Cell Therapy News Volume 19.33 | Oct 1 2018

TOP STORY

Dynamics of Genetically Engineered Hematopoietic Stem and Progenitor Cells after Autologous Transplantation in Humans

Investigators report a comprehensive study of the frequencies, dynamics and output of seven hematopoietic stem and progenitor cell (HSPC) subtypes in humans that was performed by tracking 148,093 individual clones in six patients treated with lentiviral gene therapy using autologous HSPC transplantation and followed for up to five years. [Nat Med] Abstract

(Ranked by impact factor of the journal)

Induction of Resistance to Chimeric Antigen Receptor T Cell Therapy by Transduction of a Single Leukemic B Cell

Scientists report a patient relapsing 9 months after CD19-targeted CAR T cell infusion with CD19^– leukemia that aberrantly expressed the anti-CD19 CAR. [Nat Med] Abstract | Editorial

Immunomodulatory Placental-Expanded, Mesenchymal Stromal Cells Improve Muscle Function following Hip Arthroplasty

The authors addressed surgical trauma-related muscle injuries using local intraoperative application of allogeneic placenta-derived, mesenchymal-like adherent cells, using hip arthroplasty as a standardized injury model, because of the high regenerative and immunomodulatory potency of this cell type. [J Cachexia Sarcopenia Muscle] Full Article

Extracellular Vesicles Derived from Human Umbilical Cord Mesenchymal Stem Cells Alleviate Rat Hepatic Ischemia-Reperfusion Injury by Suppressing Oxidative Stress and Neutrophil Inflammatory Response

Investigators demonstrated that human umbilical cord mesenchymal stem cell-extracellular vesicles could protect against ischemia-reperfusion injury-induced hepatic apoptosis by reducing the infiltration of neutrophils and alleviating oxidative stress in hepatic tissue in vivo. [FASEB J] Abstract

Neuroprotection of Retinal Ganglion Cells by a Novel Gene Therapy Construct that Achieves Sustained Enhancement of Brain-Derived Neurotrophic Factor/Tropomyosin-Related Kinase Receptor-B Signaling

Scientists characterized a novel adeno-associated virus gene therapy that not only increased brain-derived neurotrophic factor (BDNF) production but also improved long-term neuroprotective signaling by increasing expression of the BDNF receptor within the inner retina. [Cell Death Dis] Full Article

Nasal Administration of Cationic Nanoemulsions as Nucleic Acids Delivery Systems Aiming at Mucopolysaccharidosis Type I Gene Therapy

Formulations composed of DOPE, DOTAP, MCT, and DSPE-PEG were prepared by high-pressure homogenization, and assessed in vitro on human fibroblasts from mucopolysaccharidosis type I (MPS I) patients and in vivo on MPS I mice for α-L-iduronidase production and gene expression. [Pharm Res] Abstract

Transplantation of Cardiac Mesenchymal Stem Cell-Derived Exosomes Promotes Repair in Ischemic Myocardium

Scientists investigated the effect of exosomes from cardiac mesenchymal stem cells (C-MSCs-Exo) on angiogenesis in ischemic myocardium. They intramyocardially injected C-MSC-Exo or PBS into the infarct border zone after induction of acute mouse myocardial infarction. [J Cardiovasc Transl Res] Abstract

Icariin and Mesenchymal Stem Cells Synergistically Promote Angiogenesis and Neurogenesis after Cerebral Ischemia via PI3K and ERK1/2 Pathways

Male SD rats were subjected to transient middle cerebral artery occlusion followed by reperfusion and icariin (ICA)/mesenchymal stem cell (MSC) treatment. They showed that ICA and MSCs combined treatment greatly reduced brain infarction volume, improved neurologic deficits of motor and somatosensory function and neurobehavioral outcomes. [Biomed Pharmacother] Abstract

Evaluation of the Activity Levels of Rat FVIII and Human FVIII Delivered by Adeno-Associated Viral Vectors Both In Vitro and In Vivo

AAV-delivered rat coagulation factor VIII (FVIII) and human FVIII exerted long-term therapeutic effects on hemophilia A mice and caused a transient ALT elevation. [Blood Cells Mol Dis] Abstract

Regulatory Dendritic Cells Induced by Mesenchymal Stem Cells Ameliorate Dextran Sodium Sulfate-Induced Chronic Colitis in Mice

Researchers investigated whether mesenchymal stem cells (MSCs) could differentiate dendritic cells (DCs) into regulatory DCs (rDCs) and compared the therapeutic effects of rDCs and MSCs on dextran sodium sulfate-induced chronic colitis mice. [Gut Liver] Abstract

Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News.

Improved Therapeutic Potential of MSCs by Genetic Modification

The authors summarize the uses of genetically modified mesenchymal stem cells (MSCs) to treat different diseases, including bone diseases, cardiovascular diseases, autoimmune diseases, central nervous system disorders, and cancer. [Gene Ther] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Torque Announces the Presentation of Preclinical Data for Deep IL-15 Primed T Cells Demonstrating Superior T Cell Expansion, Persistence, and Safety Compared with Systemically Administered IL-15

Torque announced preclinical data demonstrating superior T cell expansion, persistence, and safety for the company’s Deep IL-15 Primed T cells compared to systemically administered IL-15. [Press release from Torque Therapeutics discussing research presented at the 4^th CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference, New York] Press Release

Sarepta Therapeutics and Jerry R. Mendell, M.D. from Nationwide Children’s Hospital to Provide Update on Duchenne Muscular Dystrophy Gene Therapy Program

Sarepta Therapeutics, Inc. and Jerry R. Mendell, M.D. will host a webcast conference call. The call will focus on an update regarding Sarepta’s Duchenne muscular dystrophy (DMD) micro-dystrophin gene therapy program and will be led by Dr. Mendell, a renowned researcher in the field of DMD. [Press release from Sarepta Therapeutics, Inc. discussing research to be presented at the 23^rd International Annual Congress of the World Muscle Society (WMS), Mendoza] Press Release

RXi Pharmaceuticals to Present New Data from Its Immuno-Oncology Program Using sd-rxRNA Therapeutic Compounds Targeting Intracellular Checkpoint Cbl-b in NK Cells

“RXi Pharmaceuticals Corporation announced that it will present a poster entitled, ‘The Use of Self-delivering RNAi to Enhance NK Cell Cytotoxicity'”. [Press release from RXi Pharmaceuticals discussing research presented at the 16^th Annual Discovery on Target Conference, Boston] Press Release

STEMCELL Technologies Signs Exclusive License to Commercialize Human Pluripotent Stem Cell-Derived Kidney Organoid Culture System

STEMCELL Technologies announced that they have signed an exclusive license agreement with Brigham and Women’s Hospital for rights to commercialize technologies for the generation of human pluripotent stem cell-derived kidney organoids. [STEMCELL Technologies Inc.] Press Release

Nobel Prize for Medicine Goes to Cancer Immune Therapy Pioneers

Immunologists James P. Allison and Tasuku Honjo have won this year’s Nobel Prize in Physiology or Medicine for their pioneering work in the relatively new field of cancer immunotherapy. [Scientific American] Press Release

Hitachi Chemical Advanced Therapeutics Solutions Signs Three-Year Agreement with GSK to Provide Clinical Manufacturing for T Cell Therapy

Hitachi Chemical Advanced Therapeutics Solutions, LLC announced that they have signed a three-year clinical manufacturing agreement with GSK. Under the agreement, HCATS will manufacture GSK’s SPEAR T-cell receptor therapy targeting NY-ESO-1 for the US, Canadian and European trials. [Hitachi Chemical Advanced Therapeutics Solutions, LLC (Business Wire, Inc.)] Press Release

Pluristem Therapeutics Announces FDA Orphan Drug Designation for PLX Cell Therapy for the Treatment of Graft Failure and Incomplete Recovery following Hematopoietic Cell Transplantation

Pluristem Therapeutics Inc. announced that the FDA has granted Orphan Drug Designation to the company’s PLX cell therapy for the treatment of graft failure and incomplete hematopoietic recovery following hematopoietic cell transplantation. [Pluristem Therapeutics Inc.] Press Release

Histogenics to Meet with FDA to Discuss NeoCart Phase III Clinical Trial Data and Potential Regulatory Pathway

Histogenics Corporation announced that the FDA has granted a Type C meeting on October 30, 2018 to discuss the top-line results from the NeoCart Phase III clinical trial and Histogenics’ planned Biologics License Application submission. [Histogenics Corporation] Press Release

PharmaCyte Biotech Reports Completion of Crucial FDA-Required Study for Pancreatic Cancer Trial

PharmaCyte Biotech, Inc. announced that it has successfully determined the modified site and chromosome location of the cytochrome P450-2B1 gene in the DNA of the genetically altered human cells known as 22P1G that will be encapsulated and used together with the cancer prodrug ifosfamide in PharmaCyte’s upcoming clinical trial. [PharmaCyte Biotech, Inc.] Press Release

Ultragenyx Announces Positive Topline Cohort 2 Results from Phase I/II Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency and Progression to Higher Dose

Ultragenyx Pharmaceutical Inc. announced topline positive safety and efficacy data from the second dose cohort and positive longer-term data from the first dose cohort of the ongoing Phase I/II study of DTX301, an investigational adeno-associated virus gene therapy for the treatment of OTC deficiency. [Ultragenyx Pharmaceutical Inc.] Press Release

AVROBIO Announces Updated Clinical Data for AVR-RD-01 Gene Therapy in Fabry Disease

AVROBIO, Inc. announced clinical data and patient updates from the investigator-sponsored Phase I study and the AVROBIO-sponsored Phase II clinical trial of AVR-RD-01. [AVROBIO, Inc.] Press Release

AVROBIO Receives No Objection to Clinical Trial Application from Health Canada for AVR-RD-02 Gene Therapy for Gaucher Disease

AVROBIO, Inc. announced that it has received no objection to its clinical trial application from Health Canada for a Phase I/II clinical trial of AVR-RD-02. [AVROBIO, Inc.] Press Release

Finland Joins Europe’s Bold Open-Access Push

Finland’s national research funder has signed up to Plan S — a push by a group of European organizations to make a radical change to the way that research results are published. [Nature News] Editorial

Biologists Irate at NSF’s New One-Proposal Cap

The National Science Foundation (NSF) in Alexandria, Virginia, has made several tweaks to its grant proposal policies in recent years to keep staff and reviewers from being overwhelmed by the rising number of submissions. But some biologists say the latest change goes too far. [ScienceInsider] Editorial

Hundreds of Academics at Top UK Universities Accused of Bullying

Hundreds of academics have been accused of bullying students and colleagues in the past five years, prompting concerns that a culture of harassment and intimidation is thriving in Britain’s leading universities. [The Guardian] Editorial

NIH

Prospective Grant of an Exclusive Patent License: Development and Commercialization of Cell Therapies for Cancer (FR Doc. No:2018-21096) Notice

NEW 2018 National Cancer Research Institute (NCRI) Cancer Conference
November 4-6, 2018
Glasgow, United Kingdom

NEW BIT’s 12^th World Congress of Regenerative Medicine & Stem Cell
December 7-9, 2018
Xi’an, China

Visit our events page to see a complete list of events in the community.

NEW Quality Control Scientists/Analysts – Cell and Gene Therapy (GlaxoSmithKline)

NEW Postdoctoral Fellow – Stem Cell Biology/Stroke, and Alzheimer’s Disease Research (Tulane University)

NEW Open Rank Professor – Biopharmaceutical Discovery (University of Delaware)

NEW Assistant Professor – Biology (University of Delaware)

Research Associate – Immunotherapy (Baylor College of Medicine)

Postdoctoral Position – Cancer, Immunology, and/or Single-Cell Epigenomics (CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences)

Postdoctoral Researcher – T Cell Autoantigens in Human Chronic Inflammatory Diseases (Karolinska Institutet)

Director – Clinical Development (California Institute for Biomedical Research)

Postdoctoral Research Assistant – Gene Therapy (University of Oxford)

Faculty Position – Adoptive T Cell Therapy (Dana-Farber Cancer Institute)

Professorship – Somatic Gene Therapy (University of Zurich)

Postdoctoral Staff Fellow – Biologist/Biomedical Engineer (Food and Drug Administration)

Associate Professor – Cancer Research (University of Maryland)

Senior Scientist – CAR-T Cell Research (CELLECTIS Inc.)

Postdoctoral Fellow – Immuno-Oncology (CELLECTIS Inc.)

Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.

Have we missed an important article or publication in Cell Therapy News? Click here to submit!

Comments or suggestions? Submit your feedback here.