Volume 16.28 | Jul 20

Issue 16.28 July 20, 2015
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Scientists Unlock First Critical Step Toward Gene Therapy Treatment for Patients with Mitochondrial Disease
A study has revealed the first critical step in developing novel gene and stem cell therapy treatments for patients with mitochondrial disease. This breakthrough sets the stage for replacing diseased tissue in patients and opens the door to a world of regenerative medicine where doctors are able to treat human diseases that are currently incurable. [Press release from Oregon Health & Science University discussing online publication in Nature] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Plasticity of the Human Visual System after Retinal Gene Therapy in Patients with Leber’s Congenital Amaurosis
Scientists evaluated human brain plasticity in a group of patients with Leber’s congenital amaurosis (LCA), who regained vision through gene therapy. Using non-invasive multimodal neuroimaging methods, they demonstrated that reversing blindness with gene therapy promoted long-term structural plasticity in the visual pathways emanating from the treated retina of LCA patients. [Sci Transl Med] Abstract | Press Release

Enrichment and Expansion with Nanoscale Artificial Antigen Presenting Cells for Adoptive Immunotherapy
Researchers developed an enrichment + expansion strategy using paramagnetic, nanoscale artificial antigen presenting cells to rapidly expand tumor-specific T cells from rare naïve precursors and predicted neo-epitope responses. [ACS Nano] Abstract | Graphical Abstract | Press Release

Evaluating Human T-Cell Therapy of Cytomegalovirus Organ Disease in HLA-Transgenic Mice
Investigators introduced a novel mouse model for testing human cytomegalovirus (HCMV) epitope-specific human T cells. The HCMV UL83/pp65-derived NLV-peptide was presented by transgenic HLA-A2.1 in the context of a lethal infection of NOD/SCID/IL-2rg-/- mice with a chimeric murine CMV, mCMV-NLV. [PLoS Pathog] Full Article

Molecular Mechanisms Mediating Retinal Reactive Gliosis following Bone Marrow Mesenchymal Stem Cell (BM-MSC) Transplantation
Researchers found in vivo that intravitreal BM-MSC transplantation is associated with gliosis-mediated retinal folding, up-regulation of intermediate filaments and recruitment of macrophages. [Stem Cells] Abstract

Gene Therapy with SOCS1 for Gastric Cancer Induces G2/M Arrest and Has an Antitumor Effect on Peritoneal Carcinomatosis
Investigators analyzed the mechanism of antitumor effect of suppressor of cytokine signaling1 (SOCS1) in vitro. In addition, they evaluated antitumor effect for gastric cancer using a xenograft peritoneal carcinomatosis mouse model in preclinical setting. [Br J Cancer] Abstract

Acute and Temporal Expression of TNF-α-Stimulated Gene 6 Product, TSG-6, in Mesenchymal Stem Cells Creates Microenvironments Required for Their Successful Transplantation into the Muscle Tissue
Researchers investigated micro environments crucial for cell transplantation, using the lysate of C2C12 myotubules for mimicking injured circumstances in vivo. Mesenchymal stem cells settled in the intact muscle when they were transplanted together with the C2C12 lysate or TNF-α-stimulated gene 6 (TSG6). [J Biol Chem] Abstract | Full Article

Gelatin Hydrogel Enhances the Engraftment of Transplanted Cardiomyocytes and Angiogenesis to Ameliorate Cardiac Function after Myocardial Infarction
Although the authors observed that collagen strongly induced the aggregation of platelets to potentially cause coronary microembolization, gelatin hydrogel (GH) did not enhance thrombogenicity. Therefore, GH is a suitable biomaterial in the cell therapy after heart failure. [PLoS One] Full Article

Gene Therapy with Neurogenin3, Betacellulin and SOCS-1 Reverses Diabetes in NOD Mice
Scientists demonstrated that gene therapy with helper-dependent adenovirus carrying neurogenin3, an islet lineage-defining transcription factor and betacellulin, an islet growth factor, leads to the induction of periportal insulin-positive cell clusters in the liver, which are rapidly destroyed. [Gene Ther] Abstract

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Prospects of Neurotrophic Factors for Parkinson’s Disease: Comparison of Protein and Gene Therapy
The authors discuss the emerging and existing possibilities to use neurotrophic factors as neurorestorative agents and the ways to improve their efficacy, and compare the gene therapy and recombinant protein therapy approaches for restoring dopamine circuitry in Parkinson’s disease. [Hum Gene 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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VBL Therapeutics to Present Data on Proprietary Gene Therapy Vascular Targeting System™ and Lead Product VB-111
VBL Therapeutics announced that Chief Executive Officer, Dror Harats, M.D., will present data regarding the company’s Vascular Targeting System™ and its lead product VB-111 in an oral presentation. [Press release from VBL Therapeutics discussing research to be presented at the Drug Discovery and Therapy World Congress 2015, Boston] Press Release

Argos Therapeutics to Present at 8th IAS Conference on HIV Pathogenesis, Treatment and Prevention
Argos Therapeutics Inc. announced Irina Tcherepanova, Ph.D. will participate in a roundtable discussion on combination therapy trials at the “Towards an HIV Cure” symposium. [Press release from Argos Therapeutics Inc. discussing research to be presented at the 8th International AIDS Society (IAS) Conference on HIV Pathogenesis, Treatment and Prevention, Vancouver] Press Release

Stem Cells & Regenerative Medicine Congress 2015
Congress Pushes NIH to Spur Breakthroughs through Prizes
As U.S. lawmakers search for ways to incentivize medical breakthroughs, some appear to have their eyes on the prize. A provision in a new biomedical innovation bill passed in the House of Representatives would create a new program to launch prize competitions at the National Institutes of Health (NIH). [ScienceInsider] Editorial

Iran Nuclear Deal Opens Door to Scientific Collaborations
After two years of negotiations, Iran agreed to dismantle large pieces of its nuclear program in exchange for lifting crippling economic sanctions. The agreement paves the way for a rapid expansion of scientific cooperation with Iran in areas as diverse as fusion, astrophysics, and cancer therapy using radioisotopes. [ScienceInsider] Editorial

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Medtown Ventures, LLC Announces Exclusive License Agreement with Georgia Tech Research Corporation
Medtown Ventures, LLC announced that it has entered into an exclusive license agreement with Georgia Tech Research Corporation for a new adhesive-signature based stem cell selection and isolation technology. [Medtown Ventures, LLC] Press Release

CANbridge Acquires License for Apogenix’s APG101 Onco-Immunotherapy in Glioblastoma for China
CANbridge Life Sciences has entered into an exclusive license agreement with Apogenix to develop, manufacture and commercialize Apogenix’s lead product, APG101, in glioblastoma multiforme in China, Macao, and Hong Kong, with options for other indications. [CANbridge Life Sciences Ltd.] Press Release

Fibrocell and Intrexon Announce IND Filing of FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Fibrocell Science, Inc. and Intrexon Corporation announced the submission of an Investigational New Drug (IND) Application with the U.S. Food and Drug Administration for FCX-007, Fibrocell’s lead orphan gene-therapy drug candidate for the treatment of RDEB. [Fibrocell Science, Inc.] Press Release

Wayne State Receives $1.9 Million NIH Grant for Enhancing EPC-Based Cell Therapy for Vascular Diseases
Chunying Li, Ph.D. has secured his first R01 grant from the National Heart, Lung, and Blood Institute of the National Institutes of Health to study the role and mechanism of the chemokine receptor CXCR2 in regulating new blood vessel formation. [Wayne State University] Press Release

University of Louisville Physicians Conducting Vaccine Trial for Children with Relapsed Tumors at Kosair Children’s Hospital
Investigators at Kosair Children’s Hospital and in the University of Louisville Department of Pediatrics are making progress in developing a vaccine that one day could possibly prevent recurrence of some childhood cancers. [University of Louisville] Press Release

Vanderbilt University Medical Center Joins Human Vaccines Project as First Scientific Hub
Vanderbilt University Medical Center (VUMC), the Human Vaccines Project and the International AIDS Vaccine Initiative are pleased to announce that VUMC has become the Project’s first scientific hub. [International AIDS Vaccine Initiative] Press Release

Amgen Announces Positive BLINCYTO® (Blinatumomab) Phase II Study Results in Patients with Relapsed/Refractory Philadelphia Chromosome-Positive B-Cell Precursor Acute Lymphoblastic Leukemia
Amgen announced the top-line results of a Phase II open-label, single-arm, multicenter trial to evaluate the efficacy and safety of BLINCYTO® in adults with relapsed or refractory Philadelphia chromosome-positive B-cell precursor acute lymphoblastic leukemia. [Amgen] Press Release

Bavarian Nordic Announces that the Oxford Vaccines Group has Initiated a Phase II Study of the Ebola Prime-Boost Vaccine Regimen Combining MVA-BN® Filo and Janssen’s AdVac® Technology
Bavarian Nordic A/S announced that the Oxford Vaccines Group has initiated a Phase II clinical study of the Ebola prime-boost vaccine regimen that combines Bavarian Nordic’s MVA-BN® Filo vaccine with the Ad26.ZEBOV vaccine from the Janssen Pharmaceutical Companies of Johnson & Johnson. [Bavarian Nordic A/S] Press Release
National Institute of General Medical Sciences; Notice of Closed Meeting (FR Doc. No: 2015-17219)
Suspension Letter – United States Blood Bank, Inc.
Food and Drug Administration (United States)

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting (FR Doc. No: 2015-17559)

Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry on Oversight of Clinical Investigations: A Risk-Based Approach To Monitoring (FR Doc. No: 2015-17318)

Public Meeting on Patient-Focused Drug Development for Huntington’s and Parkinson’s Diseases (FR Doc. No: 2015-17556)
NEW Tumor Immunology and Immunotherapy
September 21-25, 2015
Suzhou, China

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Cell Biology Lab Manager (Harvard Apparatus Regenerative Technology)

NEW Science Writer – Cardiovascular Regeneration (Houston Methodist Research Institute)

PhD Studentship – Veterinary and Animal Sciences (University of Milan)

Postdoctoral Research Fellow – Fat Metabolism and Stem Cells (Singapore Bioimaging Consortium)

Postdoctoral Research Associate – Breast Stem Cell Biology (University of Miami Miller School of Medicine)

Aseptic Manufacturing Associate (Lonza Biologics, Inc.)

Postdoctoral Position – Translational Immunology (University of Utah School of Medicine)

Postdoctoral Position – Splicing in Hematopoiesis (Northwestern University)

Primary Cell Transduction Process Scientist (GlaxoSmithKline)

Postdoctoral Fellow – Antibody Engineering/Immuno-Oncology (California Institute for Biomedical Research)

Postdoctoral Fellow – Stem Cell Biology (INSERM)

Cell Therapy GMP Manager (Paramount Recruitment)

Postdoctoral Fellow – Neural Stem Cells (Children’s Hospital of Philadelphia)

Lead Device and Delivery Scientist (Cell Therapy Catapult)

Postdoctoral Fellow – Immune Reconstitution in Hematopoietic Stem Cell Transplantation (Stanford University)

Process Facilitator (Opexa Therapeutics)

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