Volume 16.09 | Mar 9

Newsletter Issue
Issue 16.09 March 9, 2015
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Area-Specific Reestablishment of Damaged Circuits in the Adult Cerebral Cortex by Cortical Neurons Derived from Mouse Embryonic Stem Cells
Scientists showed that neurons of visual cortex identity, differentiated in vitro from mouse embryonic stem cells, can be transplanted successfully following a lesion of the adult mouse visual cortex. [Neuron]
Abstract | Press Release
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PUBLICATIONS (Ranked by impact factor of the journal)
Radiation and Dual Checkpoint Blockade Activate Non-Redundant Immune Mechanisms in Cancer
Researchers report major tumor regressions in a subset of patients with metastatic melanoma treated with an anti-CTLA4 antibody and radiation, and reproduced this effect in mouse models. [Nature] Abstract | Press Release

Five-Year Survival Rates for Treatment-Naive Patients with Advanced Melanoma who Received Ipilimumab plus Dacarbazine in a Phase III Trial
To demonstrate a long-term survival benefit with ipilimumab, investigators evaluated the five-year survival rates of patients treated in a randomized, controlled Phase III trial. [J Clin Oncol] Abstract | Press Release

Generation of Human Memory Stem T Cells upon Haploidentical T-Replete Hematopoietic Stem Cell Transplantation
Investigators longitudinally tracked memory stem T cells dynamics in patients undergoing haploidentical hematopoietic stem cell transplantation, thereby providing novel hints on the contribution of this subset to post transplant immune reconstitution in humans. [Blood] Abstract

DAP12-Based Activating Chimeric Antigen Receptor for NK Cell Tumor Immunotherapy
On the basis of DNAX-activation protein 12 (DAP-12), a signaling adaptor molecule involved in signal transduction of activating NK cell receptors, scientists generated a new type of chimeric Ag receptor targeting the prostate stem cell Ag (PSCA). Infused YTS cells armed with anti-PSCA-DAP12 caused delayed tumor xenograft growth and resulted in complete tumor eradication in a significant fraction of treated mice. [J Immunol] Abstract

Tolerance of Activated Pathogenic CD4+ T Cells by Transcriptional Targeting of Dendritic Cells
The authors recently showed that targeted expression of myelin oligodendrocyte glycoprotein to dendritic cells with self-inactivating-lentivirus vectors induces antigen-specific tolerance in naive antigen-specific CD4+ T cells and protects mice from experimental autoimmune encephalomyelitis (EAE). They now demonstrate that this approach also induces tolerance of activated antigen-specific CD4+ T cells and completely protects mice from passive EAE induction. [Gene Ther] Abstract

Dental Pulp Stem Cells: A New Cellular Resource for Corneal Stromal Regeneration
Researchers report that adult dental pulp cells (DPCs) isolated from third molars have the capability to differentiate into keratocytes, cells of the corneal stoma. After injection in vivo into mouse corneal stroma, human DPCs produced corneal stromal extracellular matrix containing human type I collagen and keratocan and did not affect corneal transparency or induce immunological rejection. [Stem Cells Transl Med] Abstract | Press Release

Innovative Delivery of siRNA to Solid Tumors by Super Carbonate Apatite
Investigators introduce an in vivo pH-sensitive delivery system for small interfering RNA (siRNA) using super carbonate apatite nanoparticles, which is the smallest class of nanocarrier. [PLoS One] Full Article

Enhanced Engraftment and Repairing Ability of Human Adipose-Derived Stem Cells, Conveyed by Pharmacologically Active Microcarriers Continuously Releasing HGF and IGF-1, in Healing Myocardial Infarction in Rats
Rats with four-week-old myocardial infarction were injected in the border zone with human adipose-derived stem cells conveyed by poly(lactic-co-glycolic acid) microcarriers releasing hepatocyte growth factor (HGF) and insulin-like growth factor-1 (IGF-1). [J Biomed Mater Res A] Abstract

Double-Blind, Placebo-Controlled Study of HGF Gene Therapy in Diabetic Neuropathy
Investigators aimed to evaluate the safety and efficacy of a plasmid containing two human hepatocyte growth factor isoforms given by intramuscular injections in patients with painful diabetic neuropathy. [Ann Clin Transl Neurol] Full Article | Press Release

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In Vivo Reprogramming for Tissue Repair
The authors discuss recent breakthroughs in regenerating pancreatic β-cells and neurons from various cell types, and highlight fundamental challenges that need to be overcome for the translation of in vivo lineage reprogramming into therapy. [Nat Cell Biol] Abstract

Aptamer Nanomedicine for Cancer Therapeutics: Barriers and Potential for Translation
The authors first introduce the recent progress in clinical trials of aptamer nanomedicine, followed by discussion of the barriers at the design and in vivo application stages. [ACS Nano] Abstract

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

Learn More: UM729, a Novel Small Molecule for Ex Vivo Expansion of Human HSCs
NeoStem PreSERVE AMI Clinical Trial One-Year Results to be Presented
NeoStem, Inc. announced that the latest available information from continued follow-up for its PreSERVE study will be presented. The PreSERVE study is NeoStem’s clinical trial evaluating NBS10, which is being developed to treat damaged heart muscle following an acute myocardial infarction. [Press release from NeoStem, Inc. (GlobeNewswire Inc.) discussing research to be presented at the American College of Cardiology’s 64th Annual Scientific Session and Expo, San Diego] Press Release

Pluristem to Present Data at Regenerative Medicine Conferences in Japan and South Korea
Pluristem Therapeutics Inc. announced that members of its executive and scientific teams will present at two key regenerative medicine conferences in Asia. [Press release Pluristem Therapeutics Inc. discussing research to be presented at the 14th Congress of the Japanese Society for Regenerative Medicine, Yokohama and BIT’s 8th Annual World Congress of Regenerative Medicine & Stem Cell 2015, Busan] Press Release

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In Japan, Embattled RIKEN Chief to Step Down
Ryoji Noyori plans to resign as president of RIKEN, the network of Japanese national labs that has spent much of the past year embroiled in a fraud scandal, news outlets report. [ScienceInsider] Editorial

Europe’s Human Brain Project Needs Urgent Reforms, Panel Says
The Human Brain Project, a humongous, controversial research project backed by the European Union, must reform to stay on course, a review panel has recommended and it must do so fast. A summary of the panel’s report, published by the European Commission, says a series of “corrective actions” needs to be taken in HBP’s governance, the way it collaborates, and its communication. [ScienceInsider] Editorial | Download Report

Harper Government Supports Innovation in Immunotherapy Treatments
The Honourable Michelle Rempel, Minister of State for Western Economic Diversification, announced funding of $2,167,000 to The Centre for Drug Research and Development (CDRD) to support them in the development and commercialization of innovative treatments for human disease in the field of immunotherapy. [The Centre for Drug Research and Development] Press Release
CIRM-Funded Clinical Trial Aimed at Blocking HIV/AIDS in People Gets the Go Ahead
An innovative therapy using a patient’s own stem cells, modified to resist infection with the AIDS virus, has been given approval by the Food and Drug Administration to begin a clinical trial in people. CIRM, California’s stem cell agency, is funding that trial. [California Institute for Regenerative Medicine] Press Release

The Bill & Melinda Gates Foundation and CureVac Collaborate to Accelerate the Development of Transformative Vaccine Technology
The Bill & Melinda Gates Foundation and CureVac announced that the foundation has made a commitment to invest $52 million in CureVac. As part of the agreement, the foundation will also provide separate funding for several projects to develop prophylactic vaccines based on CureVac’s proprietary mRNA platform. [Bill & Melinda Gates Foundation] Press Release

Bristol-Myers Squibb Signs Exclusive Agreement with Bavarian Nordic for PROSTVAC®, a Prostate-Specific Antigen-Targeting Cancer Immunotherapy
Bavarian Nordic and Bristol-Myers Squibb Company announced an agreement that provides Bristol-Myers Squibb an exclusive option to license and commercialize PROSTVAC®, Bavarian Nordic’s investigational Phase III prostate-specific antigen-targeting cancer immunotherapy in development for the treatment of asymptomatic or minimally symptomatic metastatic castration-resistant prostate cancer. [Bristol-Myers Squibb Company]
Press Release

Athersys and Chugai Enter License Agreement and Collaboration to Develop MultiStem® Cell Therapy for Ischemic Stroke in Japan
Athersys, Inc. and Chugai Pharmaceutical Co., Ltd. have announced a partnership and license agreement to exclusively develop and commercialize MultiStem® cell therapy for ischemic stroke in Japan. [Athersys, Inc.] Press Release

Celimmune Announces Anti-IL-15 Antibody Licensing and Option Agreements with Amgen
Celimmune LLC announced that it has licensed a Phase II-stage, anti-IL-15 monoclonal antibody (AMG 714) from Amgen. Under the provisions of this exclusive license agreement, Celimmune has the rights to develop, manufacture and commercialize AMG 714 on a worldwide basis excluding Japan. [Celimmune LLC (PR Newswire Association LLC)] Press Release

Orgenesis Acquires Cell Therapy Manufacturer MaSTherCell
Orgenesis Inc. announced the company’s plan to increase its global presence and capabilities by acquiring MaSTherCell, an emerging pioneer in the industrialization of cell-based therapeutics. [Orgenesis Inc. (Marketwire L.P.)] Press Release

Arrowhead Acquires Novartis’ RNAi Research and Development Portfolio
Arrowhead Research Corporation announced the acquisition of Novartis’ entire RNAi research and development portfolio and associated assets. [Arrowhead Research Corporation (Business Wire)] Press Release

FDA Approves Opdivo (Nivolumab) for the Treatment of Patients with Previously Treated Metastatic Squamous Non-Small Cell Lung Cancer
Bristol-Myers Squibb Company announced that the U.S. Food and Drug Administration (FDA) has approved Opdivo (nivolumab) injection, for intravenous use, for the treatment of patients with metastatic squamous non-small cell lung cancer with progression on or after platinum-based chemotherapy. [Bristol-Myers Squibb Company]
Press Release

arGEN-X Expands Preclinical Pipeline with ARGX-115: A Novel Simple Antibody™ for Cancer Immunotherapy
arGEN-X N.V. announced that it has exercised its option to exclusively license a first-in-class, preclinical therapeutic antibody candidate, now ARGX-115, to target GARP, a novel immune checkpoint with potential in cancer immunotherapy. [arGEN-X N.V.] Press Release
Center for Scientific Review Notice of Closed Meetings (FR Doc. No: 2015-05173)
Recall of Alere Determine HIV-1/2 Ag/Ab Combo Controls, PN 7D2628
Food and Drug Administration (United States)

Clinical Trial Imaging Endpoint Process Standards; Draft Guidance for Industry; Availability (FR Doc. No: 2015-05016)

Clinical Outcomes Assessment Development and Implementation: Opportunities and Challenges; Public Workshop (FR Doc. No: 2015-05017)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Current Good Manufacturing Practices and Related Regulations for Blood and Blood Components; and Requirements for Donor Testing, Donor Notification, and “Lookback” (FR Doc. No: 2015-04381)
NEW Regenerative Medicine Workshop at Hilton Head
May 13-16, 2015
Hilton Head Island, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Cell and Gene Therapy Formulation Scientist (Stelfox)

NEW Research Scientist – Cell Signaling in Normal and Leukemic Hematopoietic Stem and Progenitor Cells (Indiana University)

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 – Liver Cell Biology (STEMCELL Technologies Inc.)

Postdoctoral Position – Gene Therapy of Lysosomal Storage Diseases (Cincinnati Children’s Hospital Medical Center)

Associate Director, Center for Gene Therapy (City of Hope)

Assistant Professor – Neuroscience (Georgia State University)

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.)

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

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