Volume 16.40 | Nov 2

Issue 16.40 November 2, 2015
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Immunotherapy for Pancreatic Cancer Boosts Survival More than 75 Percent in Mice
Researchers found that a certain type of immunotherapy — even when used without chemotherapy or radiation — can boost survival from the nearly-always deadly pancreatic cancer by more than 75 percent in mice. The researchers aim to launch an early-stage clinical trial testing the safety of this technology in humans with advanced pancreatic cancer within the next year. [Press release from Fred Hutchinson Cancer Research Center discussing online publication in Cancer Cell] Press Release | Abstract | Graphical Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Convergence of Acquired Mutations and Alternative Splicing of CD19 Enables Resistance to CART-19 Immunotherapy
Using genome editing, scientists demonstrated that exon 2 skipping bypasses exon 2 mutations in B-cell acute lymphoblastic leukemia cells and allows expression of the N-terminally truncated CD19 variant, which fails to trigger killing by chimeric antigen receptor–armed T cells (CART-19) but partly rescues defects associated with CD19 loss. [Cancer Discov] Abstract | Press Release

rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and CF Mice
Investigators evaluated the preclinical potential of adeno-associated virus (rAAV) gene therapy for cystic fibrosis (CF) to restore chloride and fluid secretion in two complementary models: intestinal organoids derived from CF subjects and a CF mouse model, an important milestone towards the development of a clinical rAAV candidate for CF gene therapy. [Am J Respir Crit Care Med] Abstract

Induced Pluripotent Stem Cell-Derived Natural Killer Cells for Treatment of Ovarian Cancer
Researchers evaluated the ability of natural killer (NK) cells isolated from peripheral blood and NK cells derived from induced pluripotent stem cell to mediate killing of ovarian cancer cells in a mouse xenograft model. [Stem Cells] Abstract

Nanoparticle-Mediated Target Delivery of TRAIL as Gene Therapy for Glioblastoma
To enable effective delivery of plasmid DNA encoding tumor necrosis factor α-related apoptosis-inducing ligand (TRAIL) into glioblastoma, investigators developed a targeted iron oxide nanoparticles coated with chitosan-polyethylene glycol-polyethyleneimine copolymer and chlorotoxin and evaluated its effect in delivering TRAIL in vitro and in vivo. [Adv Healthc Mater] Abstract

Partially Oxidized Polyvinyl Alcohol as a Promising Material for Tissue Engineering
Scientists fabricated 1% and 2% oxidized polyvinyl alcohol hydrogels, which were considered for the first time for tissue-engineering applications. Twelve weeks after in vivo implantation in mice, the modified hydrogels did not elicit severe inflammatory reactions, showing them to be biocompatible and to degrade faster as the degree of oxidation increased. [J Tissue Eng Regen Med] Abstract

Comparing the Osteogenic Potential of Bone Marrow and Tendon-Derived Stromal Cells to Repair a Critical-Sized Defect in the Rat Femur
Tendon-derived stromal cells were evaluated in comparison to bone marrow stromal cells to support the functional repair of a 5 mm critical-sized, segmental defect in the rat femur. [J Tissue Eng Regen Med] Abstract

Immune Responses to WT1 in Patients with AML or MDS after Chemotherapy and Allogeneic Stem Cell Transplantation
Investigators analyzed expression of Wilms’ tumor gene 1 (WT1) in 226 peripheral blood and bone marrow samples from patients with acute myeloid leukemia or myelodysplastic syndrome (AML/MDS) before and after allogeneic stem cell transplantation. [Int J Cancer] Abstract

Enhanced Angiogenesis in Ischemic Skeletal Muscle after Transplantation of Cell Sheets from Baculovirus-Transduced Adipose-Derived Stromal Cells Expressing VEGF165
Scientists report on therapeutic efficacy of cell sheets from mouse adipose-derived stromal cells transduced to express human vascular endothelial growth factor 165 a/a isoform (VEGF165), which showed potency to restore perfusion and protect tissue in a model of limb ischemia. [Stem Cell Res Ther] Full Article

Aspirin Promotes Bone Marrow Mesenchymal Stem Cell-Based Calvarial Bone Regeneration in Mini Swine
Researchers investigated whether aspirin is capable of accelerating the regenerative potential of bone marrow mesenchymal stem cells in a mini swine calvarial bone defect model. [Stem Cell Res Ther] Full Article

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Skeletal Stem Cells and Their Contribution to Skeletal Fragility: Senescence and Rejuvenation
The author discusses targeting skeletal stem cells to enhance bone formation and to abolish age-related bone fragility in the context of using stem cells for treatment of age-related disorders. [Biogerontology]
Abstract | Full Article

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Alnylam to Report New Clinical Results with Investigational RNAi Therapeutics for the Treatment of Transthyretin-Mediated Amyloidosis
Alnylam Pharmaceuticals, Inc. announced that Alnylam scientists and collaborators will present results from ongoing Phase II open-label extension studies with its investigational RNAi therapeutics in development for the treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis). [Press release from Alnylam Pharmaceuticals, Inc. discussing research to be presented at the First European Congress on Hereditary ATTR Amyloidosis, Paris] Press Release

Adaptimmune Announces Upcoming Data Presentation
Adaptimmune announced that it will present new data from trials of its NY-ESO affinity enhanced T-cell therapy in patients with synovial sarcoma and myeloma, as well as data from preclinical safety assessments of its affinity enhanced T-cell therapy directed at MAGE A-10. [Press release from Adaptimmune Therapeutics plc discussing research to be presented at the 2015 Annual Meeting of the Society of Immunotherapy for Cancer (SITC), National Harbor] Press Release

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Scientists Call for Unified Initiative to Advance Microbiome Research
A group of leading scientists representing a wide range of disciplines has formed a unified initiative to support basic research, technological development and commercial applications to better understand and thoughtfully harness capabilities of Earth’s vast systems of microorganisms. [University of Chicago] Editorial

NIH Refocuses Research into Chronic Fatigue Syndrome
In the wake of mounting criticism that researchers pay scant attention to myalgic encephalomyelitis/chronic fatigue syndrome, the U.S. National Institutes of Health (NIH) announced that it is increasing efforts to figure out what causes the baffling illness and to find treatments for it. [ScienceInsider] Editorial

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The Institut Pasteur and Chan Soon-Shiong Institute for Molecular Medicine Partner to Form Global Genomics Center
The Chan Soon-Shiong Institute for Molecular Medicine and the Institut Pasteur announced a partnership to establish The Pasteur Global Health Genomics Center (PGHGC). The aim of the PGHGC is to advance genomic research utilizing next-generation patient information systems needed to support the acquisition and usage of high-quality sequencing data. [Institut Pasteur] Press Release

BioCision and UCSF Announce Collaboration to Advance Cell Therapy for Organ Transplantation and Diabetes
BioCision, LLC, announced a collaboration with the University of California, San Francisco Transplantation Research Laboratory of Dr. Qizhi Tang, who is researching a novel transplantation immunotherapy that could potentially minimize rates of organ rejection and the toxicity of global immunosuppression; a similar strategy is being investigated for the treatment of autoimmune diseases including Type 1 diabetes. [BioCision, LLC] Press Release

With Disclosure of Recently Met Benchmarks for Its Adult Tissue Stem Cell Counting Technology, Asymmetrex Targets Applications to Enable Gene-Editing Therapies
Asymmetrex, LLC posted results on its website from the most recently completed benchmark studies for its computer simulation technology for determining the number of stem cells in complex organ and tissue cell preparations. [Asymmetrex, LLC] Press Release

First US Patent Covering Allogeneic Chimeric Antigen Receptor T Cells Modified to Reduce Immunogenicity is Awarded to Celyad
Celyad announced the issuance of United States Patent No. 9,181,527 relating to allogeneic human primary T-cells that are engineered to be T-cell receptor-deficient and express a chimeric antigen receptor. [Celyad] Press Release

New Immunotherapy Treatment May Clear Cancer-Causing HPV Infections Faster
A new therapeutic vaccine, GTL001, developed by Genticel to clear HPV strains 16 and 18 – the types most likely to cause cancer – is being evaluated for safety in a Phase I clinical trial at the University of Louisville. [University of Louisville] Press Release

Endonovo Therapeutics Files Patent Application on Cell Therapy Created Using Its Proprietary Cell-Enhancing Cytotronics Platform
Endonovo Therapeutics, Inc. announced that it has filed a patent application with the United States Patent and Trademark Office on a perivascular cell mixture created using its proprietary Cytotronics™ Platform that would allow for the wide spread use of cord blood in the treatment of malignant and non-malignant hematological disorders. [Endonovo Therapeutics, Inc.] Press Release

Successful GMP Production Process for UCART19
Cellectis announced that a series of three production runs of UCART19, its lead TALEN® gene edited product candidate, was performed, confirming the implementation of Cellectis’ manufacturing process in GMP conditions. [Cellectis] Press Release

Georgia State Adds Two Research Centers in Healthy Development, Molecular and Translational Medicine
Georgia State University has created two new research centers—the Mark Chaffin Center for Healthy Development and the Center for Molecular and Translational Medicine—dedicated to health and medicine. [Georgia State University] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-27427)
Homologous Use of Human Cells, Tissue, and Cellular and Tissue-Based Products; Draft guidance for Industry and FDA Staff
Food and Drug Administration (United States)

Liposome Drug Products: Chemistry, Manufacturing, and Controls; Human Pharmacokinetics and Bioavailability; and Labeling Documentation; Draft Guidance for Industry; Availability (FR Doc. No: 2015-27744)

Draft Guidances Relating to the Regulation of Human Cells, Tissues, or Cellular or Tissue-Based Products; Public Hearing; Request for Comments (FR Doc. No: 2015-27703)

Minimal Manipulation of Human Cells, Tissues, and Cellular and Tissue-Based Products; Draft Guidance for Industry and Food and Drug Administration Staff; Reopening of the Comment Period (FR Doc. No: 2015-27705)
NEW Cell & Gene Therapy World
January 25-27, 2016
Washington, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Scaffold Design & Drug/Gene Delivery for Neural Tissue Engineering (Nanyang Technological University)

NEW Postdoctoral Position – Stem Cell Engineering Research (Nanyang Technological University)

NEW Postdoctoral Position – hPSC-Derived Cardiomyocyte Grafts in Injured Hearts (University Health Network)

Group Leader – Translational Research (Cancer Research Center of Toulouse)

Group Leader Position – Cancer Research (Oslo University Hospital)

PhD Studentships – Cancer Research (Cancer Research UK Manchester Institute)

Faculty Positions – Stem Cell Biology (University of Notre Dame)

Research Technician – TCR Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Technician – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant/Associate/Full Professor – Biomedical Engineering (Worcester Polytechnic Institute)

Assistant Professor – Stem Cell Biology (University of South Carolina)

Postdoctoral Fellow – Immunotherapy/T Cell Biology (California Institute for Biomedical Research)

Postdoctoral Position – Spinal Cord Injury Research (Indiana University-Purdue University)

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