Volume 16.36 | Sep 28

Issue 16.36 September 28, 2015
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‘Remote Control’ of Immune Cells Opens Door to Safer, More Precise Cancer Therapies
Researchers engineered a molecular “on switch” that allows tight control over the actions of T cells, immune system cells that have shown great potential as therapies for cancer. The innovation lays the groundwork for sharply reducing severe, sometimes deadly side effects that have been a significant hurdle to advancing T cell-based treatments. [Press release from the University of California San Francisco discussing online prepublication in Science] Press Release | Abstract | Video
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PUBLICATIONS (Ranked by impact factor of the journal)
Nivolumab versus Docetaxel in Advanced Nonsquamous Non–Small-Cell Lung Cancer
In this randomized, open-label, international Phase III study, researchers assigned patients with nonsquamous non–small-cell lung cancer that had progressed during or after platinum-based doublet chemotherapy to receive nivolumab at a dose of 3 mg per kilogram of body weight every two weeks or docetaxel at a dose of 75 mg per square meter of body-surface area every three weeks. Overall survival was longer with nivolumab than with docetaxel. [N Engl J Med] Abstract | Press Release

Tumor-Expressed IDO Recruits and Activates MDSCs in a Treg-Dependent Manner
Investigators found that indoleamine 2,3-dioxygenase (IDO) expression in human melanoma tumors is strongly associated with myeloid-derived suppressor cell (MDSC) infiltration. Treatment with a selective IDO inhibitor in vivo reversed tumor-associated immunosuppression by decreasing numbers of tumor-infiltrating MDSCs and regulatory T cells (Tregs) and abolishing their suppressive function. [Cell Rep] Full Article | Graphical Abstract

Efficient and Targeted Transduction of Nonhuman Primate Liver with Systemically Delivered Optimized AAV3B Vectors
Scientists compared transduction efficiencies of adeno-associated virus serotype 3B (AAV3B) and AAV8 vectors in cultured primary human hepatocytes and cancer cells as well as in human and mouse hepatocytes in a human liver xenograft NSG-PiZ mouse model. They also examined the safety and transduction efficacy of wild-type and capsid-optimized recombinant AAV3B in the livers of nonhuman primates. [Mol Ther] Abstract

Delivery of siRNAs to Dendritic Cells Using DEC205-Targeted Lipid Nanoparticles to Inhibit Immune Responses
Researchers showed that injection of siRNAs encapsulated in DEC-LNPs are preferentially delivered to DEC205+ dendritic cells. Gene knockdown following uptake of DEC-LNPs containing siRNAs specific for the costimulatory molecules CD40, CD80 and CD86 dramatically decreases gene expression levels. [Mol Ther]
Abstract | Full Article

A RANKL Mutant Used as an Inter-Species Vaccine for Efficient Immunotherapy of Osteoporosis
The authors propose a strategy using an inter-species cytokine as immunogen for active immunization to induce anti-cytokine antibody. As a proof of concept, an inter-species cytokine RANKL was successfully used as immunogen to induce anti-RANKL immune response. [Sci Rep] Full Article

Umbilical Cord Blood-Derived CD11c+ Dendritic Cells Could Serve as an Alternative Allogeneic Source of Dendritic Cells for Cancer Immunotherapy
The authors compared CD11c+ umbilical cord blood-dendritic cells (DCs)/cytotoxic T lymphocytes (CTLs) with the conventional source, i.e. peripheral blood monocyte DCs/CTLs, using various parameters. In vivo CTL activity was found to be similar with the two sources. [Stem Cell Res Ther] Full Article

Cell-Based Therapy in TBI: Magnetic Retention of Neural Stem Cells In Vivo
To investigate the effects of applying a magnetic field on cell homing and retention, scientists delivered human neuroprogenitor cells labeled with a superparamagnetic nanoparticle into post-traumatic brain injury (TBI) animals in the presence of a static magnetic field. [Cell Transplant] Full Article

Treatment of Perianal Fistulas with Human Embryonic Stem Cell-Derived MSCs: A Canine Model of Human Fistulizing Crohn’s Disease
Investigators evaluated the safety and efficacy of intralesional injection of human embryonic stem cell-derived mesenchymal stem/stromal cells (MSCs) in canine anal furunculosis dogs. [Regen Med]
Full Article | Press Release

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Engineering Cell Fate for Tissue Regeneration by In Vivo Transdifferentiation
The authors review the most relevant in vivo transdifferentiation studies in adult mammalian animal models, offering a critical assessment of this potentially powerful strategy for regenerative medicine. [Stem Cell Rev] Abstract

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

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Genentech Presents Positive Results from Pivotal Study of Investigational Immunotherapy Atezolizumab in Specific Type of Advanced Bladder Cancer
Genentech announced early results from a pivotal Phase II study, IMvigor 210, of the investigational cancer immunotherapy atezolizumab in people with locally advanced or metastatic urothelial carcinoma. [Press release from Genentech, Inc. discussing research presented at the European Cancer Congress (ECC 2015), Vienna] Press Release

OncoSec Announces Positive Results from Phase II Trial of ImmunoPulse™ IL-12 in Merkel Cell Carcinoma
OncoSec Medical Incorporated announced results from a Phase II trial demonstrating that its investigational therapy, ImmunoPulse™ IL-12, promotes tumor-specific, systemic anti-tumor immune responses in patients with Merkel cell carcinoma. [Press release from OncoSec Medical Incorporated discussing research presented at the European Cancer Congress (ECC 2015), Vienna] Press Release

New Data from KEYNOTE-028, Merck’s Trial Evaluating KEYTRUDA® (Pembrolizumab) Across a Range of Cancer Types
Merck announced new findings from the KEYNOTE-028 Phase Ib study, the clinical trial investigating the use of the company’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab) in multiple, difficult-to-treat cancers. [Press release from Merck discussing research presented at the European Cancer Congress (ECC 2015), Vienna] Press Release

Two Positive Studies of Genentech’s Investigational Cancer Immunotherapy Atezolizumab in Specific Type of Lung Cancer Presented
Genentech announced positive results from two Phase II studies that evaluated the investigational cancer immunotherapy atezolizumab in people with advanced non-small cell lung cancer. [Press release from Genentech, Inc. discussing research presented at the European Cancer Congress (ECC 2015), Vienna] Press Release

Positive Topline Results Announced from Phase I/II Trial in Sanfilippo B Syndrome Patients Using uniQure’s Novel AAV5-Based Gene Therapy
uniQure N.V. announced the topline results of one-year follow-up data from a Phase I/II clinical trial conducted by Institut Pasteur in partnership with the French Muscular Dystrophy Association and Vaincre les Maladies Lysosomales in four Sanfilippo B syndrome patients treated with a novel gene therapy, AMT-110. [Press release from uniQure N.V. discussing research presented at the European Society of Gene and Cell Therapy (ESGCT) and Finnish Society of Gene Therapy (FSGT) Collaborative Congress, Helsinki] Press Release

AlloCSC-01 Phase I Data Presented
TiGenix NV, an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platforms of allogeneic expanded stem cells, announced that the CAREMI dose-escalation open-label phase data were presented. [Press release from TiGenix NV (GlobeNewswire, Inc.) discussing research presented at the 2015 Congress of the European Society of Cardiology (ESC), London] Press Release

Celimmune to Present Overview of Non-Dietary Celiac Disease Therapies in Development
Dr. Francisco Leon, CEO and Chief Medical Officer, will discuss the characteristics and mechanism of action of several investigational non-dietary treatments for celiac disease and gluten-related conditions, including AMG 714, a Phase II-stage, anti-IL-15 monoclonal antibody that Celimmune is developing for the treatment of diet non-responsive celiac disease and refractory celiac disease type II. [Press release from Celimmune LLC (PR Newswire Association LLC) discussing research to be presented at the Kansas City Gluten & Allergen-Free Wellness Event, Overland Park] Press Release

Cellular Biomedicine Group Announces Positive Results from CAR-T EGFR Immunotherapy in Advanced Relapsed/Refractory Patients with Solid Tumors
Cellular Biomedicine Group Inc. announced results from an expanded Phase I/II clinical trial evaluating the safety, feasibility and anti-tumor activity of its chimeric antigen receptor-modified T-cells (CAR-T) immunotherapy targeting wild type EGFR (epidermal growth factor receptor) for the treatment of patients with EGFR expressing advanced relapsed/refractory solid tumors. [Press release from Cellular Biomedicine Group Inc. (GlobeNewswire, Inc.) discussing research presented at the 5th World Congress on Cancer Therapy, Atlanta] Press Release

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New U.S. Board Proposed to Tackle Regulatory Burden on Research Universities
The best way to ease the regulatory burden on U.S. academic researchers is to create another layer of bureaucracy. That surprising conclusion is the top recommendation in a report by a National Academies committee that Congress asked to examine the current regulatory jungle confronting universities that receive federal research dollars. [ScienceInsider] Editorial

STAP Revisited
Nature revisited one of the most controversial scientific episodes in recent years: the now-retracted discovery of a claimed new way to reprogram cells, stimulus-triggered acquisition of pluripotency (STAP). [Nature News] Editorial
$25 Million for UQ Genomics Research
A $25 million boost to genomics research at Queensland research institutions will help experts in their efforts to improve healthcare. The State Government announced new funding for genomics research today at The University of Queensland (UQ). [The University of Queensland] Press Release

Arrowhead Reports Peak Reduction in HBsAg of Up to 99% after a Single Dose with Hepatitis B Candidate ARC-520 in Treatment Naïve Cohort of Phase IIa Study
Arrowhead Research Corporation discussed top-line findings from the Heparc-2001 Phase IIa clinical study of ARC-520, its candidate for the treatment of chronic hepatitis B infection. [Arrowhead Research Corporation] Press Release

Syndax and Merck Announce First Patients Dosed in Phase Ib/II Clinical Trial of Entinostat and KEYTRUDA®
Syndax Pharmaceuticals, Inc. and Merck announced the dosing of the first patients in the Phase Ib/II clinical trial of Syndax’s entinostat in combination with Merck’s anti-PD-1 therapy KEYTRUDA® (pembrolizumab) in patients with non-small cell lung cancer or melanoma. [Merck] Press Release

ImmunoCellular Therapeutics Awarded $19.9 Million Grant from California Institute for Regenerative Medicine (CIRM) to Fund ICT-107 Phase III Registration Program in Glioblastoma
ImmunoCellular Therapeutics, Ltd. announced that the governing Board of the CIRM has awarded the company $19.9 million to support the ICT-107 Phase III registration trial in patients with newly diagnosed glioblastoma. [ImmunoCellular Therapeutics, Ltd.] Press Release

RuiYi Announces the First Human Dosing of a Novel Antibody, Gerilimzumab, for the Treatment of Rheumatoid Arthritis and other Autoimmune Disorders
RuiYi, Inc. announced the first human dosing of Gerilimzumab, a novel monoclonal antibody that is directed against the IL-6 cytokine, for the treatment of autoimmune disorders, including rheumatoid arthritis. [RuiYi, Inc.] Press Release

Merck KGaA and Pfizer Announce FDA Orphan Drug Designation for Investigational Immunotherapy Avelumab in Merkel Cell Carcinoma
Merck KGaA and Pfizer announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for the investigational cancer immunotherapy avelumab for the treatment of Merkel cell carcinoma, a rare and aggressive type of skin cancer. [Pfizer Inc.] Press Release

Macrophage Therapeutics Produces First Two Immunoconstructs
Macrophage Therapeutics, Inc. announced that it has developed a process for producing the first two therapeutic Manocept™ immunoconstructs, MT-1001, designed to specifically target and kill activated CD206+ macrophages and MT-2001, designed to inhibit the inflammatory activity of activated CD206+ macrophages. [Navidea Biopharmaceuticals, Inc.] Press Release

Bristol-Myers Squibb Announces U.S. Food and Drug Administration Regulatory Filing Update for the Opdivo+Yervoy Regimen in Patients with Previously Untreated Advanced Melanoma
Bristol-Myers Squibb Company announced that the U.S. Food and Drug Administration has accepted for filing and review a supplemental biologics license application for the Opdivo+Yervoy regimen to include clinical data from CheckMate -067, a landmark trial in patients with previously untreated advanced melanoma. [Bristol-Myers Squibb Company] Press Release

Sorrento Therapeutics and City of Hope Announce Exclusive License to Develop First-in-Class Immunotherapies against Intracellular Targets
Sorrento Therapeutics, Inc. announced that they have formed a new company – named LA Cell, Inc. (LA Cell) – to focus on the development of cell-penetrating antibody therapies. LA Cell has exclusively licensed technology developed at City of Hope that enables modified monoclonal antibodies to penetrate into cells and target “undruggable” disease-causing molecules. [Sorrento Therapeutics, Inc.] Press Release

RXi Pharmaceuticals Granted Patent in China for Lead Clinical Candidate RXI-109
RXi Pharmaceuticals Corporation announced that it has been granted a patent from the State Intellectual Property Office of the People’s Republic of China for the composition and methods of use for RXI-109 and other connective tissue growth factor targeting self-delivering RNAi compounds for the treatment of fibrotic disorders, including skin fibrosis. [RXi Pharmaceuticals Corporation] Press Release
National Institute of Allergy and Infectious Diseases; Notice of Closed Meetings (FR Doc. No: 2015-24488)

National Institute of Allergy and Infectious Diseases; Notice of Closed Meetings (FR Doc. No: 2015-24489)

Center For Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-24320)
Joint Meeting of the Cellular, Tissue and Gene Therapies Advisory Committee and Oncologic Drugs Advisory Committee Meeting Announcement
Food and Drug Administration (United States)

Joint Meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee and the Oncologic Drugs Advisory Committee; Notice of Meeting (FR Doc. No: 2015-24541)

Biosimilar User Fee Act; Public Meeting (FR Doc. No: 2015-24524)
NEW Cell Culture 2016
February 2-4, 2016
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Assistant/Associate/Full Professor – Biomedical Engineering (Worcester Polytechnic Institute)

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

Senior Stem Cell Scientist (Medicines and Healthcare Products Regulatory Agency)

Postdoctoral Researcher – Epigenetic Modifiers (Van Andel Research Institute)

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

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

Postdoctoral Fellow – HMGA Chromatin Remodeling Proteins in Cancer and Stem Cell Biology (Johns Hopkins University School of Medicine)

Postdoctoral Fellow Position – Genome Editing, Synthetic Biology, and Cell Evolution and Engineering (University of California – Irvine)

Research Associate – Cell Biology (Editas Medicine)

Senior Research Associate (Editas Medicine)

Cell Biology Lab Manager (Harvard Apparatus Regenerative Technology)

Science Writer – Cardiovascular Regeneration (Houston Methodist Research Institute)

Aseptic Manufacturing Associate (Lonza Biologics, Inc.)

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