Volume 16.27 | Jul 13

Newsletter Issue
Issue 16.27 July 13, 2015
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New Measurements Reveal Differences between Stem Cells for Treating Retinal Degeneration
By growing two types of stem cells in a “3-D culture” and measuring their ability to produce retinal cells, a team of researchers has found one cell type to be better at producing retinal cells. The research not only reveals which stem cell type might be better for treating retinal degeneration, but it also demonstrates a standardized method for quantifying the effectiveness of different stem cells for such therapies. [Press release from St. Jude Children’s Research Hospital discussing online publication in Cell Stem Cell] Press Release | Abstract | Graphical Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Tmc Gene Therapy Restores Auditory Function in Deaf Mice
To investigate gene therapy as a potential biologic strategy for restoration of auditory function in patients with genetic hearing loss, scientists tested a gene augmentation approach in mouse models of genetic deafness. [Sci Transl Med] Abstract | Press Release

PDGFB-Based Stem Cell Gene Therapy Increases Bone Strength in the Mouse
Transplantation of phosphoglycerate kinase (PGK)-PDGFB–transduced Sca1+ cells increased mesenchymal stem cell (MSC) proliferation, raising the possibility that PDGF-BB enhances expansion of MSCs in the vicinity of the hematopoietic niche where the osteogenic milieu propels the differentiation of MSCs toward an osteogenic destination. [Proc Natl Acad Sci USA] Abstract

3D Scaffolds with Different Stiffness but Same Microstructure for Bone Tissue Engineering
Scientists developed novel three-dimensional (3D) scaffolds with different degrees of stiffness, but the same 3D microstructure, that was maintained by using decellularized cancellous bone. [ACS Appl Mater Interfaces] Abstract

Effect of siRNA Pre-Exposure on Subsequent Response to siRNA Therapy
Investigators explored the possibility of therapeutic resistance against siRNA nanoparticles in human cancer cells. Two approaches to siRNA treatment were undertaken using lipid-modified polyethylenimines, a single high concentration and repeated increasing concentrations. [Pharm Res] Abstract

Suppression of Tumor Growth in Lung Cancer Xenograft Model Mice by Poly(Sorbitol-co-PEI)-Mediated Delivery of Osteopontin siRNA
To study in vivo tumor growth suppression, two lung cancer cell-xenograft mouse models were prepared and polysorbitol-based transporter/small interfering RNA (siRNA) targeting osteopontin complexes were delivered into the mice through intravenous injection. [Eur J Pharm Biopharm] Abstract

Enhanced Bone Tissue Regeneration by Porous Gelatin Composites Loaded with the Chinese Herbal Decoction Danggui Buxue Tang
Researchers investigated the efficacy of adding Danggui Buxue Tang (DBT) to bone substitutes on bone regeneration following bone injury. DBT was incorporated into porous composites made from genipin-crosslinked gelatin and β-triclacium phosphates as bone substitutes. [PLoS One] Full Article

Dual Silencing of Bcl-2 and Survivin by HSV-1 Vector Shows Better Antitumor Efficacy in Higher PKR Phosphorylation Tumor Cells In Vitro and In Vivo
RNA interference (RNAi) of oncogenes Bcl-2 and Survivin was combined with oncolytic HSV-1 and a new vector HSV010-BS was constructed. Transfected cell viability assays and animal experiments revealed that the dual silencing of Bcl-2 and Survivin improved the antitumor effect of oncolytic herpes simplex virus type 1 (HSV-1) in vitro and in vivo, while the antitumor effect was correlated with the phosphorylation levels of PKR of the tumor cells. [Cancer Gene Ther] Abstract

Adeno-Associated Virus Mediated Gene Transfer of Shepherdin Inhibits Gallbladder Carcinoma Growth In Vitro and In Vivo
Scientists designed and constructed a recombinant adeno-associated virus loading fusion gene NT4-TAT-6His-Shepherdin. The expression of Shepherdin in gallbladder carcinoma (GBC) cells was detected and its strong inhibitory effects against GBC growth were evaluated after AAV mediated gene transfer of Shepherdin into GBC cells and xenograft tumors. [Gene] Abstract

Induction of Angiogenesis by Matrigel Coating of VEGF-Loaded PEG/PCL-Based Hydrogel Scaffolds for hBMSC Transplantation
Biodegradable elastic hydrogels consisting of hydrophilic poly(ethylene glycol) (PEG) and hydrophobic poly(epsilon-caprolactone) (PCL) scaffolds were evaluated for tissue engineering because of its biocompatibility and the ability to control the release of bioactive peptides. [Mol Cells] Abstract

Webinar: New Tools for the Ex Vivo Expansion of Human Hematopoietic Stem and Progenitor Cells
Modified mRNA as an Alternative to Plasmid DNA (pDNA) for Transcript Replacement and Vaccination Therapy
The authors summarize the currently available strategies of mRNA modification to increase the therapeutic efficacy; they also highlight the recent improvements of mRNA delivery for in vivo applications of gene therapy. [Expert Opin Biol Ther] Abstract

Scaffolds and Tissue Regeneration: An Overview of the Functional Properties of Selected Organic Tissues
The authors provide an overview on the biomedical application of scaffolds and on the characteristics of the (bio)materials commonly used for manufacturing these biological devices used in tissue engineering, taking into consideration the cellular specificity of the target tissue. [J Biomed Mater Res B Appl Biomater] Abstract

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

Cryopreservation Media for Stem Cells - Learn More!
AXON Neuroscience’s Vaccine to Halt Alzheimer’s Finishes Phase I Clinical Trial
A vaccine offering new hope for Alzheimer’s disease patients, their physicians and caregivers has passed a Phase I safety trial. [Press release from AXON Neuroscience discussing research to be presented at the Alzheimer’s Association International Conference (AAIC), Washington, DC] Press Release

Roche’s Investigational Immunotherapy Atezolizumab Shrank Tumors in People with a Specific Type of Bladder Cancer
Roche announced that in the IMvigor 210 study, the investigational cancer immunotherapy atezolizumab (MPDL3280A; anti-PDL1) shrank tumors in people with locally advanced or metastatic urothelial bladder cancer who had progressed on initial treatment (second-line or later). [F. Hoffmann-La Roche Ltd] Press Release

Stem Cells & Regenerative Medicine Congress 2015
Use of Regulated Animals in U.S. Biomedical Research Falls to Lowest Levels on Record
The number of federally regulated animals used in U.S. biomedical research dropped last year to its lowest level since data collection began in 1972, according to new statistics posted by the U.S. Department of Agriculture. [ScienceInsider] Editorial

Russia’s Only Private Science Funder Closes Its Doors
The Dynasty Foundation, Russia’s only private funder of scientific research, announced its closure after being labeled a “foreign agent” by the Russian government. [ScienceInsider] Editorial

Southern California Schools in Legal Clash Over Alzheimer’s Grant
In an unusual dispute, the University of California, San Diego, is suing a former faculty member and rival school, the University of Southern California in Los Angeles, for allegedly conspiring to take over a federally funded Alzheimer’s disease study. [ScienceInsider] Editorial

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apceth and University of Cologne to Join Forces on Combination Cellular Immunotherapies for Cancer
apceth announced a broad partnership with the Center for Molecular Medicine Cologne, University of Cologne, to combine technologies and expertise, on the development of immunotherapies for solid tumors and hematological malignancies. [apceth GmbH & Co. KG ] Press Release

Sorrento and NantBioScience Form Joint Venture to Develop Moonshot Program Targeted Small Molecule Therapies against Cancer
Sorrento Therapeutics, Inc. and NantBioScience, Inc. announced that they have established a joint venture to focus on the development of ‘first-in-class’ small molecules against targets that have eluded the pharmaceutical industry to date and which may address important drivers of cancer growth including cancer stem cells. [Sorrento Therapeutics, Inc.] Press Release

UC San Diego and GSK Collaborate to Eradicate Cancer Stem Cells, Treat Leukemia
Researchers at the University of California, San Diego School of Medicine and Moores Cancer Center are working with GSK on a bench-to-bedside project to treat leukemia and other diseases by eliminating cancer stem cells. [University of California, San Diego] Press Release

Ludwig Cancer Research and University of Oxford Launch Cancer Immunotherapy Spinout
Isis Innovation, the University of Oxford’s technology commercialization company, and Ludwig Cancer Research announced the launch of a new spinout company, iOx Therapeutics. iOx Therapeutics will develop a novel cancer immunotherapy discovered through a collaboration between Ludwig Cancer Research and Professor Vincenzo Cerundolo, the director of the MRC Human Immunology Unit within the University of Oxford’s Weatherall Institute of Molecular Medicine. [Ludwig Cancer Research] Press Release

St. Jude to Receive $12 Million in Grants from NIH
Investigators at St. Jude Children’s Research Hospital have received grants totaling more than $12 million from the National Institutes of Health’s Pharmacogenomics Research Network for a new Center for Precision Medicine in Leukemia and for the Clinical Pharmacogenetics Implementation Consortium. [St. Jude Children’s Research Hospital] Press Release

Imbruvica® (Ibrutinib) Now Approved to Treat Waldenstrom’s Macroglobulinemia in Europe
AbbVie announced the European Commission granted marketing authorization for Imbruvica® (ibrutinib) as the first treatment option available in all 28 member states of the European Union for the treatment of Waldenstrom’s macroglobulinemia, a rare, slow growing blood cancer, in adult patients who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo-immunotherapy. [AbbVie Inc.] Press Release

Benitec Acquires Full Rights to Hepatitis B Program
Benitec Biopharma and China-based Biomics Biotechnologies announced Benitec has acquired the full rights to the pre-clinical ddRNAi-based Hepatitis B therapeutic program, Hepbarna™, which was previously under development as a joint venture between the two companies. [Benitec Biopharma Limited] Press Release

Adaptimmune Announces FDA Acceptance of Investigational New Drug (IND) Application for MAGE-A10 T in Patients with Non-Small Cell Lung Cancer
Adaptimmune announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s IND application for autologous genetically modified T-cells expressing enhanced T cell receptors specific for MAGE A10 (MAGE-A10 T) in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC), and that the IND is now active. [Adaptimmune Therapeutics plc] Press Release

Cancer Patients Treated in World-First Clinical Trial of Canadian Viral Therapy
Canadian researchers have launched the world’s first clinical trial of a novel investigational therapy that uses a combination of two viruses to attack and kill cancer cells, and stimulate an anti-cancer immune response. [Ontario Institute for Cancer Research] Press Release
Government-Owned Inventions; Availability for Licensing (FR Doc. No: 2015-16838)
DSCSA Implementation: Product Tracing Requirements for Dispensers — Compliance Policy Guidance for Industry
Food and Drug Administration (United States)

Meetings With the Office of Orphan Products Development; Guidance for Industry, Researchers, Patient Groups, and Food and Drug Administration Staff; Availability (FR Doc. No: 2015-16773)

Permanent Discontinuance or Interruption in Manufacturing of Certain Drug or Biological Products (FR Doc. No: 2015-16659)

Qualification of Biomarker-Plasma Fibrinogen in Studies Examining Exacerbations and/or All-Cause Mortality for Patients With Chronic Obstructive Pulmonary Disease; Draft Guidance for Industry; Availability (FR Doc. No: 2015-16563)
NEW 4th International Conference on Immunotherapy in Pediatric Oncology (CIPO2015)
September 25-26, 2015
Seattle, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW PhD Studentship – Veterinary and Animal Sciences (University of Milan)

NEW Postdoctoral Research Fellow – Fat Metabolism and Stem Cells (Singapore Bioimaging Consortium)

Postdoctoral Research Associate – Breast Stem Cell Biology (University of Miami Miller School of Medicine)

Aseptic Manufacturing Associate (Lonza Biologics, Inc.)

Postdoctoral Position – Translational Immunology (University of Utah School of Medicine)

Postdoctoral Position – Splicing in Hematopoiesis (Northwestern University)

Primary Cell Transduction Process Scientist (GlaxoSmithKline)

Postdoctoral Fellow – Antibody Engineering/Immuno-Oncology (California Institute for Biomedical Research)

Postdoctoral Fellow – Stem Cell Biology (INSERM)

Cell Therapy GMP Manager (Paramount Recruitment)

Postdoctoral Fellow – Neural Stem Cells (Children’s Hospital of Philadelphia)

Lead Device and Delivery Scientist (Cell Therapy Catapult)

Postdoctoral Fellow – Immune Reconstitution in Hematopoietic Stem Cell Transplantation (Stanford University)

Process Facilitator (Opexa Therapeutics)

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