Volume 16.11 | Mar 23

Newsletter Issue
Issue 16.11 March 23, 2015
Cell Therapy News - Your Industry in an Instant
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Researchers Tweak the Immune System to Target Cells Bearing Tumor Antigens
Researchers have succeeded in generating cells of the immune system to specifically target and destroy cancer cells. They developed human T cell receptors that have no tolerance toward human cancer antigens and specifically recognize the antigen MAGE-A1, which is present on various human tumor cells. [Press release from the Max Delbrück Center for Molecular Medicine discussing online publication in Nature Biotechnology] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Improved Antitumor Activity of Immunotherapy with BRAF and MEK Inhibitors in BRAFV600E Melanoma
Using a mouse model of syngeneic BRAFV600E-driven melanoma, SM1, scientists tested whether addition of the MEK inhibitor trametinib would enhance the antitumor activity of combined immunotherapy with the BRAF inhibitor dabrafenib. [Sci Transl Med] Abstract | Press Release

A Synthetic Erectile Optogenetic Stimulator Enabling Blue-Light-Inducible Penile Erection
An erectile optogenetic stimulator (EROS), a synthetic designer guanylate cyclase producing a blue-light-inducible surge of the second messenger cyclic guanosine monophosphate in mammalian cells, enabled blue-light-dependent penile erection associated with occasional ejaculation after illumination of EROS-transfected corpus cavernosum in male rats. [Angew Chem Int Ed Engl] Abstract | Press Release

Engraftment of Embryonic Stem Cells and Differentiated Progeny by Host Conditioning with Total Lymphoid Irradiation and Regulatory T Cells
Investigators aimed to establish tolerance or promote engraftment of transplanted embryonic stem cells (ESCs) as well as mature cell populations derived from ESCs. [Cell Rep] Full Article | Graphical Abstract

IL-12/15/18-Preactivated NK Cells Suppress GvHD in a Mouse Model of Mismatched Hematopoietic Cell Transplantation
Scientists showed that adoptively transferred IL-12/15/18-preactivated NK cells suppress graft versus host disease (GvHD) in a mouse model of fully mismatched hematopoietic cell transplantation. [Eur J Immunol] Abstract

Controlled Release of Vascular Endothelial Growth Factor from Spray-Dried Alginate Microparticles in Collagen-Hydroxyapatite Scaffolds for Promoting Vascularization and Bone Repair
Researchers developed a method to incorporate growth factor-eluting alginate microparticles into freeze-dried, collagen-based scaffolds. When implanted in vivo in a rat calvarial defect model, this scaffold enhanced vessel formation, resulting in increased bone regeneration compared to empty-defect and vascular endothelial growth factor-free scaffolds. [J Tissue Eng Regen Med] Abstract

Foamy Viral Vector Integration Sites in SCID-Repopulating Cells after MGMTP140K-Mediated In Vivo Selection
Human CD34+ human cord blood cells were transduced with an foamy virus vector encoding a methylguanine methyltransferase (MGMT)P140K transgene, transplanted into immunodeficient NOD/SCID IL2Rγnull mice, and selected in vivo for gene-modified cells. [Gene Ther] Abstract

Expression of an Engineered Soluble Coxsackievirus and Adenovirus Receptor by a Dimeric AAV9 Vector Inhibits Adenovirus Infection in Mice
Investigators analyzed the potential of a virus receptor trap based on a soluble coxsackievirus and adenovirus (Ad) receptor for inhibition of Ad infection. [Gene Ther] Abstract

Focused Ultrasound-Induced Blood-Brain Barrier Opening to Enhance Interleukin-12 Delivery for Brain Tumor Immunotherapy: A Preclinical Feasibility Study
Since focused ultrasound (FUS) has been confirmed to temporally and locally open the blood-brain barrier (BBB), investigators aimed to elucidate the possibility of combining FUS-induced BBB opening with interleukin-12 delivery to enhance the anticancer immunological response for glioma treatment. [J Transl Med] Abstract | Full Article

Tracking the Fate of Stem Cell Implants with Fluorine-19 MRI
Investigators used cellular magnetic resonance imaging (MRI) to detect mesenchymal stem cells labeled with a Fluorine-19 (19F) agent. 19F-MRI offers unambiguous detection and in vivo quantification of labeled cells. [PLoS One] Full Article

shRNA Off-Target Effects In Vivo: Impaired Endogenous siRNA Expression and Spermatogenic Defects
Using a reproductive homeobox 3 (Rhox3) short hairpin (sh) RNA with 100% complementarity to all eight Rhox3 paralogs, expressed from a CRE-regulated transgene, scientists successfully knocked down Rhox3 expression in male germ cells in vivo. [PLoS One] Full Article

Treating Diet-Induced Diabetes and Obesity with Human Embryonic Stem Cell-Derived Pancreatic Progenitor Cells and Antidiabetic Drugs
Exposure to high-fat diet (HFD) did not impact the maturation of macroencapsulated pancreatic progenitor cells into glucose-responsive insulin-secreting cells following transplantation, and the cell therapy improved glucose tolerance in HFD-fed transplant recipients after 24 weeks. [Stem Cell Reports] Full Article | Press Release

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Bladder Recovery by Stem Cell Based Cell Therapy in the Bladder Dysfunction Induced by Spinal Cord Injury: Systematic Review and Meta-Analysis
Researchers conducted a meta-analysis of urodynamic findings of experimental trials that included studies of stem cell-based cell therapy in spinal cord injury. [PLoS One] Full Article

Adeno-Associated Virus-Mediated Gene Therapy in Cardiovascular Disease
Significant progress in clinical translation of adeno-associated virus-mediated cardiac gene therapy has been achieved in recent years. This will prompt further clinical trials, and positive results could open a new era for cardiac gene therapy. [Curr Opin Cardiol] Abstract

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

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Prothena Reports Robust Reduction of Free Serum Alpha-Synuclein of Up to 96% after Single Dose of PRX002, a Novel Protein Immunotherapy for Parkinson’s Disease
Prothena Corporation plc announced positive results from a Phase I single ascending dose study of PRX002, a monoclonal antibody for the potential treatment of Parkinson’s disease and other related synucleinopathies. [Prothena Corporation plc] Press Release

RepliCel Life Sciences in Japan for Key Industry Meetings
RepliCel Life Sciences Inc. announced that it will be presenting at two biotech conferences in Japan. They will be presenting at the 14th Congress of the Japanese Society for Regenerative Medicine 2015 and the 12th Annual BioAsia International Conference. [RepliCel Life Sciences Inc.] Press Release

4th Annual Cell Line Development & Engineering Asia
Reprieve for Australian Research Facilities
The Australian government has bowed to pressure from the scientific community to fund key national research infrastructure that had been under threat of closure. [Nature News] Editorial

Stemming the Media Hype on Stem Cell Therapies
A new study by University of Alberta law researchers reveals sometimes overly optimistic news coverage of clinical translation of stem cell therapies — and as spokespeople, scientists need to be mindful of harnessing public expectations. [University of Alberta] Press Release | Abstract

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ImmunoCellular Therapeutics Establishes Manufacturing Agreement with PharmaCell B.V. for European Production of ICT-107 for Phase III Registration Trial
PharmaCell B.V. announced that it finalized an agreement with ImmunoCellular Therapeutics Ltd. to support its pivotal Phase III clinical trial in Europe with ICT-107. [ImmunoCellular Therapeutics, Ltd.] Press Release

TaiGen Biotechnology Announces Burixafor Collaboration with Cellex and the University Hospital Carl Gustav Carus Dresden in Germany
TaiGen Biotechnology Company, Limited announced the signing of an agreement to collaborate with Cellex GmbH and the University Hospital Carl Gustav Carus Dresden in Germany in an investigator-initiated clinical trial using burixafor in stem cell mobilization in healthy donors. [TaiGen Biotechnology Company, Limited] Press Release

Celldex Therapeutics Announces Clinical Trial Collaboration with Roche to Evaluate the Combination of Varlilumab and MPDL3280A
Celldex Therapeutics, Inc. announced that it has entered into a clinical trial collaboration with Roche to evaluate the safety, tolerability and preliminary efficacy of varlilumab, Celldex’s CD27 targeting investigational antibody, and MPDL3280A (anti-PDL1), Roche’s investigational cancer immunotherapy in a Phase I/II study in renal cell carcinoma. [Celldex Therapeutics, Inc.] Press Release

NantWorks and Sorrento Announce Second Comprehensive Global Collaboration on Discovery and Development of Novel Anti-Cancer Immunotherapies
NantWorks, Inc. and Sorrento Therapeutics, Inc. announced that they have entered into a binding agreement to initiate a global collaboration to discover and develop novel anti-cancer immunotherapies derived from Sorrento’s proprietary G-MAB® library against neoepitopes of tumor-specific antigens discovered using NantWorks’ proprietary pan-omics based, precision medicine approach. [Sorrento Therapeutics, Inc.] Press Release

Inovio Pharmaceuticals and Academic Collaborators Receive $16 Million HIV Grant from National Institute of Allergy and Infectious Diseases
Inovio Pharmaceuticals, Inc. announced that the company and its academic collaborators, including the University of Pennsylvania, were awarded a new five-year $16 million Integrated Preclinical/Clinical AIDS Vaccine Development Program grant from the National Institute of Allergy and Infectious Diseases. [Inovio Pharmaceuticals, Inc.] Press Release

Kite Pharma Strengthens Its T Cell Receptor (TCR) Cancer Gene Therapy Platform through Acquisition of T-Cell Factory B.V. (TCF™)
Kite Pharma, Inc. announced that it has further strengthened its TCR product platform and established a European presence through the acquisition of TCF™, which has been renamed Kite Pharma EU. [Kite Pharma, Inc.] Press Release

Bone Therapeutics Treats First Patients in ALLOB® Phase IIa Spinal Fusion Trial
Bone Therapeutics announced that the first four patients in the Phase IIa proof-of-concept study for spinal fusion procedures have been treated with Bone Therapeutics’ allogeneic osteoblastic (bone-forming) cell therapy product, ALLOB®. [Bone Therapeutics S.A.] Press Release

Advaxis Announces First Patient Treated in Phase I/II Dose-Escalation Study of ADXS-HPV for Recurrent Cervical Cancer
Advaxis, Inc. announced that the first patient was dosed in a Phase I/II clinical trial evaluating higher doses and repeat cycles of ADXS-HPV (ADXS11-001), an investigational Lm-LLO immunotherapy, for the treatment of patients with recurrent cervical cancer. [Advaxis, Inc.] Press Release
Submission for OMB Review; 30-Day Comment Request: Application for Collaboration With the Therapeutic Development Branch (TDB), Division of Preclinical Innovation (DPI), National Center for Advancing Translational Sciences (NCATS) (FR Doc. No: 2015-06084)
2015 Biological Device Application Approvals
Food and Drug Administration (United States)

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting (FR Doc. No: 2015-06116)

Reprocessing Medical Devices in Health Care Settings: Validation Methods and Labeling; Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No: 2015-06029)
NEW 21st International Society for Cellular Therapy (ISCT) Annual Meeting
May 27-30, 2015
Las Vegas, United States

NEW EMBO Workshop – Cellular and Molecular Mechanism of Tumor-Microenvironment Crosstalk
July 9-12, 2015
Tomsk, Russia

NEW Gordon Research Conference – Hormone-Dependent Cancers
August 16-21, 2015
Newry, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Research Associate – Cell Culture Specialist (Tisch MS Research Center of New York)

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

Scientist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

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)

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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