Cell Therapy News Volume 19.03 | Jan 29 2018

TOP STORY

A Modular Platform for Targeted RNAi Therapeutics

Scientists present a self-assembled modular platform that enables the construction of a theoretically unlimited repertoire of short interfering RNA targeted carriers. The therapeutic potential of the platform was demonstrated in an inflammatory bowel disease model by targeting colon macrophages to reduce inflammatory symptoms, and in a mantle cell lymphoma xenograft model by targeting cancer cells to induce cell death and improve survival. [Nat Nanotechnol] Abstract

(Ranked by impact factor of the journal)

Inhibition of Methyltransferase Setd7 Allows the In Vitro Expansion of Myogenic Stem Cells with Improved Therapeutic Potential

Upon transplantation, both mouse and human myogenic stem cells (MuSCs) expanded with a Setd7 small-molecule inhibitor were better able to repopulate the satellite cell niche, and treated mouse MuSCs showed enhanced therapeutic potential in preclinical models of muscular dystrophy. [Cell Stem Cell] Abstract | Press Release | Graphical Abstract

Transplantation of Human Embryonic Stem Cell-Derived Cardiovascular Progenitors for Severe Ischemic Left Ventricular Dysfunction

Investigators tested the feasibility of leveraging the properties of human embryonic stem cells to generate clinical-grade cardiovascular progenitor cells and assessed their safety in patients with severe ischemic left ventricular dysfunction. [J Am Coll Cardiol] Abstract

Genetically Modified Lentiviruses that Preserve Microvascular Function Protect against Late Radiation Damage in Normal Tissues

Researchers evaluated the efficacy of lentivirally delivered superoxide dismutase 2 overexpression and connective tissue growth factor knockdown by short hairpin RNA in reducing the severity of late adverse effects (LAEs) in an animal model of free flap LAEs. [Sci Transl Med] Abstract | Press Release

T Cell Gene Therapy for Perforin Deficiency Corrects Cytotoxicity Defects and Prevents Hemophagocytic Lymphohistiocytosis Manifestations

Scientists developed a gammaretroviral vector to transduce murine CD8-T cells in the prf^-/- mouse model. To verify functional correction of prf^-/- CD8-T cells in vivo, they used a lymphocytic choriomeningitis virus epitope transfected murine lung carcinoma cell tumor model. [J Allergy Clin Immunol] Abstract | Full Article

CRISPR/Cas9 Genome Editing in Human Hematopoietic Stem Cells

Researchers provide a detailed protocol for the production, enrichment, and in vitro and in vivo analyses of homologous recombination-targeted hematopoietic stem cells by combining CRISPR/Cas9 technology with the use of rAAV6 and flow cytometry. [Nat Protoc] Abstract

Cornea-Derived Mesenchymal Stromal Cells Therapeutically Modulate Macrophage Immunophenotype and Angiogenic Function

In vivo application of cornea-derived mesenchymal stromal cells (cMSCs) to murine corneas after injury lead to reduced macrophage infiltration and higher expression of CD206 in macrophages. Macrophages cocultured by cMSCs expressed significantly higher levels of anti-angiogenic and anti-inflammatory factors compared with control macrophages. [Stem Cells] Abstract

A Novel Epstein–Barr Virus-Latent Membrane Protein-1-Specific T-Cell Receptor for TCR Gene Therapy

Investigators describe a TCR gene transfer regimen to rapidly and reliably generate T-cells specific to Epstein–Barr virus-encoded latent membrane protein-1, which is a potential target for T-cell-based immunotherapy. [Br J Cancer] Abstract

Regeneration of Functional Alveoli by Adult Human SOX9⁺ Airway Basal Cell Transplantation

Researchers showed that a rare population of SOX9⁺ basal cells (BCs) located at airway epithelium rugae can regenerate adult human lung. Human SOX9⁺ BCs can be readily isolated by bronchoscopic brushing and indefinitely expanded in feeder-free condition. [Protein Cell] Full Article

Evaluation of Ex Vivo Produced Endothelial Progenitor Cells for Autologous Transplantation in Primates

Human and nonhuman primate endothelial progenitor cells from mobilized peripheral blood CD34⁺ cells were cultured according to a previously reported system. Efficacy and safety studies were performed by autologous transplantation via hepatic portal vein injection in a nonhuman primate model with acute liver sinusoidal endothelial cell injury. [Stem Cell Res Ther] Full Article

Posttransplant Chimeric Antigen Receptor Therapy

The authors review different approaches to overcome various challenges, including CAR therapies based on donor leukocyte infusion, virus-specific T cells, T cell receptor-deficient T cells, lymphoid progenitor cells and regulatory T cells. [Blood] Abstract

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

MultiVir Reports Reversal of Immunotherapy Resistance by Tumor Suppressor Immune Gene Therapy

MultiVir Inc. presented updated data demonstrating the ability of its immune gene therapies to reverse resistance to immune checkpoint inhibitor therapy. [Press release from MultiVir Inc. (PR Newswire Association LLC.) discussing research presented at the 2018 American Society of Clinical Oncology-Society of Immunotherapy of Cancer (ASCO-SITC) Symposium, San Francisco] Press Release

Synpromics Announces New Partnership with UCL to Develop Novel Gene Therapies for Blood-Based Disorders

Synpromics Ltd announced a collaboration with University College London (UCL) Great Ormond Street Institute of Child Health to develop novel gene therapies for pathologies affecting the hematopoietic system. [Synpromics Ltd.] Press Release

John Theurer Cancer Center First NJ Site Certified to Offer CAR-T Cell Cancer Therapy

John Theurer Cancer Center at Hackensack Meridian Health Hackensack University Medical Center is among limited U.S. sites, and the only hospital in New Jersey certified to offer Yescarta, a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded or who have relapsed after at least two other kinds of treatment. [Hackensack Meridian Health] Press Release

Consortium including TrakCel Awarded £1.4 Million from Innovate UK for Commercialization of Rexgenero Cell Therapy

TrakCel announced that the consortium comprising itself, the Cell and Gene Therapy (CGT) Catapult, Rexgenero Ltd and Fisher Bioservices have secured funding of £1.4 million from Innovate UK, the UK’s innovation agency, to lead an industrial research project. [TrakCel] Press Release

Spark Therapeutics Enters into a Licensing and Supply Agreement for Investigational Voretigene Neparvovec outside the U.S.

Spark Therapeutics announced it has entered into a licensing agreement with Novartis Pharmaceuticals to develop and commercialize investigational voretigene neparvovec outside the U.S., while Spark Therapeutics will continue to exclusively commercialize LUXTURNA™ in the U.S. [Spark Therapeutics] Press Release

Voyager Therapeutics Announces FDA Clearance of Investigational New Drug Application for VY-AADC for Advanced Parkinson’s Disease

Voyager Therapeutics, Inc. announced that the FDA has cleared the Investigational New Drug application for VY-AADC, allowing the company to formally initiate clinical trial sites, screen and begin dosing patients for its pivotal Phase II-III program for advanced Parkinson’s disease. [Voyager Therapeutics] Press Release

UF Launches New Center for Regenerative Medicine

The University of Florida (UF) launched a Center for Regenerative Medicine to develop lifesaving therapies “to heal the body from within” using stem cells that repair damaged tissue and organs. [University of Florida] Press Release

NIH to Launch Genome Editing Research Program

The National Institutes of Health (NIH) will launch an effort aimed at removing barriers that slow the adoption of genome editing for treating patients. This program, Somatic Cell Genome Editing, plans to award researchers approximately $190 million over six years beginning this year, pending availability of funds. [National Institutes of Health] Press Release

Fujifilm Announces the Opening of Its Flexible Manufacturing Facility for the Production of Clinical and Commercial Gene Therapies

FUJIFILM Diosynth Biotechnologies (Fujifilm) announced the opening of its state-of-the art Flexible Manufacturing Facility in College Station, Texas. [FUJIFILM Diosynth Biotechnologies] Press Release

Rexgenero Treats First Patient in Its Phase III Program with Lead Product REX-001, a Novel Cell Therapy for the Treatment of Critical Limb Ischemia (CLI)

Rexgenero Ltd announced the treatment of its first patient in a Phase III program evaluating the company’s lead product, REX-001, in patients with CLI and diabetes mellitus. [Rexgenero Ltd] Press Release

Computer Scientist to Lead French Research Giant; Interim Head Leaves Amid Misconduct Allegations

Computer scientist Antoine Petit, 57, is the new head of Europe’s largest research organization. There was an unusual twist to Petit’s appointment, however. A week before the official procedure ended, the French government named Petit interim president of the National Center for Scientific Research, a position that had been held by cell biologist Anne Peyroche since Fuchs’s departure. [ScienceInsider] Editorial

Gender Bias Goes Away When Grant Reviewers Focus on the Science

But female scientists suffer when their research proposals are judged primarily on the strength of their CVs. [Nature News] Editorial

FDA

Select Updates for Recommendations for Clinical Laboratory Improvement Amendments of 1988 Waiver Applications for Manufacturers of In Vitro Diagnostic Devices; Draft Guidance for Industry and Food and Drug Administration Staff; Availability; Extension of Comment Period (FR Doc. No:2018-01349) Notice

NEW Gordon Research Conferences: Barriers of the CNS
June 17-22, 2018
New London, New Hampshire, United States

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

NEW Lecturer or Senior Lecturer – Gene Therapy (Imperial College London)

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

Postdoctoral Research Scientist – Autophagy and Cell Death in Cancer (Beatson Institute for Cancer Research)

Postdoctoral Fellowships – Cancer Research (University of Miami)

Postdoctoral Scientist – Systems Oncology (Cancer Research UK Manchester Institute)

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

Postdoctoral Research Fellows – Bioengineering and Nanotechnology (University of Queensland)

Research Associate – Immunology and Gene Therapy (Fred Hutchinson Cancer Research Center)

Postdoctoral Associate – Gene Therapy (University of Massachusetts)

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

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