Cell Therapy News Volume 19.06 | Feb 26 2018

TOP STORY

Fragile X Syndrome Neurons Restored Using CRISPR/Cas9-Guided Activation Strategy

Reactivated FMR1 gene rescued neurons derived from fragile X syndrome induced pluripotent stem cells, reversing the abnormal electrical activity associated with the syndrome. When rescued neurons were engrafted into the brains of mice, the FMR1 gene remained active in the neurons for at least three months. [Press release from Whitehead Institute discussing online prepublication in Cell] Press Release | Abstract | Graphical Abstract

(Ranked by impact factor of the journal)

Exosome-Mediated Benefits of Cell Therapy in Mouse and Human Models of Duchenne Muscular Dystrophy

Researchers report that clinical-stage cardiac progenitor cells, known as cardiosphere-derived cells (CDCs), improved cardiac and skeletal myopathy in the mdx mouse model of Duchenne muscular dystrophy. Injection of CDCs into the hearts of mdx mice augmented cardiac function, ambulatory capacity, and survival. [Stem Cell Reports] Full Article | Press Release | Graphical Abstract

Phase I Study of Intrapleural Gene Mediated Cytotoxic Immunotherapy in Patients with Malignant Pleural Effusion

A Phase I dose escalation trial of gene-mediated cytotoxic immunotherapy followed by chemotherapy was conducted in patients with malignant pleural effusion. An adenovirus-based vector expressing the thymidine kinase gene was administered intrapleurally in three cohorts, at a dose of 1 x 10¹² to 10¹³ vector particles. [Mol Ther] Abstract

Interaction between Hypothermia and Delayed Mesenchymal Stem Cell Therapy in Neonatal Hypoxic-Ischemic Brain Injury

Mesenchymal stem cells co-cultured with brain extracts of hypothermia treatment-treated animals revealed increased pro-inflammatory cytokine and decreased growth factor expression. In vivo protein analysis showed higher pro-inflammatory cytokine levels after combined treatment compared to single therapy. [Brain Behav Immun] Full Article

Therapeutic Neural Stem Cells Engineered during Different Stages of Reprogramming Reveal Varying Therapeutic Efficacies

Scientists explored the potential of creating induced pluripotent stem cell derived therapeutic neural stem cells by using either unmodified or gene-modified somatic cells and tested their fate and therapeutic efficacies in vitro and in vivo. [Stem Cells] Abstract

Intratumoral Delivery of Interferon γ-Secreting MSCs Repolarizes Tumor-Associated Macrophages and Suppresses Neuroblastoma Proliferation In Vivo

The authors developed an intra-adrenal model of neuroblastoma, characterized by liver and lung metastases. They then engineered mesenchymal stromal cells (MSCs) to deliver interferon-gamma (IFNγ) directly to the tumor microenvironment (TME). In vivo, they attained a therapeutically active TME concentration of IFNγ without increased systemic concentration or toxicity. [Stem Cells] Abstract

Intraperitoneal Administration of Mesenchymal Stem Cells Ameliorates Acute Dextran Sulfate Sodium-Induced Colitis by Suppressing Dendritic Cells

Clinical, histological, immunophenotypic analysis and passive transfer of mesenchymal stem cell (MSC)-primed dendritic cells (DCs) were used to evaluate capacity of MSCs to suppress inflammatory phenotype of DCs in vivo. [Biomed Pharmacother] Abstract

Successful Hematopoietic Stem Cell Transplantation for Osteopetrosis Using Reduced Intensity Conditioning

Scientists detail the outcomes of bone marrow transplant in a cohort of 31 patients treated with a combination of fludarabine, treosulphan, thiotepa, and antithymocyte globulin. [Pediatr Blood Cancer] Abstract

Heterogenic Transplantation of Bone Marrow-Derived Rhesus Macaque Mesenchymal Stem Cells Ameliorates Liver Fibrosis Induced by Carbon Tetrachloride in Mouse

Enhanced green fluorescent protein-labeled mesenchymal stem cells (MSCs) were transplanted into mice with carbon tetrachloride-induced liver fibrosis via tail vein injection. The ability of the heterogenic MSC infusion to ameliorate liver fibrosis in mice was evaluated by a blood plasma chemistry index, pathological examination and liver fibrosis-associated gene expression. [PeerJ] Full Article

Platelet-Targeted Gene Therapy for Hemophilia

The author summarizes studies on platelet-specific gene therapy of hemophilia A as well as hemophilia B. [Mol Ther Methods Clin Dev] Abstract

Treating Childhood Traumatic Brain Injury with Autologous Stem Cell Therapy

Researchers discuss the use of umbilical cord blood cells and bone marrow stromal cells for acute and chronic treatment of experimental neonatal traumatic brain injury. Autologous stem cell transplantation may dampen neuroinflammation. [Expert Opin Biol Ther] Abstract

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

TxCell Provides Details of CAR-Treg Manufacturing Process

TxCell SA announced details of its CAR-regulatory T cell (Treg) manufacturing process. [Press release from TxCell SA discussing research presented at the 2018 CAR-TCR Summit Europe, London] Press Release

AbbVie and Voyager Therapeutics Announce Global Strategic Collaboration to Develop Potential New Treatments for Alzheimer’s Disease and Other Tau-Related Neurodegenerative Diseases

AbbVie and Voyager Therapeutics, Inc. announced that they have entered into an exclusive strategic collaboration and option agreement to develop and commercialize vectorized antibodies directed against tau for the treatment of Alzheimer’s disease and other neurodegenerative diseases. [AbbVie Inc.] Press Release

Kite, a Gilead Company, and Sangamo Therapeutics Announce Collaboration to Develop Next-Generation Engineered Cell Therapies for the Treatment of Cancer

Kite and Sangamo Therapeutics, Inc. announced the companies have entered into a worldwide collaboration using Sangamo’s zinc finger nuclease technology platform for the development of next-generation ex vivo cell therapies in oncology. [Sangamo Therapeutics, Inc.] Press Release

Fate Therapeutics Announces Receipt of CIRM Grant for Clinical Translation of FT516 Off-the-Shelf Engineered NK Cell Cancer Immunotherapy

Fate Therapeutics, Inc. announced that the California Institute for Regenerative Medicine (CIRM) awarded the company a $4.0 million grant to advance FT516 into a first-in-human clinical trial. [Fate Therapeutics, Inc.] Press Release

Merck and Viralytics Announce Acquisition Agreement, Expanding Merck’s Leading Immuno-Oncology Pipeline

Merck and Viralytics Limited announced that the companies have signed a definitive agreement under which it is proposed that Merck, through a subsidiary, will acquire Viralytics by way of a scheme of arrangement for AUD 1.75 cash per Viralytics share. [Merck & Co., Inc.] Press Release

Primary Endpoint Successfully Achieved in Mesoblast’s Phase III Cell Therapy Trial for Acute Graft versus Host Disease

Mesoblast Limited announced that the Phase III trial of its allogeneic mesenchymal stem cell product candidate MSC-100-IV in children with steroid refractory acute graft versus host disease has successfully met the primary endpoint of Day 28 overall response rate. [Mesoblast Limited] Download Press Release

Fortress Biotech Announces Aevitas Therapeutics Enters Sponsored Research Agreement with the University of Massachusetts Medical School to Advance AAV Gene Therapies

Fortress Biotech announced that its subsidiary, Aevitas Therapeutics, Inc., has entered into a sponsored research agreement with the laboratory of Guangping Gao, Ph.D., at the University of Massachusetts Medical School. [Fortress Biotech, Inc.] Press Release

MilliporeSigma Receives Two More Patents for CRISPR Technology

MilliporeSigma announced that the Korean Intellectual Property Office and the Israel Patent Office have each issued notices granting MilliporeSigma’s patent applications for the company’s CRISPR technology used in a genomic-integration method for eukaryotic cells. [MilliporeSigma] Press Release

PharmaCyte Announces Successful Completion of 6-Month Stability Study on Encapsulated Cells for Clinical Trial

PharmaCyte Biotech, Inc. announced that its six-month study on storage of the frozen encapsulated cells necessary for the determination of an “initial shelf life” has been successfully completed by Austrianova. [PharmaCyte Biotech, Inc. (PR Newswire Association LLC)] Press Release

Florida Residents Could Soon Get the Power to Alter Science Classes

Policymakers in the United States are pushing to give the public more power to influence what educators teach students. Florida’s legislature started considering two related bills that, if enacted, would let residents recommend which instructional materials teachers in their school district use in their classrooms. [Nature News] Editorial

National Science Foundation to Close Its Overseas Offices

A plan by the U.S. National Science Foundation (NSF) to close its overseas offices, first reported on the Science | Business website, is getting mixed reviews in the scientific community. The NSF announced it would shutter its outposts in Beijing, Brussels, and Tokyo by summer; two U.S. staff will return to the agency’s headquarters in Alexandria, Virginia, and local staff will be reassigned to U.S. embassies. [ScienceInsider] Editorial

One of the Most Powerful Science Policy Jobs in Brussels Changes Hands

It’s the end of an era at the European Commission’s research department: The most powerful civil servant in Brussels’s science policy circles, Director-General for Research and Innovation Robert-Jan Smits, is leaving his post. [ScienceInsider] Editorial

Half of Canada’s Government Scientists Still Feel Muzzled

More than half of government scientists in Canada—53%—do not feel they can speak freely to the media about their work, even after Prime Minister Justin Trudeau’s government eased restrictions on what they can say publicly, according to a survey released by a union that represents more than 16,000 federal scientists. [ScienceInsider] Editorial

U.K. Scientists Increasingly Anxious about Brexit Confusion

With the second phase of Brexit negotiations approaching, scientists in the United Kingdom are urging their government to clarify its position on funding agreements and migration of research talent after the country separates from the European Union in March 2019. [ScienceInsider] Editorial

Meet the Scientists Running to Transform Congress in 2018

The first big test for the cohort of science candidates comes on 6 March, when Texas holds the nation’s first primary elections. Several candidates are running to become Democratic nominees in that Lone Star state’s House districts. [ScienceInsider] Editorial

NIH

A Strategic Roadmap for Establishing New Approaches to Evaluate the Safety of Chemicals and Medical Products in the United States; Availability of Report (FR Doc. No:2018-03476) Notice

NEW Advanced Therapies Commercialisation Congress
June 13-14, 2018
Amsterdam, The Netherlands

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

NEW Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

NEW Postdoctoral Fellowship – Functional Genomics of Human Hematopoiesis (Boston Children’s Hospital)

NEW Postdoctoral Researcher – Molecular Cardiology (National Centre for Cardiovascular Research)

Research Assistant – Cancer Therapy (Philipps-Universität Marburg)

Postdoctoral Fellow – Cancer Research (Indiana University School of Medicine)

Postdoctoral Associate – Cardiovascular Science (Duke University)

Postdoctoral Position – Pancreas Development and Stem Cells (Helmholtz Zentrum München)

Postdoctoral Fellowships – Cancer Research (University of Miami)

Tenure-Track Group Leader Position – Pancreatic Cancer Biology (University of Copenhagen)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

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