabetes. [ViaCyte, Inc.] Press Release
Prospective Grant of Co-Exclusive Option License: The Development of a Single Domain Human Anti-Mesothelin Monoclonal Antibody for the Treatment of Human Cancers (FR Doc. No: 2014-16853)
Providing Submissions in Electronic Format – Postmarketing Safety Reports for Vaccines
Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; User Fee Waivers, Reductions, and Refunds for Drug and Biological Products (FR Doc. No: 2014-16709)

Confidentiality of Interim Results in Cardiovascular Outcome Safety Trials; Public Hearing; Request for Comments (FR Doc. No: 2014-16374)
NEW EMBO Conference: Stem Cells in Cancer and Regenerative Medicine
October 9-12, 2014
Heidelberg, Germany

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Technical Account Manager (GlobalStem)

NEW Product Marketing Manager (Cesca Therapeutics)

NEW Research Scientist – iPSC Biology and Genome Editing (Stem Cell Theranostics)

NEW Research Assistant – iPSC Biology and Genome Editing (Stem Cell Theranostics)

Postdoctoral Researcher – Generation of Myocardium In Situ (Van Andel Research Institute)

Postdoctoral Fellow – Genome Editing in Human Embryonic Stem Cells to Study Hereditary Cancer-Associated Mutations (UConn Health Center)

Production Manager – Cell Banking Program (Medicines and Healthcare Products Regulatory Agency)

Senior Scientist – Stem Cell (Medicines and Healthcare Products Regulatory Agency)

PhD Studentship – Effects of Aging on MSC Therapy Activity (Loughborough University)

Cell Therapy Lab Assistant Supervisor (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – AAV Vectors for Gene Therapy of Neurological and Other Disorders (University of Massachusetts Medical School)

Postdoctoral Scientist – Immune Focused In Vitro Analytics (Celentyx Ltd.)

Assistant or Associate Professor – Stem Cell Transplantation Program (Dana-Farber Cancer Institute)

Postdoctoral Fellow – Prostate Cancer Research (Henry Ford Hospital)

Postdoctoral Position – Neurotoxicity and Epigenetics (Thomas Jefferson University)

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.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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