Volume 16.07 | Feb 23

Issue 16.07 February 23, 2015
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Study Describes Development of Personalized Cellular Therapy for Brain Cancer
Immune cells engineered to seek out and attack a type of deadly brain cancer were found to be both safe and effective at controlling tumor growth in mice that were treated with these modified cells. The results paved the way for a newly opened clinical trial for glioblastoma patients. [Press release from the University of Pennsylvania discussing online publication in Science Translational Medicine] Press Release | Abstract
Learn More: UM729, a Novel Small Molecule for Ex Vivo Expansion of Human HSCs
PUBLICATIONS (Ranked by impact factor of the journal)
Multiplex CRISPR/​Cas9-Based Genome Editing for Correction of Dystrophin Mutations that Cause Duchenne Muscular Dystrophy
Investigators used the CRISPR/​Cas9 system to restore the expression of the ​dystrophin gene in cells carrying ​dystrophin mutations that cause Duchenne muscular dystrophy. [Nat Commun] Abstract | Press Release

Defined TLR3-Specific Adjuvant that Induces NK and CTL Activation without Significant Cytokine Production In Vivo
Researchers created an RNA ligand for Toll-like receptor (TLR)3 with no ability to activate the RIG-I/MDA5 pathway. This TLR3 ligand is a chimeric molecule consisting of phosphorothioate ODN-guided dsRNA, which elicits far less cytokine production than poly(I:C) in vitro and in vivo. [Nat Commun] Abstract

Tumor Infiltrating Lymphocytes Genetically Engineered with an Inducible Gene Encoding Interleukin-12 for the Immunotherapy of Metastatic Melanoma
Scientists evaluated the anti-tumor activity of adoptively transferred human tumor infiltrating lymphocytes genetically engineered to secrete single-chain interleukin-12 selectively at the tumor site. [Clin Cancer Res] Abstract

K562-Derived Whole-Cell Vaccine Enhances Antitumor Responses of CAR-Redirected Virus-Specific Cytotoxic-T Lymphocytes In Vivo
Investigators developed an off-the-shelf whole-cell vaccine to boost chimeric antigen receptor (CAR)-redirected virus-specific cytotoxic T cells in vivo after adoptive transfer. [Clin Cancer Res] Abstract

The Sulfated Polysaccharide Fucoidan Rescues Senescence of Endothelial Colony Forming Cells for Ischemic Repair
Researchers investigated the potential of sulfated polysaccharide fucoidan to rescue endothelial colony-forming cells (ECFCs) from cellular senescence and to improve in vivo vascular repair by ECFCs. [Stem Cells] Abstract

Enhanced Restoration of In Situ-Damaged Hairs by Intradermal Transplantation of Trichogenous Dermal Cells
Scientists developed a nude rat model for determining the capacity of trichogenous cells to restore in vivo-damaged hair follicles (HFs). A surgical scalpel was inserted into the rat’s dermis to generate the in vivo-damaged pelage HFs, the HFs whose lower parts were lost, but the upper parts containing sebaceous and bulge regions remained intact. [J Tissue Eng Regen Med] Abstract

Large Animal In Vivo Evaluation of a Binary Blend Polymer Scaffold for Skeletal Tissue-Engineering Strategies; Translational Issues
Investigators explored the hypothesis that cell therapy would enhance bone formation in a critical-sized defect compared to scaffold alone, using an external fixation construct, to bridge the scale-up gap between small animal studies and potential clinical translation. [J Tissue Eng Regen Med] Abstract

Influence of Bone Marrow-Derived Mesenchymal Stem Cells (MSCs) Pre-Implantation Differentiation Approach on Periodontal Regeneration In Vivo
The implantation of bone marrow-derived MSCs has previously been shown successful to achieve periodontal regeneration. However, the preferred pre-implantation differentiation strategy (e.g. maintenance of stemness, osteogenic or chondrogenic induction) to obtain optimal periodontal regeneration is still unknown. Scientists explored which differentiation approach is most suitable for periodontal regeneration. [J Clin Periodontol] Abstract

RNAi-Mediated CCR5 Knockdown Provides HIV-1 Resistance to Memory T Cells in Humanized BLT Mice
Transplantation of hematopoietic stem/progenitor cells modified with a lentiviral vector bearing a potent nontoxic short hairpin RNA (sh1005) directed to the HIV coreceptor CCR5 is capable of continuously producing CCR5 downregulated CD4+ T lymphocytes. Researchers characterized HIV-1 resistance of the sh1005-modified CD4+ T lymphocytes in vivo in humanized bone marrow/liver/thymus mice. [Mol Ther Nucleic Acids] Full Article

Free Nature Reviews Immunology Poster: The Immune Response to HIV
Humanized Models of Tumor Immunology in the 21st Century: Convergence of Cancer Research and Tissue Engineering
The authors provide an overview of the state of the art of humanized models of tumor immunology and highlights future developments in the field such as the application of tissue engineering & regenerative medicine strategies to further enhance humanized murine model systems. [Stem Cells] Abstract

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

Free Sample: TeSR™2 Defined, Feeder-Free and Xeno-Free Human ES and iPS Cell Medium
Lycera Announces Research on Anti-Cancer Activity of Oral Immunotherapy Candidates
Lycera Corp. recently announced promising data related to the activity of an anti-tumoral RORγ agonist candidate. The study shows that the company’s RORγ agonists have the ability to reprogram T cells and enhance their activity and their capacity to survive, allowing them to resist tumors’ immunosuppressive mechanisms that can impose limits to the efficacy of cancer immunotherapies. [Press release from Lycera Corp. discussing research to be presented at the 2015 Keystone Tumor Immunology Symposium, Banff] Press Release

Celsion Corporation Presents Clinical and Preclinical Data for GEN-1 IL-12 Immunotherapy in Ovarian Cancer
Celsion Corporation presented clinical and preclinical data demonstrating the safety, biological activity and clinical benefits of GEN-1, its DNA-based immunotherapy, as a single agent in advanced platinum-resistant and recurrent ovarian cancer patients. [Press release from Celsion Corporation discussing research presented at the Molecular Medicine TRI-Conference, San Francisco] Press Release

From our sponsor: Free wallchart from Nature Neuroscience – Neural Stem Cells. Request your copy.
State Funding Boosts Stem Cell Research in California, Other States
A new study analyzed stem cell funding programs in four states that provided their own funding and found that in both California and Connecticut, state programs have contributed to an increase in the share of publications in the field produced in these states. [Georgia Institute of Technology] Editorial | Report

A New Shot at Reducing Research Red Tape
No matter how much scientists complain about it, federal oversight of academic research isn’t going away. The chair of a new National Academies panel examining how the government keeps tabs on its $40-billion-a-year investment wants that oversight “to be sensible enough so that investigators have more time to do research.” [ScienceInsider] Editorial
Agilvax Announces Strategic Collaboration with Humabs BioMed SA to Develop Targeted Vaccines and Immunotherapies
Agilvax, Inc. announced a collaboration with Humabs BioMed that will contribute to the rapid advancement of Agilvax’s ever expanding vaccine and immunotherapy pipeline. Humabs BioMed has established itself as an innovator and leader in the development of human monoclonal antibodies. [Agilvax, Inc.] Press Release

Dako, an Agilent Technologies Company, and ONO PHARMACEUTICAL CO., LTD. Announce Collaboration on Development of PD-L1 Companion Diagnostic Test for Investigational Cancer Drug Opdivo® (Nivolumab)
Dako and ONO PHARMACEUTICAL CO., LTD. announced a new partnership to develop a potential diagnostic test specifically for use with Opdivo in the treatment of non-small cell lung cancer. [Agilent Technologies, Inc.] Press Release

Heat Biologics, Inc. and OncoSec Medical Inc. Announce Collaboration to Evaluate Combination of Immunotherapy Platforms
Heat Biologics, Inc. and OncoSec Medical Inc. announced that they have entered into an agreement to evaluate the combination of the immunotherapy approaches developed by each company. [Heat Biologics, Inc.]
Press Release

UCSF Receives $100 Million Gift to Advance Health Sciences Mission
UC San Francisco has received a $100 million gift from visionary philanthropist Charles F. “Chuck” Feeney to support its new Mission Bay hospitals, world-class faculty and students, and research programs focused on the neurosciences and aging. [University of California, San Francisco] Press Release

Kaul Foundation Donates $7 Million for UAB Personalized Medicine Institute
The Hugh Kaul Foundation has donated $7 million to the University of Alabama at Birmingham (UAB) to establish the Hugh Kaul Personalized Medicine Institute at UAB. [University of Alabama at Birmingham] Press Release

Stem Cell ‘Tool’ Grants Given to Five Researchers
Five Stanford researchers were awarded grants of between $1 million and $2 million each by the California Institute for Regenerative Medicine at its most recent board meeting. The awards were given through the agency’s “tools and technology” grant program, which encourages researchers to develop new methods and techniques to overcome stumbling blocks in the field. [Stanford Medicine] Press Release

Exelixis Announces Acceptance of New Drug Application for Cobimetinib in Combination with Vemurafenib for Treatment of Patients with BRAF V600 Mutation-Positive Advanced Melanoma
Exelixis, Inc. announced that the U.S. Food and Drug Administration has accepted for review Genentech’s New Drug Application for cobimetinib in combination with vemurafenib for patients with unresectable or metastatic melanoma harboring a BRAF V600 mutation. [Exelixis, Inc.] Press Release

Europe Approves Holoclar®, the First Stem Cell-Based Medicinal Product
The European Commission has granted a conditional marketing authorization, under Regulation (EC) No 726/2004, to Holoclar®, an advanced therapy based on autologous stem cells and capable to restore the eyesight of patients with severe cornea damage. [Chiesi Farmaceutici S.p.A.] Press Release

RepliCel Submits Clinical Trial Application for Its Dermatological Clinical Trial
RepliCel Life Sciences Inc. announced the submission of a Clinical Trial Application to the German Competent Authority, the Paul-Ehrlich-Institut, requesting clearance to initiate a Phase I clinical trial investigating the use of RCS-01 to treat patients suffering from aged and UV-damaged skin. [RepliCel Life Sciences Inc.] Press Release

Kite Pharma Establishes Manufacturing Capabilities to Support Development and Commercialization of CAR and TCR Cancer Immunotherapy Products
Kite Pharma, Inc. announced that it has entered into a lease agreement for a commercial manufacturing facility in El Segundo, California. Kite also recently secured a lease for a clinical manufacturing facility in Santa Monica, California. The two facilities will not only support the planned clinical trials of Kite’s product candidates, but also will prepare Kite for the commercial launch and supply of its lead product candidate, KTE-C19. [Kite Pharma, Inc.] Press Release

Prospective Grant of Exclusive License: Start-up Evaluation License for the Development of Theranostic Kits for Taxane-based Chemotherapy (FR Doc. No: 2015-03088)
Investigating and Reporting Adverse Reactions Related to Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Regulated Solely under Section 361 of the Public Health Service Act and 21 CFR Part 1271; Draft Guidance
Food and Drug Administration (United States)

Mixing, Diluting, or Repackaging Biological Products Outside the Scope of an Approved Biologics License Application; Draft Guidance for Industry; Availability (FR Doc. No: 2015-03418)

Repackaging of Certain Human Drug Products by Pharmacies and Outsourcing Facilities; Draft Guidance for Industry; Availability (FR Doc. No: 2015-03417)
NEW Stem Cell Summit 2015
April 27-29, 2015
Boston, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Assistant Professor – Neuroscience (Georgia State University)

Research Associate – Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

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

Scientist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

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.)

Research Technician in AAV Gene Therapy (Icahn School of Medicine at Mount Sinai)

T Cell Process Development Scientist/Senior Scientist (Adaptimmune Ltd.)

Senior Process Development Engineer (Sangamo BioSciences, Inc.)

Director – Vector Production (Sangamo BioSciences, Inc.)

Postdoctoral Fellow – Vascular Bioengineering (University of Pittsburgh)

Assistant/Associate Professor – Gastroenterology (North Carolina State University College of Veterinary Medicine)

Postdoctoral Fellow – Wnt Signaling in Development and Disease (Van Andel Research Institute)

Postdoctoral Fellowship – CNS Inflammation (Rutgers University)

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