Volume 16.13 | Apr 6

Newsletter Issue
Issue 16.13 April 6, 2015
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Team Identifies Highly Efficient New Cas9 for In Vivo Genome Editing
Researchers have identified a highly efficient Cas9 nuclease that overcomes one of the primary challenges to in vivo genome editing. The Cas9 nuclease from the bacteria Staphylococcus aureus is 25% smaller than the Cas9 enzyme from Streptococcus pyogenes. [Press release from The Broad Institute discussing online publication in Nature] Press Release | Abstract
View Webinar: Genome Editing: From Modeling Disease to Novel Therapeutics
PUBLICATIONS (Ranked by impact factor of the journal)
A Dendritic Cell Vaccine Increases the Breadth and Diversity of Melanoma Neoantigen-Specific T Cells
Investigators showed that a dendritic cell vaccine increased naturally occurring and revealed new HLA class I-restricted neoantigens in patients with advanced melanoma. [Science] Abstract | Press Release

An Anti-B Cell Maturation Antigen Bispecific Antibody for Multiple Myeloma
Researchers report a bispecific antibody against B cell maturation antigen (BiFab-BCMA), which potently and specifically redirects T cells to lyse malignant multiple myeloma cells. The in vitro and in vivo activity of BiFab-BCMA was comparable to that of anti-BCMA chimeric antigen receptor T cell therapy, for which two clinical trials have recently been initiated. [J Am Chem Soc] Abstract

Enriching a Cellulose Hydrogel with a Biologically Active Marine Exopolysaccharide for Cell-Based Cartilage Engineering
Scientists evaluated the potential of hydroxypropyl methylcellulose hydrogel/GY785 associated with rabbit articular chondrocytes to form cartilaginous tissue in vivo by transplantation into the subcutis of nude mice for three weeks. [J Tissue Eng Regen Med] Abstract

Use of Ultrasound-Targeted Microbubble Destruction (UTMD) to Transfect IGF-1 cDNA to Enhance the Regeneration of Rat Wounded Achilles Tendon In Vivo
Investigators aimed to determine whether using UTMD to transfect rat wounded Achilles tendon with IGF-1 cDNA would enhance tissue regeneration. The IGF-1 cDNA expression of the Achilles tendons was evaluated by histological adhesion finding, qRT-PCR examination and biomechanical test. [Gene Ther] Abstract

Adenovirus-Mediated Artificial MicroRNAs Targeting Matrix or Nucleoprotein Genes Protect Mice against Lethal Influenza Virus Challenge
The authors describe a novel strategy for treatment of influenza based on RNAi delivered by a replication-defective adenovirus vector, derived from chimpanzee serotype 68 (AdC68). Their results demonstrated that prophylactic treatment with AdC68 expressing amiRNAs directed against M1, M2 or NP genes of influenza virus completely protected mice from homologous A/PR8 virus challenge and partially protected the mice from heterologous influenza A virus strains such as H9N2 and H5N1. [Gene Ther] Abstract

Angiogenic Role of MMP-2/9 Expressed on the Cell Surface of Early Endothelial Progenitor Cells/Myeloid Angiogenic Cells
In order to elucidate the angiogenic properties of myeloid angiogenic cells (MACs)/endothelial progenitor cells, scientists clarified the characteristics of MACs as compared to M2 macrophages. MACs caused an increase in vessel formation in in vivo models through the production of IL-8. [J Cell Physiol] Abstract

Intra-Articular Transplantation of Atsttrin-Transduced Mesenchymal Stem Cells Ameliorate Osteoarthritis Development
Investigators developed genetically modified mesenchymal stem cells (MSCs) that expressed recombinant Atstrrin (MSC-Atsttrin). Osteoarthritis animal models were induced by anterior cruciate ligament transection, and MSC-GFP/Atsttrin were injected into the articular cavity one week postsurgery. [Stem Cells Transl Med] Abstract

Telomerase Inhibition Decreases Alpha-Fetoprotein Expression and Secretion by Hepatocellular Carcinoma Cell Lines: In Vitro and In Vivo Study
Researchers investigated the effect of telomerase inhibition on alpha-fetoprotein (AFP) secretion and the involvement of the PI3K/Akt/mTOR signaling pathway. The in vivo effect of costunolide on AFP production was tested in NSG mice. [PLoS One] Full Article

FREE Wallchart: Glioblastoma Multiforme
Adoptive Cell Transfer as Personalized Immunotherapy for Human Cancer
Adoptive cell therapy (ACT) using naturally occurring tumor-reactive lymphocytes has mediated durable, complete regressions in patients with melanoma, probably by targeting somatic mutations exclusive to each cancer. These results have expanded the reach of ACT to the treatment of common epithelial cancers. [Science]
Abstract | Press Release

The Future of Immune Checkpoint Therapy
Immune checkpoint therapy, which targets regulatory pathways in T cells to enhance antitumor immune responses, has led to important clinical advances and provided a new weapon against cancer. This therapy has elicited durable clinical responses and, in a fraction of patients, long-term remissions where patients exhibit no clinical signs of cancer for many years. [Science] Abstract | Press Release

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

Enter to Win a Travel Support Award to ISSCR2015!
Tekmira Presents Preclinical Results Demonstrating Super-Additive Effects on Plasma Triglyceride Lowering by Silencing of ApoC3 and ANGPTL3 Genes
Tekmira Pharmaceuticals Corporation announced that new preclinical data on TKM-HTG, an RNAi therapeutic, were presented. TKM-HTG is a product candidate in Tekmira’s non-hepatitis B virus product pipeline for the treatment of hypertriglyceridemia (HTG). [Press release from Tekmira Pharmaceuticals Corporation discussing research presented at the Keystone Symposia Conference: Liver Metabolism and Nonalchoholic Fatty Liver Diseases, Whistler] Press Release | Poster

Immunotherapy Delays Recurrence for Stage III and IV Ovarian Cancers
An experimental immunotherapy is in the works that can target an individual woman’s tumor and extend the time period between initial treatment and the cancer’s return. [Press release from the Society of Gynecologic Oncology (SGO) discussing research presented at the SGO Annual Meeting on Women’s Cancer, Chicago] Press Release

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UK Clarifies Controversial Media Restrictions on Scientists
A controversial policy change that seemed to deter UK scientists in government laboratories from talking to the media is not as far-reaching as it at first appeared — although the issue is still mired in confusion. [Nature News] Editorial

New RIKEN Chief Pledges to Restore Public Faith in Japanese Lab System
On his first day on the job as the new president of RIKEN Hiroshi Matsumoto pledged to follow through on his predecessor’s plans for addressing shortcomings that created an environment for research misconduct. [ScienceInsider] Editorial
Syndax and Merck to Collaborate on Immuno-Oncology Study Evaluating Entinostat in Combination with KEYTRUDA® (Pembrolizumab) in Lung Cancer and Melanoma
Syndax Pharmaceuticals, Inc., and Merck announced that they have entered into a clinical trial collaboration to evaluate the safety and efficacy of combining Syndax’s entinostat, an investigational epigenetic therapy, with Merck’s KEYTRUDA® (pembrolizumab), the first anti-PD-1 therapy approved in the United States. [Merck & Co., Inc.] Press Release

Advaxis and RTOG Foundation to Collaborate on Pivotal Phase II/III Clinical Trial of ADXS-HPV in Anal Cancer
Advaxis, Inc. announced that it is entering into a clinical trial collaboration agreement with the Radiation Therapy Oncology Group (RTOG) Foundation to evaluate the safety and efficacy of Advaxis’s lead cancer immunotherapy, ADXS-HPV (ADXS11-001), in a pivotal Phase II/III anal cancer trial, which will be run by NRG Oncology. [Advaxis, Inc.] Press Release

Novartis Accelerates Cancer Immunotherapy Efforts with Aduro Biotech Alliance and Launch of New Immuno-Oncology Research Group
Novartis announced that it is stepping up its efforts to harness the body’s immune system to combat cancer. The company has entered into a major multiyear alliance with Aduro Biotech that is focused on the discovery and development of next generation cancer immunotherapies targeting the stimulator of interferon genes pathway and launched a new immuno-oncology research group. [Novartis AG] Press Release

Four Biomedical Research Centers in Barcelona Join Forces to Bridge the Gap between Investigation and Clinical Practice
The Centre for Genomic Regulation, the Institute for Research in Biomedicine, the August Pi i Sunyer Biomedical Research Institute, and the Vall d’Hebron Research Institute have designed a training program called “PhD for Medical Doctors”. This is a pioneering initiative seeking to speed up the transfer of results generated by biomedical research activities in Spain to clinical practice. [Institute for Research in Biomedicine] Press Release

Fujifilm Holdings to Acquire Cellular Dynamics International, Inc.
FUJIFILM Holdings Corporation and Cellular Dynamics International, Inc. (CDI) announced that the two companies have entered into a definitive agreement whereby Fujifilm will acquire CDI via an all-cash tender offer to be followed by a second step merger. [Cellular Dynamics International, Inc.] Press Release

£25.7 Million to Create Research Powerhouse
A £25.7 million funding boost will advance biological research and aid the quest for new therapies to treat damaged tissues. The investment by the UK Government will enable the University of Edinburgh to create a new biology complex with world-leading laboratory space for 350 researchers and to establish a new Centre for Tissue Repair. [The University of Edinburgh] Press Release

Immunomic Therapeutics, Inc. Awarded SBIR Grant From NIH for Nanoplasmid Peanut Allergy Vaccine Development
Immunomic Therapeutics, Inc. announced that the National Institutes of Health has awarded ITI’s Small Business Innovation Research (SBIR) grant application titled, “Development of Nanoplasmid LAMP-based Peanut Allergy Immunotherapy.” [Immunomic Therapeutics, Inc. (GlobeNewswire, Inc.)] Press Release

NIH Funds Study of Fully Personalized Immunotherapy AGS-004 Combined with a Latency Reversing Therapy for the Treatment of HIV
Argos Therapeutics, Inc. announced that the NIH Division of AIDS has approved $6.6 million in funding for an investigator-initiated Phase IIa adult eradication study of AGS-004, the company’s investigational fully personalized immunotherapy for HIV. [Argos Therapeutics, Inc.] Press Release

Neurotech Announces FDA Acceptance of Investigational New Drug Application and Clinical Trial Initiation of Novel Therapy for Wet AMD
Neurotech Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted and communicated Neurotech’s ability to proceed with its Investigational New Drug application to conduct a Phase II clinical study of NT-503 encapsulated cell therapy for the treatment of recurrent subfoveal choroidal neovascularization secondary to age related macular degeneration (wet AMD). [Neurotech Pharmaceuticals, Inc.] Press Release

Clinical Trial Uses Patients’ Own Cells for Personalized Treatment after Bone Marrow Transplant
An innovative clinical trial using the science of “personalized” cellular therapy has begun enrolling children and adults suffering from graft-versus-host-disease (GvHD). By infusing large doses of personalized bone marrow cells into bone marrow transplant recipients, the physician-researchers aim to target sites of inflammation, potentially reducing GvHD in the intestine, liver and skin and limiting long-term organ damage. [Emory University] Press Release
National Institute of Neurological Disorders and Stroke; Notice of Closed Meeting (FR Doc. No: 2015-07342)
Annual Summary for Fiscal Year 2014
Food and Drug Administration (United States)

Society of Clinical Research Associates-Food and Drug Administration Clinical Trial Requirements, Regulations, Compliance and Good Clinical Practice (FR Doc. No: 2015-07810)
NEW British Society for Gene & Cell Therapy (BSGCT) Conference 2015
June 9-11, 2015
Glasgow, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Research Scientist – Cell and Gene Therapy Group (Biogen Idec)

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

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Lecturer – Gene Therapy (University College London)

Postdoctoral Position – Cancer Imaging (King’s College London)

Research Associate – Cell Culture Specialist (Tisch MS Research Center of New York)

Manufacturing Manager – Cell Therapy (Opexa Therapeutics)

Postdoctoral Positions – Developmental Biology (Joslin Diabetes Center)

Cell and Gene Therapy Formulation Scientist (Stelfox)

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

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)

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