Volume 16.18 | May 11

Issue 16.18 May 11, 2015
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New Stem Cell May Overcome Hurdles for Regenerative Medicine
Scientists have discovered a novel type of pluripotent stem cell–cells capable of developing into any type of tissue–whose identity is tied to their location in a developing embryo. They report using these new stem cells to develop the first reliable method for integrating human stem cells into nonviable mouse embryos in a laboratory dish in such a way that the human cells began to differentiate into early-stage tissues. [Press release from The Salk Institute for Biological Studies discussing online prepublication in Nature] Press Release | Video | Abstract
Live Webinar: New Tools for Ex Vivo Expansion of Human Hematopoietic Stem and Progenitor Cells
PUBLICATIONS (Ranked by impact factor of the journal)
Therapeutic Effects of Cell-Permeant Peptides that Activate G Proteins Downstream of Growth Factors
Investigators developed cell-permeable Gα-interacting vesicle associated protein (GIV) C terminus peptides by fusing a TAT-peptide transduction domain (TAT-PTD) to the minimal modular elements of GIV that are necessary and sufficient for activation of Gi downstream of receptor tyrosine kinases, and used them to engineer signaling networks and alter cell behavior. [Proc Natl Acad Sci USA] Abstract | Press Release

Uterine-Derived Progenitor Cells Are Immunoprivileged and Effectively Improve Cardiac Regeneration When Used for Cell Therapy
The uterus has distinct immune characteristics, and the authors showed that reparative uterine stem cells (SCs) home to the myocardium post-myocardial infarction. They identified immunoprivileged uterine SCs and assessed their effects on cardiac regeneration after allogeneic transplantation. [J Mol Cell Cardiol] Abstract

Multiscale Fabrication of Biomimetic Scaffolds for Tympanic Membrane Tissue Engineering
Researchers used a multiscale approach based on electrospinning and additive manufacturing was investigated to fabricate scaffolds, based on FDA approved copolymers, resembling the anatomic features and collagen fiber arrangement of the human tympanic membrane. [Biofabrication] Abstract | Press Release

Safety and Antitumor Effect of Oncolytic and Helper-Dependent Adenoviruses Expressing Interleukin-12 Variants in a Hamster Pancreatic Cancer Model
Different vectors and interleukin-12 modifications have been developed to avoid side effects associated with high serum levels of the cytokine, while preserving its antitumor properties. Investigators evaluated two alternative strategies using the Syrian hamster as a model for pancreatic cancer metastatic to the liver. [Gene Ther] Abstract

Deproteinized Bovine Bone in Association with GTR or EMD Procedures in Aggressive Periodontitis Patients: A One-Year Retrospective Study
Researchers retrospectively evaluated and compare two regenerative periodontal procedures in young individuals with aggressive periodontitis (AgP). Thirty-two patients aged 14-25 years were diagnosed as having AgP with multiple intrabony defects and treated by one of two regenerative modalities of periodontal therapy: guided-tissue regeneration (GTR) using deproteinized bone xenograft (DBX) particles and a resorbable membrane, or an application of enamel matrix derivatives (EMD) combined with DBX. [J Clin Periodontol] Abstract

Immunobiology of Fibrin-Based Engineered Heart Tissue
Fibrin-based engineered heart tissue was generated from neonatal Lewis (Lew) rat heart cells and transplanted onto the left ventricular surface of three different rat strains: syngeneic Lew, allogeneic Brown Norway, and immunodeficient Rowett Nude rats. [Stem Cells Transl Med] Abstract

Endoglin (CD105) Silencing Mediated by shRNA under the Control of Endothelin-1 Promoter for Targeted Gene Therapy of Melanoma
Researchers constructed a eukaryotic expression plasmid encoding the shRNA molecules against CD105 under the control of endothelin-1 promoter and evaluated its therapeutic potential both in vitro in murine B16F10-luc melanoma and SVEC4-10 endothelial cells and in vivo in mice bearing highly metastatic B16F10-luc tumors. [Mol Ther Nucleic Acids] Full Article

Tumoricidal Effects of Macrophage-Activating Immunotherapy in a Murine Model of Relapsed/Refractory Multiple Myeloma
Scientists showed potent anti-myeloma activity of macrophage-activating immunotherapy that resulted in prolongation of progression-free and overall survival in an immunocompetent, preclinically validated, transplant-based model of multi-drug resistant, relapsed/refractory myeloma. [Cancer Immunol Res] Abstract

Generation of Potent T Cell Immunotherapy for Cancer Using DAP12-Based, Multichain, Chimeric Immunoreceptors
The authors describe an alternative chimeric immunoreceptor design in which they fused a single chain variable fragment for antigen recognition to the transmembrane and cytoplasmic domains of KIR2DS2, a stimulatory killer immunoglobulin-like receptor (KIR). [Cancer Immunol Res] Abstract

Free wallchart: The Intestinal Crypt
Immune-Related Strategies Driving Immunotherapy in Breast Cancer Treatment: A Real Clinical Opportunity
The authors describe the most recently awarded immune-based therapies with a consolidated or potential implication for the treatment of solid malignancies. They focus on immune checkpoints and on the clinical potential of their abrogation, with a further overview of novel vaccine-based approaches and the most relevant immunotherapeutic techniques. [Expert Rev Anticancer Ther] Abstract

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

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3D Printed Trachea among Key Mount Sinai Research Presented
A team of researchers from Icahn School of Medicine have combined 3D printing technology with human stem cells to create the first successful 3D-printed biologic tracheal graft in an animal model. [Press release from Icahn School of Medicine at Mount Sinai discussing research presented at the 2015 American Association for Thoracic Surgery (AATS) Meeting, Seattle] Press Release

Evidence of Efficacy of Gene Therapy in Rodents Affected by a Rare Genetic Liver Disease, Crigler-Najjar Syndrome
Généthon presented work done in collaboration with an Italian and Dutch teams showing long term correction of a genetic defect causing toxic buildup of bilirubin in murine and rat models of Crigler-Najjar syndrome. [Press release from Généthon discussing research presented at the 48th Annual Meeting of the European Society for Pediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN), Amsterdam] Press Release

Team Develops Novel Gene Therapy for Achromatopsia
Researchers from the University of Pennsylvania and Michigan State University presented new preclinical data that evaluates the efficacy of a gene therapy treatment for achromatopsia, a rare inherited retinal disease that involves cone cells. The disease affects humans as well as dogs. [Press release from The University of Pennsylvania discussing research presented at the Association for Research in Vision and Ophthalmology (AVRO) Annual Meeting, Amsterdam] Press Release

13th International Cord Blood Symposium
Industry Collaborations Are Key CCRM Strategy to Drive Commercialization in Regenerative Medicine Field
In under four years, the Centre for Commercialization of Regenerative Medicine (CCRM) has established an industry consortium of 45 companies, from around the globe, including Big Pharma and Canadian startups that cover the key sectors in regenerative medicine. [Centre for Commercialization of Regenerative Medicine] Press Release

House Panel Holds Hearing on ‘Politically Driven Science’—Sans Scientists
Gohmert, the chair of the oversight and investigations subpanel of the U.S. House of Representatives’ Natural Resources Committee, held a hearing to explore “the consequences of politically driven science.” Notably absent, however, were any scientists, including those alleged to have gone astray. [ScienceInsider] Editorial

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Webinar: Neural Stem Cell Culture Systems – Neurosphere vs. Adherent Monolayer Culture.
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ISMC, E-P Therapeutics, Forever Identity and ClinImmune/Caricord Collaborate on Studies and Testing of UCB Stem Cell Survivability under Microgravity (Micro-G) Simulation
International Space Medicine Consortium (ISMC) will commence a study comparing the differentiation of umbilical cord blood (UCB) stem cell survivability under 1G and micro-G conditions at its Micro-G laboratory. [International Space Medicine Consortium] Press Release

Juno Therapeutics and Fate Therapeutics Announce Strategic Research Collaboration to Improve the Therapeutic Profile of Engineered T Cell Immunotherapies
Juno Therapeutics, Inc. and Fate Therapeutics, Inc. announced that they have executed a strategic research collaboration and license agreement to identify and utilize small molecules to modulate Juno’s genetically-engineered T cell product candidates to improve their therapeutic potential for cancer patients. [Fate Therapeutics, Inc.] Press Release

The Michael J. Fox Foundation and Prothena Partner to Accelerate Discovery of Novel Biomarkers for Parkinson’s Disease
The Michael J. Fox Foundation for Parkinson’s Research and Prothena Corporation plc announced an agreement under which they will seek to accelerate the discovery of novel biomarkers and development of assays to measure Parkinson’s disease progression. The joint initiative will focus on biomarkers that may facilitate therapeutic approaches targeting alpha-synuclein, a protein potentially involved in the onset and progression of Parkinson’s disease. [Prothena Corporation plc] Press Release

Nikon and Lonza Form Collaboration for Cell and Gene Therapy Manufacturing in Japan
Lonza and Nikon Corporation announced an exclusive collaboration in the field of cell and gene therapy manufacturing in Japan. Nikon will have access to Lonza’s quality and operating systems, facility design and ongoing consulting services for the establishment of a wholly owned Nikon cell and gene therapy contract manufacturing business. [Lonza Group Ltd.] Press Release

GSK, Fondazione Telethon and Ospedale San Raffaele Announce EU Regulatory Submission for Gene Therapy to Treat Rare Disease ADA-SCID
GSK, Fondazione Telethon and Ospedale San Raffaele announced the submission of a marketing application to the European Medicines Agency for a gene therapy to treat patients with a rare disease, adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID), for whom no suitable human leukocyte antigen-matched related stem cell donor is available. [GlaxoSmithKline plc] Press Release

Penn Medicine Researchers Receive $7.5 Million to Expand HIV Gene Therapy Work
Researchers from the Perelman School of Medicine and the Penn Center for AIDS Research have been awarded $7.5 million over five years from the National Institutes of Health to initiate a multi-project HIV study investigating a new gene therapy approach to render immune cells of HIV positive patients resistant to the virus. [Penn Medicine] Press Release

STEMCELL Technologies to Commercialize BrainPhys™ Neuronal Culture Medium for Improved Support of Neurophysiological Activity
STEMCELL Technologies Inc. has announced that it has signed a license agreement with the Salk Institute for Biological Sciences for the rights to commercialize BrainPhys™ Neuronal Medium. [STEMCELL Technologies Inc.] Press Release

Theraclone Sciences’ I-STAR™ Technology Chosen by Wellcome Trust to Identify Therapeutic Antibodies Against Ebola Virus
Theraclone Sciences, Inc. announced that it will identify potential therapeutic antibodies against the Ebola virus utilizing the company’s powerful human memory B-cell interrogation platform. [Theraclone Sciences, Inc.] Press Release

FDA Greenlights UCI Clinical Trial of Treatment for Blinding Disease
A first-of-its-kind stem cell-based treatment for retinitis pigmentosa developed at UC Irvine has received consent from the U.S. Food & Drug Administration for use in a clinical trial. [University of California, Irvine] Press Release

Bellicum Pharmaceuticals Leases Space for Pilot Manufacturing Facility
Bellicum Pharmaceuticals, Inc. announced that it has entered into a lease agreement to enable in-house cell therapy manufacturing to supply clinical trials. The additional 27,000 square feet will be located in the company’s current headquarters building adjacent to the Texas Medical Center in Houston. [Bellicum Pharmaceuticals, Inc.] Press Release
Government-Owned Inventions; Availability for Licensing (FR Doc. No: 2015-10275)
PakPlus Assay
Food and Drug Administration (United States)

Providing Regulatory Submissions in Electronic Format-Certain Human Pharmaceutical Product Applications and Related Submissions Using the Electronic Common Technical Document Specifications; Guidance for Industry; Availability (FR Doc. No: 2015-10539)

Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices; Draft Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No: 2015-10482)
NEW 13th Annual International Cord Blood Symposium (ICBS)
June 11-13, 2015
San Francisco, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW In Vivo Biologist – Novel Cell and Gene Therapies (GlaxoSmithKline)

NEW Process Facilitator (Opexa Therapeutics)

NEW Research Scientist – Occupational and Environmental Lung Disease (University of British Columbia)

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Cell Therapy Production Scientist (Paramount Recruitment)

Research Assistant – Exosome-Based Gene Therapy for Huntington’s Disease (University of Oxford)

Postdoctoral Fellow – Neural Tissue Engineering (Nanyang Technological University)

Director – Clinical Trial Operations (BioRestorative Therapies, Inc.)

Director – Cell Therapy Manufacturing (BioRestorative Therapies, Inc.)

Professor – Gene Therapy (University of Massachusetts)

Research Associate – Cell Culture Specialist (Tisch MS Research Center of New York)

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