Volume 16.10 | Mar 16

Newsletter Issue
Issue 16.10 March 16, 2015
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Repairing the Cerebral Cortex: It Can Be Done
Researchers have just taken an important step in the area of cell therapy: repairing the cerebral cortex of the adult mouse using a graft of cortical neurons derived from embryonic stem cells. [Press release from INSERM discussing online prepublication in Neuron] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Tetanus Toxoid and CCL3 Improve Dendritic Cell Vaccines in Mice and Glioblastoma Patients
Scientists showed that pre-conditioning the vaccine site with a potent recall antigen such as tetanus/diphtheria toxoid can significantly improve the lymph node homing and efficacy of tumor-antigen-specific dendritic cells. [Nature] Abstract | Press Release

Mutational Landscape Determines Sensitivity to PD-1 Blockade in Non-Small Cell Lung Cancer
To unravel the genomic determinants of response to immune checkpoint inhibitors, researchers used whole-exome sequencing of non-small cell lung cancers treated with pembrolizumab, an antibody targeting programmed cell death-1 (PD-1). [Science] Abstract | Press Release | Video

Immunological Mechanisms of the Antitumor Effects of Supplemental Oxygenation
Scientists showed that hyperoxia stimulates enhanced intratumoral infiltration and reduced inhibition of endogenously developed or adoptively transferred tumor-reactive CD8 T cells, increased proinflammatory cytokines and decreased immunosuppressive molecules, such as transforming growth factor–β, weakened immunosuppression by regulatory T cells, and improved lung tumor regression and long-term survival in mice. [Sci Transl Med] Abstract | Press Release

Liver-Directed Lentiviral Gene Therapy in a Dog Model of Hemophilia B
Researchers investigated the efficacy of liver-directed gene therapy using lentiviral vectors in a large animal model of hemophilia B and evaluated the risk of insertional mutagenesis in tumor-prone mouse models. [Sci Transl Med] Abstract | Press Release

Haploidentical Hematopoietic Transplantation from KIR Ligand-Mismatched Donors with Activating KIRs Reduces Non-Relapse Mortality
As activating killer cell immunoglobulin-like receptors (KIRs) are heterogeneously expressed in the population, researchers investigated the role of donor activating KIRs in haploidentical hematopoietic transplants for acute leukemia. [Blood] Abstract

Mesenchymal Stem/Stromal Cells Protect against Autoimmunity via CCL2-Dependent Recruitment of Myeloid-Derived Suppressor Cells
Researchers report a novel mechanism by which mesenchymal stem/stromal cells modulate the immune system by recruiting myeloid-derived suppressor cells in a mouse model of experimental autoimmune uveitis. [J Immunol] Abstract

Shedding of Clinical-Grade Lentiviral Vectors Is Not Detected in a Gene Therapy Setting
Scientists investigated factors influencing the extent of lentiviral vector shedding upon ex vivo transduction of human hematopoietic stem and progenitor cells. [Gene Ther] Abstract

Hematopoietic Stem Cell Gene Therapy Corrects the Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II
Investigators showed that lentiviral isogeneic hematopoietic stem cell gene therapy corrects the neuronal manifestation through the amelioration of lysosomal storages and autophagic dysfunctions in brains from mucopolysaccharidosis type II mice. [Hum Gene Ther] Abstract

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Gene Therapy of Inherited Retinal Degenerations: Prospects and Challenges
The authors summarize the strategies that are currently being exploited to overcome challenges and drive the clinical development of retinal gene therapy. [Hum Gene Ther] Abstract

Beyond Consolidation: Auto-SCT and Immunotherapy for Plasma Cell Myeloma
The authors discuss the preclinical data supporting immunotherapy in autologous hematopoietic cell transplantation (auto-SCT) for myeloma, clinical investigation of these strategies and the future prospects of immunotherapy in pursuit of the goal of curative therapy. [Bone Marrow Transplant] Abstract

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

View Webinar: Genome Editing: From Modeling Disease to Novel Therapeutics
Alnylam Presents Results from Natural History Study of Patients with Familial Amyloidotic Cardiomyopathy (FAC) and Presents Complete Results from Phase II Clinical Trial for Revusiran, an Investigational RNAi Therapeutic for the Treatment of FAC
Alnylam Pharmaceuticals, Inc. announced results from a retrospective natural history study evaluating disease progression in transthyretin-mediated amyloidosis patients with FAC. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the American College of Cardiology (ACC) Annual Scientific Session, San Diego] Press Release

RXi Pharmaceuticals to Present at the 73rd Annual Meeting of the American Academy of Dermatology
RXi Pharmaceuticals Corporation announced that the company’s Dr. Lyn Libertine will present two posters that will provide an overview of the company’s first clinical compound, RXI-109, a self-delivering RNAi compound currently being evaluated in Phase IIa clinical trials. [Press release from RXi Pharmaceuticals Corporation discussing research to be presented at the 73rd Annual Meeting of the American Academy of Dermatology (AAD), San Francisco] Press Release

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In a Turnabout, Key Congressional Critic Backs NSF Peer Review
A new statement from Representative Lamar Smith, the chair of the science committee in the U.S. House of Representatives that oversees National Science Foundation (NSF), appears to be a significant softening of his long-standing criticism of NSF’s grantmaking process. Although a different congressional panel is expected to register the same complaint against the agency at a hearing, the shift in the political landscape is good news for U.S. scientists. [ScienceInsider] Editorial

Don’t Edit Embryos, Researchers Warn
Scientists should refrain from studies that alter the genome of human embryos, sperm, or egg cells, researchers warn in a commentary published in Nature. In it, they sound the alarm about new genome-editing techniques known as CRISPR and zinc-finger nucleases that make it much easier for scientists to delete, add, or change specific genes. [ScienceInsider] Editorial | Commentary
The Alliance for Regenerative Medicine Enters into Memorandum of Understanding with Japan’s Forum for Innovative Regenerative Medicine
The Alliance for Regenerative Medicine announced it has entered into a memorandum of understanding with Japan’s Forum for Innovative Regenerative Medicine, effective immediately. [The Alliance for Regenerative Medicine] Press Release

Cell Medica Collaborates with Cell Therapy Catapult to Help Bring Its New Treatment to NHS
The Cell Therapy Catapult and Cell Medica announced a collaboration to accelerate NHS adoption of Cell Medica’s Cytovir™ CMV. Cell Medica is a leading UK cellular immunotherapy company while the Cell Therapy Catapult is focused on the development of the UK cell therapy industry to increase the nation’s health and wealth. [Cell Medica Limited] Press Release

New Immunotherapy Drug ‘Fast-Tracked’ for Melanoma Patients
A new immunotherapy drug, pembrolizumab, has become the first treatment ‘fast-tracked’ for NHS patients with advanced melanoma, under a new Government scheme. Clinical trials have shown that it has the potential to ease symptoms and extend survival. [Cancer Research UK] Press Release

Nightstar Receives U.S. and European Orphan Drug Designation for Gene Therapy to Treat Choroideremia
NightstaRx Ltd has received both U.S. Food and Drug Administration and European Medicines Agency Orphan Drug Designation for its lead program, a gene therapy to treat choroideremia, an X-linked recessive disorder that leads to progressive blindness. [NightstaRx Limited] Press Release

VAXIL Receives EU Orphan Drug Designation for ImMucin for the Treatment of Multiple Myeloma
Vaxil Bio reports that its lead drug candidate, ImMucin has received orphan drug designation from the European Medicines Agency of the European Commission for the treatment of multiple myeloma. [Vaxil Bio Ltd.] Press Release

First FDA Breakthrough Therapy Designation for KLH-Conjugate Immunotherapy Signals Key Step for Novel Carrier Molecule
Stellar Biotechnologies, Inc. noted that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for a Keyhole Limpet Hemocyanin (KLH)-based immunotherapy compound in the treatment of adult patients with EGFRvIII-positive glioblastoma being developed by Celldex Therapeutics, Inc. [Stellar Biotechnologies, Inc.] Press Release

Neuralstem Announces Topline Results of Phase II ALS Trial
Neuralstem, Inc. announced top line data from the Phase II trial of NSI-566 spinal cord-derived neural stem cells under development for the treatment of amyotrophic lateral sclerosis (ALS). The study met primary safety endpoints. Secondary efficacy endpoints at nine months post-surgery indicate a 47% response rate to the stem cell treatment. [Neuralstem, Inc.] Press Release

WuXi PharmaTech to Build New Cell Therapy Manufacturing Facility for CAR T Cells and Other Cancer Immunotherapies
WuXi PharmaTech (Cayman) Inc. announced that it will soon begin construction of a new, 145,000-square-foot cGMP facility in Philadelphia for the manufacture of cell therapy products. [WuXi PharmaTech (Cayman) Inc.] Press Release

Two Penn Medicine Gene Therapy Researchers Receive Pennsylvania Bio Awards
Two researchers from the Perelman School of Medicine at the University of Pennsylvania will be honored for their contributions for the burgeoning field of gene therapy by Pennsylvania Bio. [Perelman School of Medicine at the University of Pennsylvania] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-05566)
ALBAcyte C3 Coated Red Blood Cells
Food and Drug Administration (United States)

Agency Information Collection Activities; Proposed Collection; Comment Request; Postmarketing Adverse Drug Experience Reporting and Recordkeeping Biological Products (FR Doc. No: 2015-05591)

Formal Meetings Between the Food and Drug Administration and Sponsors or Applicants of Prescription Drug User Fee Act Products; Draft Guidance for Industry; Availability (FR Doc. No: 2015-05523)
NEW 47th Annual Meeting of the European Pancreatic Club (EPC) 2015
June 24-26, 2015
Toledo, Spain

NEW 2015 World Stem Cell Summit (WSCS15)
December 10-12, 2015
Atlanta, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Manufacturing Manager – Cell Therapy (Opexa Therapeutics)

NEW Postdoctoral Positions – Developmental Biology (Joslin Diabetes Center)

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.)

Cell and Gene Therapy Formulation Scientist (Stelfox)

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

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)

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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