Volume 17.16 | Apr 25

Issue 17.16 April 25, 2016
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Gene Therapy Shows Early Success against “Bubble Boy” Disease
Early results suggest novel gene therapy is safe and effective for treatment of inherited immune disease. [Press release from St. Jude Children’s Research Hospital discussing online prepublication in Science Translational Medicine] Press Release | Abstract
Register Now: Webinar by Dr. Chad Cowan on Investigating Metabolic Disease with Human Pluripotent Stem Cells
PUBLICATIONS (Ranked by impact factor of the journal)
Development of Engineered T Cells Expressing a Chimeric CD16-CD3ζ Receptor to Improve the Clinical Efficacy of Mogamulizumab Therapy against Adult T Cell Leukemia
The authors lentivirally gene-modified peripheral blood T cells from healthy volunteers and adult T-cell leukemia (ATL) patients expressing the affinity-increased chimeric CD16-CD3ζ receptor (cCD16ζ-T cells). Subsequently they examined the antibody-dependent cellular cytotoxicity effect mediated by those cCD16ζ-T cells in the presence of Mog against ATL tumor cells both in vitro and in vivo. [Clin Cancer Res] Abstract

Three Year Follow Up of GMCSF/Bi-ShRNAfurin DNA Transfected Autologous Tumor Immunotherapy (Vigil™) in Metastatic Advanced Ewing’s Sarcoma
Investigators describe a follow-up through year three of a prospective, non-randomized study comparing an expanded group of Vigil-treated advanced disease Ewing’s sarcoma patients with a contemporaneous group of Ewing’s sarcoma patients not treated with Vigil. [Mol Ther] Abstract

Genipin Cross-Linked Decellularized Tracheal Tubular Matrix for Tracheal Tissue Engineering Applications
Scientists investigated the mechanical, pro-angiogenic and in vivo biocompatibility properties of decellularized airway matrices cross-linked with genipin. [Sci Rep] Full Article

Hemagglutinin-Targeting Artificial MicroRNAs Expressed by Adenovirus Protect Mice from Different Clades of H5N1 Infection
Researchers designed several artificial microRNAs (amiRNAs) targeting the hemagglutinin gene of H5N1, a major determinant of pathogenicity. Expression and delivery efficiency were enhanced by presenting functional amiRNA with chimpanzee adenovirus serotype 68. [Mol Ther Nucleic Acids] Full Article

Allogeneic Stem Cell Transplantation for Patients with Mantle Cell Lymphoma Who Failed Autologous Stem Cell Transplantation: A National Survey of the SFGM-TC
The French SFGM-TC performed a national survey on reduced-intensity conditioning allogeneic stem cell transplantation (SCT) for fit relapsed/refractory patients who failed after autologous SCT. [Bone Marrow Transplant] Abstract 

mRNA-Mediated Gene Supplementation of Toll-Like Receptors as Treatment Strategy for Asthma In Vivo
Researchers investigated the effect of a preventative treatment with combinations of Tlr1, Tlr2 and Tlr6 mRNA in a house dust mite-induced mouse model of asthma. [PLoS One] Full Article

Construction of Minicircle DNA Vectors Capable of Correcting Familial Hypercholesterolemia Phenotype in a LDLR Deficient Mouse Model
Earlier gene therapy studies have generated promising results for treating familial hypercholesterolemia caused by a defect in LDLR, but further development is hampered, because the cells harboring the viral vectors were eliminated by host immune system soon after delivery while the non-viral vectors were too bulky to be delivered to target cells. To overcome these problems, the authors constructed multiple minicircle DNA vectors to express the therapeutic LDLR. [Gene Ther] Abstract

Liver-Specific Overexpression of LPCAT3 Reduces Postprandial Hyperglycemia and Improves Lipoprotein Metabolic Profile in Mice
Researchers tested the possibility that increased expression of lysophosphatidylcholine acyltransferase-3 (LPCAT3), an enzyme that converts lysophosphatidylcholine to phosphatidylcholine in the liver, may alleviate the adverse effects of lysophospholipids absorbed after a lipid-glucose mixed meal. [Nutr Diabetes] Full Article

Local Injection of Bone Mesenchymal Stem Cells and Fibrin Glue Promotes the Repair of Bone Atrophic Nonunion In Vivo
The authors aimed to evaluate the efficacy of local injection of bone mesenchymal stem cells and fibrin glue in the treatment of atrophic nonunion in an animal model. [Adv Ther] Abstract

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Toxicity and Management in CAR T-Cell Therapy
Abrogating toxicity has become a critical step in the successful application of this emerging technology. To this end, the authors review the reported and theoretical toxicities of CAR T-cells and their management. [Mol Ther Oncolytics] Full Article

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

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MabVax Therapeutics’ Fully Human Antibody Approach to Cancer Therapeutics and Imaging Featured in Three Presentations
MabVax Therapeutics Holdings, Inc. announces that its fully human antibody approach to cancer therapeutics and imaging will be featured in three separate poster presentations. [Press release from MabVax Therapeutics Holdings, Inc. discussing research presented at the 2016 American Association of Cancer Research (AACR), New Orleans] Press Release

Antibody Therapy Reduces Cancer Stem Cells in Multiple Myeloma
An experimental antibody treatment decreased by half the number of cancer stem cells that drive the growth of tumors in nearly all patients with multiple myeloma, a cancer of the bone marrow and bone tissue, according to results of a preliminary clinical trial. [Press release from The Johns Hopkins University discussing research presented at the 2016 American Association for Cancer Research (AACR) Annual Meeting, New Orleans] Press Release

Penn-Led Team Presents Results from Clinical Trial of Personalized Cellular Therapy in Brain Tumor Patients
Immune cells engineered to seek out and attack a type of deadly brain cancer known as glioblastoma were found to have an acceptable safety profile and successfully migrate to and infiltrate tumors. [Press release from Penn Medicine discussing research presented at the 2016 American Association for Cancer Research (AACR) Annual Meeting, New Orleans] Press Release

Study Supports Evidence that Rab Disruption May Be Effective Strategy for Treating Multiple Myeloma
Roswell Park Cancer Institute researchers investigated agents that target and disrupt the trafficking of monoclonal antibodies in multiple myeloma, a cancer of the bone marrow. [Press release from Roswell Park Cancer Institute discussing research presented at the 2016 American Association for Cancer Research (AACR) Annual Meeting, New Orleans] Press Release

Alnylam Reports Complete 18-Month Data from Ongoing Phase II Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic
Alnylam Pharmaceuticals, Inc. announced complete 18-month data from its ongoing Phase II open-label extension study with patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of hereditary amyloidosis with polyneuropathy, also known as familial amyloidotic polyneuropathy. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the 68th American Academy of Neurology (AAN) Annual Meeting, Vancouver] Press Release

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US Agency Suspends Clinical Trials after Contamination Risk Discovered
The US National Institutes of Health has suspended two of its manufacturing facilities that were found to be at risk of contaminating materials intended for use in patients, the agency announced. Several clinical trials that use products made by those facilities are also on hold and will not be recruiting new patients until the issues are resolved. [Nature News] Editorial

Germany to Continue Building Its Ivy League
The program designed to create a German Ivy League will be extended indefinitely, giving a handful of the country’s top universities a yearly bonus of at least €10 million in extra funding, the German government announced. [ScienceInsider] Editorial

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TxCell and Ospedale San Raffaele Launch Collaboration for the Development of CAR-Tregs in Lupus Nephritis
TxCell SA and Ospedale San Raffaele announced they have entered into a strategic R&D collaboration. The collaboration will include a development arm focused on Lupus Nephritis, as well as a research program dedicated to CAR-Treg biology. [TxCell SA] Press Release

FDA Awards $2.25M Grant to Study Immunosuppresive Drug in High-Risk Patients
Rita Alloway, PharmD, University of Cincinnati (UC) research professor of medicine and director of transplant clinical research within the UC Department of Internal Medicine, received a $2.25 million FDA grant to run two clinical trials studying the effects of immunosuppressant tacrolimus (Prograf and generics) in high-risk transplant patients. [University of Cincinnati] Press Release

Researcher Receives NIH Grant to Continue Development of New Therapeutic Agents for Multiple Sclerosis, Cancer and Sepsis
A leading scientist at Florida Atlantic University has received $540,250 from the National Cancer Institute of the National Institutes of Health (NIH) to continue his groundbreaking research to develop new therapeutic agents for collagen-based diseases including multiple sclerosis, cancer and sepsis. [Florida Atlantic University] Press Release

Research in Liver Cancer, Tissue Regeneration at Children’s Research Institute at UTSW Garners Stand Up to Cancer Grant
Dr. Hao Zhu, Assistant Professor of Children’s Medical Center Research Institute at UT Southwestern, is one of ten researchers in the nation to receive a Stand Up To Cancer grant to further his studies of a gene whose absence protects mice against liver cancer and promotes liver tissue regeneration in mammals. [The University of Texas Southwestern Medical Center] Press Release

NanoCor Therapeutics Announces FDA Acceptance of Investigational New Drug (IND) for Carfostin®
NanoCor Therapeutics, Inc. announced that the United States Food and Drug Administration (FDA) has accepted the company’s IND application for Carfostin®, a cardiac gene therapy in development for the treatment of congestive heart failure. [NanoCor Therapeutics, Inc. (PR Newswire Association LLC)] Press Release

FDA Grants Orphan Drug Exclusivity to EVOMELA™ (Melphalan) for Injection, Indicated for Multiple Myeloma
Spectrum Pharmaceuticals announced that the U.S. Food and Drug Administration Office of Orphan Products Development has granted seven years of Orphan Drug Exclusivity for EVOMELA for use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma. [Spectrum Pharmaceuticals] Press Release

Diplomat to Dispense VENCLEXTA™ for the Treatment of Chronic Lymphocytic Leukemia
Diplomat Pharmacy, Inc. announced that it is now filling prescriptions for VENCLEXTA™ (venetoclax). VENCLEXTA™ was recently approved by the U.S. Food and Drug Administration for the treatment of people with chronic lymphocytic leukemia who have received at least one prior therapy, including those with 17p deletion. [Diplomat Pharmacy, Inc.] Press Release

RXi Pharmaceuticals Fortifies Its Novel RNAi Patent Portfolio with Notice of Allowance for Lead Clinical Candidate RXI-109
RXi Pharmaceuticals Corporation announced that it has received a Notice of Allowance, from the United States Patent and Trademark Office, for both the composition of matter and methods of use for its self-delivering RNAi (sd-rxRNA®) compounds targeting connective tissue growth factor. [RXi Pharmaceuticals Corporation] Press Release

Creative Medical Health, Inc. Files Patent on Stem Cell Therapy for Zika Virus Neurological Effects
Creative Medical Health, Inc. (CMH) announced filing of a patent covering the use of proprietary mesenchymal stem cell types in the treatment of neurological complications of Zika virus. The patent is a continuation of ongoing research at CMH that previously led to filing of intellectual property covering use of mesenchymal stem cells for multiple sclerosis. [Creative Medical Health, Inc. (Business Wire)] Press Release

SpinalCyte, LLC Receives New Canadian Patent for Spinal Disc Tissue Engineering
SpinalCyte, LLC announced the issuance of Canadian Patent No. 2641170, “Methods and Compositions for Repair of Cartilage Using an In Vivo Bioreactor.” [SpinalCyte, LLC] Press Release
Prospective Grant of Exclusive License: The Development of Anti-CD70 Chimeric Antigen Receptors (CARs) for the Treatment of Chronic Myelogenous Leukemia (FR Doc. No: 2016-09324)

Prospective Grant of Exclusive License: Development of the CB1/iNOS Series of Compounds as a Therapeutic to Treat System Sclerosis, Scleroderma, and Other Skin Fibrotic Diseases in Humans (FR Doc. No: 2016-08985)

Prospective Grant of Exclusive License: The Development of MRI-1569, MRI-2213 and MRI-2214 as a Therapeutic to Treat Obesity, Diabetes, Fatty Liver Disease and Liver Fibrosis (FR Doc. No: 2016-08986)

National Institutes of Health (NIH) Office of Science Policy (OSP) Recombinant or Synthetic Nucleic Acid Research: Action under the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines) (FR Doc. No: 2016-08810)
Scientific Evidence in Development of Human Cells, Tissues, and Cellular and Tissue-Based Products Subject to Premarket Approval; Public Workshop

Comparability Protocols for Human Drugs and Biologics: Chemistry, Manufacturing, and Controls Information; Draft Guidance for Industry; Availability
Food and Drug Administration (United States)

Authorization of Emergency Use of an In Vitro Diagnostic Device for Detection of Zika Virus; Availability (FR Doc. No: FDA-2016-N-0969)

Authorization of Emergency Use of an In Vitro Diagnostic Device for Detection of Ebola Zaire Virus; Availability (FR Doc. No: FDA-2014-N-1206)
NEW The Bioprocessing Summit
August 15-19, 2016
Boston, United States

NEW CELLS | Musculoskeletal 2016
October 7-9, 2016
Amsterdam, Netherlands

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Fellow – Immunology (The Pirbright Institute)

NEW Full Professorship – Radiopharmaceutical Development (German Cancer Research Center)

NEW Director – Stem Cells (PharmaScouts)

Scientist – Bioengineering (STEMCELL Technologies Inc.)

Scientist – Human Pluripotent Stem Cell Neural Differentiation (STEMCELL Technologies Inc.)

PhD Studentship – Stem Cell Biology (Masaryk University)

Assistant Professor – Cell Factory Design (Chalmers University of Technology)

Postdoctoral Fellow – Non-Viral Gene Delivery (Nanyang Technological University)

Project Lead – Engineering (Cytonome)

Faculty Position – Tumor Immunology (John Wayne Cancer Institute at Providence Saint John’s Health Center)

Postdoctoral Fellow – Non-Coding RNAs in Malignant Progression (The University of Texas MD Anderson Cancer Center)

Postdoctoral Associate – Stem Cell Biology (University of Illinois Chicago College of Medicine)

Postdoctoral Associate – Genetic Medicine (Cornell University)

Postdoctoral Fellow – Retinal Disease Genomics and Gene Therapy (Ocular Genomics Institute, Mass Eye and Ear and Harvard Medical School)

Postdoctoral Fellow – T Cell Reconstitution (Fred Hutchinson Cancer Research Center)

Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Fellowship – Stem Cells, Oligodendrogenesis and Remyelination (UC Davis)

Postdoctoral Position – Hematology/Oncology (Penn State College of Medicine)

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

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

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