Volume 17.02 | Jan 18

Issue 17.02 January 18, 2016
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California Institute for Biomedical Research (Calibr) Releases a Pair of Research Articles Describing a Novel Switchable CAR-T Cell Platform to Treat Cancer
Researchers describe the development of a “switchable” chimeric antigen receptor (sCAR)-T cell system that provides control over the activation, targeting, and anti-tumor activity of CAR-T cells. [Press release from Calibr (PR Newswire Association LLC) discussing online prepublications in the Proceedings of the National Academy of Sciences]
Press Release | Abstract 1 | Full Article 1 | Abstract 2 | Full Article 2
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PUBLICATIONS (Ranked by impact factor of the journal)
IFNα Gene/Cell Therapy Curbs Colorectal Cancer Colonization of the Liver by Acting on the Hepatic Microenvironment
Scientists showed that TEMs accumulate in the proximity of hepatic metastatic areas and that TEM-mediated delivery of interferon-α (IFNα) inhibits tumor growth when administered prior to metastasis challenge as well as on established hepatic lesions, improving overall survival. [EMBO Mol Med] Full Article

Human Mesenchymal Stem Cells Genetically Engineered to Overexpress Brain-Derived Neurotrophic Factor Improve Outcomes in Huntington’s Disease Mouse Models
Double-blinded studies were performed to examine the effects of intrastriatally transplanted human mesenchymal stem cell/brain-derived neurotrophic factor on disease progression in two strains of immune suppressed Huntington’s disease transgenic mice. [Mol Ther] Abstract | Full Article

An Interneuron Progenitor Maintains Neurogenic Potential In Vivo and Differentiates into GABAergic Interneurons after Transplantation in the Postnatal Rat Brain
The authors tested GE6 cells’ differentiation behavior in vivo by transplanting them into the postnatal rat forebrain. They found that GE6 cells migrate extensively in the neonatal forebrain and differentiate into both neurons and glia, but preferentially into neurons when compared with a sister progenitor clone CTX8. [Sci Rep] Full Article

Antitumor Activity of cGAMP via Stimulation of cGAS-cGAMP-STING-IRF3 Mediated Innate Immune Response
Researchers report that the STING activator cGAMP possesses significant antitumor activity in mice by triggering the STING-dependent pathway directly. cGAMP enhanced innate immune responses by inducing production of cytokines such as interferon-β, interferon-γ, and stimulating dendritic cells activation, which induces the cross-priming of CD8+ T cells. [Sci Rep] Full Article

Phase II Study of Necitumumab plus Modified FOLFOX6 as First-Line Treatment in Patients with Locally Advanced or Metastatic Colorectal Cancer
This single-arm Phase II study investigated the EGFR monoclonal antibody necitumumab plus modified FOLFOX6 in first-line treatment of locally advanced or metastatic colorectal cancer. [Br J Cancer] Full Article

Macrophage-Mediated Inflammation and Glial Response in the Skeletal Muscle of a Rat Model of Familial Amyotrophic Lateral Sclerosis (ALS)
Researchers evaluated inflammatory and glial responses near neuromuscular junctions in the limb muscle collected from a rat model of familial ALS during disease progression and following human mesenchymal stem cells modified to release glial cell line-derived neurotrophic factor transplantation. [Exp Neurol] Abstract

Antigen-Specific T Cells Fully Conserve Antitumor Function following Cryopreservation
The authors showed that cryopreservation fully conserves the differentiation potential of effector T cells, secretion of pro-inflammatory cytokines, cytotoxic function and does not impair the three-dimensional scanning motility of T cells or their capacity to infiltrate and reject tumors. [Immunol Cell Biol] Full Article

Durable Responses to Ibrutinib in Patients with Relapsed CLL after Allogeneic Stem Cell Transplantation
Researchers report clinical data from five CLL patients treated with ibrutinib for relapse after first or even second allogeneic transplantation. [Bone Marrow Transplant] Abstract

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Biology and Applications of CRISPR Systems: Harnessing Nature’s Toolbox for Genome Engineering
The authors discuss recent advances in understanding the diverse mechanisms by which clustered regularly interspaced short palindromic repeats (CRISPR) associated proteins respond to foreign nucleic acids and how these systems have been harnessed for precision genome manipulation in a wide array of organisms. [Cell] Abstract

Genome Editing Technologies for Gene and Cell Therapy
The authors present the mechanisms of different gene editing strategies and describes each of the common nuclease-based platforms, including zinc finger nucleases, TALE nucleases, meganucleases, and the CRISPR/Cas9 system. They then summarize the progress made in applying genome editing to various areas of gene and cell therapy, including antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders. [Mol Ther] Abstract | Full Article

Deciphering CD137 (4-1bb) Signaling in T-Cell Costimulation for Translation into Successful Cancer Immunotherapy
Anti-CD137 mAb and adoptive T-cell therapy strongly synergize, since CD137 expression can be used to select the T cells endowed with the best activities against the tumor, costimulation of the lymphocyte cultures to be used in adoptive T-cell therapy can be done with CD137 agonist antibodies or CD137L, and synergistic effects upon co-administration of T cells and antibodies are readily observed in mouse models. [Eur J Immunol] Abstract

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

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SELLAS Life Sciences Announces Preliminary Positive WT1 Cancer Vaccine Clinical Results in Multiple Myeloma Patients
SELLAS Life Sciences Group reported positive results from the company’s Phase I/II clinical study of its WT1 cancer vaccine in patients with multiple myeloma following autologous stem cell transplantation. [Press release from SELLAS Life Sciences Group discussing research presented at the 34th Annual J.P. Morgan Healthcare Conference, San Francisco] Press Release

MedGenome to Present on OncoPept – A Point Solution in Cancer Immunotherapy
MedGenome will present results from their head and neck cancer study in which published genomics data from over 400 head and neck cancer samples have been analyzed to identify potential vaccine candidates using MedGenome’s proprietary OncoPept platform. [Press release from MedGenome Inc. (PR Newswire Association LLC) discussing research to be presented at the Novel Immunotherapeutics Summit 2016, San Diego] Press Release

Perinatal Stem Cell Society 2016
Bitter Fight over CRISPR Patent Heats Up
A versatile technique for editing genomes has been called the biggest biotechnology advance since the polymerase chain reaction, and the US Patent and Trademark Office is set to determine who will reap the rewards. [Nature News] Editorial

Turkish Scientists Rocked by Accusations of Supporting Terrorism
Hundreds of Turkish academics are waiting to find out whether they will be prosecuted or sacked for spreading “terrorist propaganda”, after they signed a petition calling for violence to end in Turkey’s southeast, where government forces have been fighting Kurdish separatists. [Nature News] Editorial

U.K. Researcher Details Proposal for CRISPR Editing of Human Embryos
A regulatory committee in the United Kingdom evaluated a request to knock out development genes in day-old embryos. Kathy Niakan, a researcher at the Francis Crick Institute, discussed the rationale behind her proposed project and the hope that this line of inquiry might one day improve treatments for infertility. [ScienceInsider] Editorial

More Details Emerge on Fateful French Drug Trial
After news broke about a clinical research tragedy in France, more details are beginning to emerge about what happened at Biotrial, the private research company in Rennes where the Phase I study took place. [ScienceInsider] Editorial

What Vice President Biden’s Moonshot May Mean for Cancer Research
Cancer researchers are welcoming but eagerly awaiting more details on Vice President Joe Biden’s newly announced plan to lead “a moonshot” to cure cancer. [ScienceInsider] Editorial

Brazil’s Scientist-Entrepreneurs Unfettered
Full-time professors at public universities in Brazil will now be allowed to carry out research in the private sector—and get paid for it, without having to drop their academic jobs. The change is the result of a new law, signed by President Dilma Rousseff, designed to bring science and industry closer together. [ScienceInsider] Editorial

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GE Healthcare, FedDev Ontario Commit CAD $40 Million for New CCRM-Led Center to Solve Cell Therapy Manufacturing Challenges
GE Healthcare, the Federal Economic Development Agency for Southern Ontario (FedDev Ontario), and the Center for Commercialization of Regenerative Medicine (CCRM) are building a center for advanced therapeutic cell technologies in Toronto with an investment of $40 million from GE and FedDev Ontario. [Center for Commercialization of Regenerative Medicine] Press Release

ReNeuron Wins Major UK Grant to Advance Its Exosome Nanomedicine Platform
ReNeuron Group plc announced that it has been awarded a £2.1 million grant from Innovate UK to further advance its emerging exosome nanomedicine platform. The grant, entitled “Stem cell-derived exosomes for regenerative medicine,” has been awarded under Innovate UK’s Developing Regenerative Medicine & Cell Therapies’ grant scheme. [ReNeuron Group plc] Press Release

BioLineRx Announces Collaboration with MSD to Investigate the Combination of KEYTRUDA® (Pembrolizumab) and BL-8040 in Pancreatic Cancer
BioLineRx Ltd. announced a collaboration with MSD to support a Phase II study investigating BioLineRx’s BL-8040 in combination with KEYTRUDA® in patients with metastatic pancreatic cancer. [BioLineRx Ltd.] Press Release

Gritstone Oncology Enters into Exclusive Licensing Agreement and Collaboration with Memorial Sloan Kettering Cancer Center
Gritstone Oncology announced that the company has entered into an exclusive, worldwide licensing agreement and collaboration with Memorial Sloan Kettering Cancer Center. [Gritstone Oncology] Press Release

ATCC Licenses CRISPR/Cas9 Technology from the Broad Institute
ATCC announces that it licensed CRISPR/Cas9 gene editing technology from the Broad Institute of MIT and Harvard. [ATCC (PR Newswire Association LLC)] Press Release

Regeneus’ Progenza Stem Cell Therapy Clears Initial Safety Review
Regeneus’ off-the-shelf allogeneic stem cell treatment for knee osteoarthritis, Progenza, has enhanced its potential for commercialization for the company after an encouraging review of safety data from an oversight committee in early trialing. The regenerative medicine technology received no safety concerns after trialing with its first cohort of ten patients in the trial. [Regeneus Ltd.] Press Release

Immunotherapy for Epstein-Barr Related Lymphomas: First Health Canada Green Light Goes for Clinical Trial
Health Canada approved, for the first time in Canada, a clinical project for a Phase I study aimed at treating lymphomas associated with the Epstein-Barr virus (EBV) through adoptive cellular immunotherapy that is specific to EBV. [University of Montreal] Press Release

Abeona Therapeutics Announces Initial European Regulatory Approvals for Phase I/II Gene Therapy Clinical Studies for Patients with Sanfilippo Syndromes Type A (MPS IIIA) and Type B (MPS IIIB)
Abeona Therapeutics, Inc. announced the Interministerial Council of Genetically Modified Organisms has approved the genetically modified organism voluntary release regulatory filings for both Phase I/II gene therapy clinical studies to treat patients with AB0-101 and ABO-102 for patients with Sanfilippo syndrome type A or type B. [Abeona Therapeutics, Inc.] Press Release

Bone Therapeutics Expands Its Delayed-Union Program with ALLOB® into Multiple Fractures
Bone Therapeutics announced the initiation of a Phase IIA study for the treatment of multiple delayed-union fractures with ALLOB®, its novel allogeneic bone cell therapy product. [Bone Therapeutics S.A.] Press Release

Strategia Therapeutics Initiates a Phase I Clinical Trial of the Radioimmunotherapeutic “FF-21101″ For FUJIFILM in Patients with Advanced Cancers
Strategia Therapeutics has initiated a Phase I clinical trial of FUJIFILM’s radioimmunotherapeutic agent FF-21101 in the United States in patients with advanced solid tumor cancers. [Strategia Therapeutics, Inc.] Press Release

Intellia Therapeutics Launches New Division to Accelerate Ex Vivo Programs with CRISPR/CAS9
Intellia Therapeutics has launched a new division, eXtellia Therapeutics, with the intent of focusing resources and research on ex vivo applications of the novel technology, CRISPR/Cas9. [Intellia Therapeutics, Inc.] Press Release

FDA Grants Priority Review for Venetoclax New Drug Application
Genentech announced that the U.S. Food and Drug Administration (FDA) has accepted the new drug application and granted priority review for venetoclax for the treatment of people with chronic lymphocytic leukemia who have received at least one prior therapy, including those with 17p deletion. [Genentech, Inc.] Press Release
National Center for Advancing Translational Sciences; Notice of Closed Meeting (FR Doc. No: 2016-00799)

National Cancer Institute: Notice of Closed Meetings (FR Doc. No: 2016-00459)
Revised Preventive Measures to Reduce the Possible Risk of Transmission of Creutzfeldt-Jakob Disease and Variant Creutzfeldt-Jakob Disease by Blood and Blood Products: Guidance for Industry
Food and Drug Administration (United States)

How Should Liver Injury and Dysfunction Caused by Drugs Be Measured, Evaluated, and Acted Upon in Clinical Trials? (FR Doc. No: 2016-00690)

Advisory Committee: Vaccines and Related Biological Products Advisory Committee, Renewal (FR Doc. No: 2016-00675)
NEW Cell Therapy Manufacturing & Gene Therapy Congress
February 2-5, 2016
Brussels, Belgium

NEW Immune Checkpoint Inhibitors 2016
March 15-17, 2016
Boston, United States

NEW Cell Therapy Bioprocessing & Commercialization
October 5-7, 2016
Boston, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Cancer Immunotherapy (German Cancer Research Center)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Postdoctoral Position – CAR T Cell Engineering & Cancer Immunotherapy (University Hospital Würzburg)

Postdoctoral Fellow – Cell Therapy (Toronto Western Hospital)

Postdoctoral Researcher – Prostate Cancer Biology (Roswell Park Cancer Institute)

Postdoctoral Research Scientist – Follicular Lymphoma (Herbert Irving Comprehensive Cancer Center)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

PhD/MSc Position – Cancer Research and Wound Healing (Technion, Israel Institute of Technology)

Postdoctoral Position – Cell Mechanobiology (Washington University in St. Louis)

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