Cell Therapy News 17.24 June 27, 2016 | |
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TOP STORYScientists developed a chimeric antigen receptor (CAR) that recognized cancer-associated Tn glycoform of MUC1, a neoantigen expressed in a variety of cancers. Anti-Tn-MUC1 CAR T cells demonstrated target-specific cytotoxicity and successfully controlled tumor growth in xenograft models of T cell leukemia and pancreatic cancer. [Immunity] Full Article | |
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PUBLICATIONS(Ranked by impact factor of the journal)CD19-chimeric antigen receptor (CAR) T-cells engineered with an enhanced Sleeping Beauty (SB) approach conferred potent reactivity in vitro and eradicated lymphoma in a xenograft model in vivo. Intriguingly, electroporation of SB minicircles was substantially more effective and less toxic compared to conventional plasmids, and enabled cost-effective rapid preparation of therapeutic CAR T-cell doses. [Leukemia] Abstract Researchers showed that αHER2/CD3 specifically targets HER2+ tumor cells, such as those found in gastric cancer and breast cancer, and CD3+ human T cells. αHER2/CD3 can be introduced into CD3+ human T cells via electroporation of the in vitro transcribed capped αHER2/CD3 RNAs instead of lentiviral or retroviral transduction, circumventing the liabilities resulted from viral integration in the host genome. [Cancer Res] Full Article Investigators constructed a novel recombinant Ad5 vector carrying the capsid protein IX modified by the tumor necrosis factor related apoptosis-inducing ligand, which targets tumor cells bearing high levels of its receptor far above those of normal cells. [Oncotarget] Full Article The authors investigated angiogenesis in 3D scaffolds by in vivo multiphoton microscopy during bone formation in a murine calvarial critical bone defect model and evaluated bone regeneration 8 weeks post-implantation. To verify that pdgfb-expressing vectors carried by the scaffolds can promote angiogenesis in 3D-printed scaffolds in vivo, they monitored angiogenesis within the implants by multiphoton microscopy. [Acta Biomater] Abstract The authors investigated the role of apurinic/apyrimidinic endonuclease/redox factor 1 (APE1) in cardiac progenitor cells (CPCs) under ischemic conditions and evaluated the therapeutic efficacy of transplanted APE1-overexpressing CPCs in a mouse model of myocardial infarction. APE1 hindered apoptosis in CPC grafts subjected to oxidative stress caused in part by increased TAK1-NF-κB pathway activation. [Stem Cells Transl Med] Abstract The in vivo potential of a cell-material construct based on periodontal ligament tissue stem cell sheets (PDLSCs), platelet-rich fibrin (PRF) scaffolds and jaw bone mesenchymal stem cells (JBMSCs) sheets to form periodontal tissue was assessed in a nude mouse model. In this model, PDLSC sheet/PRF/JBMSC sheet composites were placed in a simulated periodontal space comprising human treated dentin matrix and hydroxyapatite/tricalcium phosphate frameworks. [Sci Rep] Full Article Under a Phase I dose escalation in prostate cancer, a 20% engraftment target was met or exceeded in three subjects with adequate safety, leading to study conclusion. Clinical responses were obtained but were suggested to be restrained by low plasma IL2 when depleted by high levels of engrafted activated T cells. [Prostate] Abstract To reduce side effects due to non-specific expression, the heme oxygenase-1 gene under control of a hypoxia-inducible erythropoietin enhancer was developed for site-specific gene therapy of ischemic stroke. [Pharm Res] Abstract Researchers have developed a lentiviral vector (CCoq9WP) able to overexpress Coq9 mRNA and COQ9 protein in mouse embryonic fibroblasts and hematopoietic progenitor cells from Coq9R239X mice, an animal model of mitochondrial encephalopathy due to primary Coenzyme Q (CoQ) deficiency. [PLoS One] Full Article | |
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REVIEWSAdvances in Gene Therapy for Diseases of the Eye The reviewers discuss the evolution of preclinical and clinical strategies that have improved gene therapy in the eye, showing that treatment of vision loss has a bright future. [Hum Gene Ther] Full Article | Press Release Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSViaCyte to Present at ISSCR 2016 Annual Meeting ViaCyte, Inc. announced a poster presentation on insulin-producing cells with characteristics of mature human beta cells originated within the Janssen BetaLogics group. In February 2016, the assets of the Janssen BetaLogics group were consolidated into ViaCyte. [Press release from ViaCyte, Inc. discussing research presented at the 2016 International Society for Stem Cell Research (ISSCR) Annual Meeting, San Francisco] Press Release Asterias Biotherapeutics, Inc. announced that a review of its AST-OPC1 (oligodendrocyte progenitor cells) spinal cord injury program will be presented during an oral session. [Press release from Asterias Biotherapeutics, Inc. discussing research presented at the 2016 International Society for Stem Cell Research (ISSCR) Annual Meeting, San Francisco] Press Release | |
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INDUSTRY NEWSCellular Dynamics International, Inc. announced that it has entered into a CRADA with the National Eye Institute a division of the National Institutes of Health, to conduct research to advance a cell transplantation therapy for the treatment of retinal degenerative disease using retinal pigment epithelium derived from induced pluripotent stem cells. [Cellular Dynamics International, Inc.] Press Release ORIG3N and Kangstem Biotech Announce Collaboration to Study Neural Stem Cells ORIG3N, Inc. and Kangstem Biotech announced that they have begun a scientific collaboration to study the utility of induced neural stem cells (iNSC) in disease modeling. As part of the collaboration, Kangstem Biotech will provide iNSCs to ORIG3N for use in its disease-modeling platform. [ORIG3N, Inc. (PR Newswire Association LLC.)] Press Release Kiadis Pharma Appoints PCT as its Contract Manufacturing Organization in the United States Kiadis Pharma N.V. announced that it has appointed PCT, LLC as Kiadis Pharma’s contract manufacturing organization in the United States as Kiadis Pharma makes preparations for its Phase III clinical trial with lead product ATIR101â„¢, on track to be initiated in the second half of 2016. [Kiadis Pharma N.V.] Press Release Celyad Treats First Patient in the Fourth Dose Level of Its NKR-2 Trial Celyad announced the infusion of the first patient enrolled in the fourth dose level of its Phase I/IIa clinical trial. The study is evaluating the safety and feasibility of its NKR-2 T-cell therapy using T-cells with NKG2D receptor in cancer patients suffering from acute myeloid leukemia or multiple myeloma. [Celyad] Press Release Cellectis announced that the first patient has been treated in the Phase I study of UCART19 in pediatric B-ALL at the University College of London. This UCART19 clinical trial is sponsored by Servier in close collaboration with Pfizer. [Cellectis] Press Release Capricor Therapeutics, Inc. announced that patient enrollment in its ongoing randomized HOPE (Halt cardiomyOPathy progrEssion in Duchenne)-Duchenne clinical trial has exceeded 50% of its 24-patient target. [Capricor Therapeutics, Inc.] Press Release Creative Medical Technologies announced filing of a patent application containing novel data suggesting that specialized cells obtained from adipose tissue are capable of preventing recurrent miscarriages in an animal model. These data support the company’s entry into the field of Reproductive Immunology, which aims to address issues such as recurrent miscarriages and preterm labor. [Creative Medical Technology Holdings, Inc. (PR Newswire Association LLC.)] Press Release The Re-Programmer: Qian Receives Global Award for Stem Cell Research The 36-year-old assistant professor at University of Northern Carolina School of Medicine’s Department of Pathology and Laboratory Medicine has been recognized internationally for her scientific developments in cellular reprogramming and stem cell research. Qian is the first-ever recipient of the Boyalife, Science and Science Translational Medicine Award in Stem Cell and Regenerative Medicine. [University of Northern Carolina] Press Release Commence Bio Receives 1st Patent for MSC1: A New Cancer Immunotherapy Platform Commence Bio, Inc. announced the issuance of a patent covering the company’s STaRT 41TM technology utilized for producing MSC1 cells. Representing a new class of cell-based therapy, the MSC1 platform is expected to provide oncologists with a powerful new tool in the fight against cancer. [Commence Bio, Inc.] Press Release Cellular Dynamics International, Inc. announced the appointment of Derek Hei, Ph.D., as Vice President of Clinical Manufacture, Quality, and Regulatory activities to lead efforts in support of manufacturing induced pluripotent stem cell (iPSC)-based products for cell therapies. [Cellular Dynamics International, Inc.] Press Release Incyte Corporation announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for ruxolitinib (Jakafi®) for the treatment of patients with acute graft-versus-host disease. [Incyte Corporation] Press Release New Cell-Based Therapy to Support Stem Cell Transplantation in Patients with High-Risk Blood Cancer The European Medicines Agency has recommended granting a conditional marketing authorization in the European Union for a new advanced therapy medicinal product. Zalmoxis is recommended as an adjunctive, or add on, treatment for adult patients receiving a haploidentical hematopoietic stem cell transplant for various types of blood cancer to aid immune reconstitution and reduce the risk of graft-versus-host disease. [European Medicines Agency] Press Release TxCell SA announces the grant by the European Patent Office of the patent covering all redirected, genetically engineered T regulatory cells (CAR-Tregs) and their use in the suppression of autoimmune and inflammatory diseases. [TxCell SA] Press Release
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POLICY NEWSResearchers Reeling as UK Votes to Leave EU Future of science uncertain after referendum result. [Nature News] Editorial First Proposed Human Test of CRISPR Passes Initial Safety Review A cancer study that would represent the first use of the red-hot gene-editing tool CRISPR in people passed a key safety review. The proposed clinical trial, in which researchers would use CRISPR to engineer immune cells to fight cancer, won approval from the Recombinant DNA Advisory Committee at the U.S. National Institutes of Health, a panel that has traditionally vetted the safety and ethics of gene therapy trials funded by the U.S. government and others. [Science Insider] Editorial Glioproliferative Lesion of the Spinal Cord as a Complication of Stem-Cell Tourism Commercial stem-cell clinics have been highly publicized in the lay press and operate worldwide with limited or no regulation. The authors report the case of a 66-year-old man who underwent intrathecal infusions for the treatment of residual deficits from an ischemic stroke at commercial stem-cell clinics in China, Argentina, and Mexico. [N Engl J Med] Editorial
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REGULATORYFDAInternational Conference on Harmonization; Electronic Transmission of Postmarket Individual Case Safety Reports for Drugs and Biologics, Excluding Vaccines; Availability of Food and Drug Administration Regional Implementation Specifications for ICH E2B(R3) Reporting to the Food and Drug Administration Adverse Event Reporting System (FR Doc. No:2016-14845) Notice Revisions to Exceptions Applicable to Certain Human Cells, Tissues, and Cellular and Tissue-Based Products (FR Doc. No:2016-14721) Notice Medical Devices; Obstetrical and Gynecological Devices; Classification of the Gynecologic Laparoscopic Power Morcellation Containment System (FR Doc. No:2016-14627) Notice
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EVENTSNEW From Stem Cells to Human Development NEW Stem Cell Society Singapore (SCSS) Symposium 2016 Visit our events page to see a complete list of events in the cell therapy community.
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JOB OPPORTUNITIESNEW Faculty Position – Cancer Immunology (University of New Mexico) Scientist – Bioengineering (STEMCELL Technologies Inc.) Postdoctoral Fellow – Oxidative Lung Injury (Temple University) Scientist – Target Validation (Innate Pharma) Postdoctoral Fellow – Data Analysis and Stem Cell Research (Nestle Institute of Health Sciences) Clinical Specialist – Chimeric Antigen Receptor Cell Products (Celgene Corporation) Postdoctoral Fellow – Stem Cells to Study Disease (Hamad bin Khalifa University) Postdoctoral Fellow – Protein and Antibody Engineering (Hamad bin Khalifa University) Postdoctoral Fellow – Cancer Immunology and Immunotherapy (Hamad bin Khalifa University) Postdoctoral Fellow – 3D Microtissues from Pluripotent Stem Cells (Gladstone Institutes) Postdoctoral Fellow – Gene Therapy for Metabolic Homeostasis (Legacy REsearch Institute) PhD Studentship – Various Projects (Vienna Biocenter) Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital) Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.
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Home Cell Therapy News Volume 17.24 | Jun 27 2016