Cell Therapy News Volume 19.39 | Nov 26 2018

TOP STORY

Linking a Cell-Division Gene and a Suicide Gene to Define and Improve Cell Therapy Safety

Using genome-engineering strategies, the authors demonstrated the protection of a suicide system from inactivation in dividing cells. They created a transcriptional link between the suicide gene herpes simplex virus thymidine kinase and a cell-division gene; this combination is designated the safe-cell system. [Nature] Abstract | Press Release

(Ranked by impact factor of the journal)

GM-CSF Inhibition Reduces Cytokine Release Syndrome and Neuroinflammation but Enhances CAR-T Cell Function in Xenografts

Researchers showed that GM-CSF neutralization with lenzilumab does not inhibit chimeric antigen receptor T cell (CART)19 function in vitro or in vivo. [Blood] Abstract

Epigenetically Modified Cardiac Mesenchymal Stromal Cells Limit Myocardial Fibrosis and Promote Functional Recovery in a Model of Chronic Ischemic Cardiomyopathy

Investigators examined the effects of pharmacologic HDAC1 inhibition, using the benzamide class 1 isoform-selective HDAC inhibitor entinostat, on cardiac mesenchymal cell (CMC) cardiomyogenic lineage commitment and CMC-mediated myocardial repair in vivo. [Basic Res Cardiol] Abstract

An Investigational RNAi Therapeutic Targeting Factor XII (ALN-F12) for the Treatment of Hereditary Angioedema

A subcutaneously administered GalNAc-conjugated small interfering RNA targeting F12 mRNA (ALN-F12) was developed and potency was evaluated in mice, rats and cynomolgus monkeys. The effect of FXII reduction by ALN-F12 administration was evaluated in two different vascular leakage mouse models. [RNA] Abstract

Co-Transplantation of Mesenchymal Stem Cells Improves Spermatogonial Stem Cell Transplantation Efficiency in Mice

Co-transplantation of mesenchymal stem cells might increase colonization efficiency of spermatogonial stem cells (SSCs) by restoring the SSC niche after gonadotoxic treatment. [Stem Cell Res Ther] Full Article

Therapeutic Efficiency of Human Amniotic Epithelial Stem Cell-Derived Functional Hepatocyte-Like Cells in Mice with Acute Hepatic Failure

Hepatocyte-like cells were transplanted into acute liver failure non-obese diabetic severe combined immunodeficient mice, and the therapeutic effects were determined via liver function test, histopathology, and survival rate analysis. [Stem Cell Res Ther] Full Article

Effects of Physiological Aging Factor on Bone Tissue Engineering Repair Based on Fetal BMSCs

Scientists studied whether aging factor has influence on the skull repair effect of fetal-tissue engineered bone graft, at the level of the large animal models. They used the fetal bone marrow stromal cells (Fetal-BMSCs) as the seed cells, combining the decalcified bone matrix scaffolds, to repair the skull defects of the aged goats and the young goats. [J Transl Med] Full Article

Efficiency of Coculture with Angiogenic Cells or Physiological BMP-2 Administration on Improving Osteogenic Differentiation and Bone Formation of MSCs

Investigators evaluated the efficiency of coculture with angiogenic cells or a physiological dose of bone morphogenetic protein 2 (BMP-2) on improving osteogenic differentiation of mesenchymal stem cells (MSCs) and bone formation in vivo. [J Biomed Mater Res A] Abstract

DRG-Derived Neural Progenitors Differentiate into Functional Enteric Neurons following Transplantation in the Postnatal Colon

The authors showed that neural progenitors derived from the dorsal root ganglion (DRG) of EGFP mice survived, differentiated into enteric neurons and glia cells, migrated widely from the site of injection, and established neuron-muscle connections following transplantation into the distal colon of postnatal mice. [Cell Transplant] Full Article

Role of IL-7 in Fusion of Rat Bone Marrow Mesenchymal Stem Cells with Cardiomyocytes In Vitro and Improvement of Cardiac Function In Vivo

Researchers used interleukin-7 (IL-7), a cell survival growth factor, to enhance the potential of rat bone marrow mesenchymal stem cells in terms of cell fusion in vitro and cardiac function in vivo. [Cardiovasc Ther] Abstract

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Myths, Reality and Future of Mesenchymal Stem Cell Therapy

The authors consolidate the in vivo effects of mesenchymal stem cell (MSC) therapy, discuss the fate of MSCs after intravascular and local delivery and propose possible trends in MSC therapy. [Cell Tissue Res] Abstract

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Sensorion Enters into Exclusive Negotiations with Pasteur Institute for Hearing Loss Gene Therapy Programs

Sensorion announced that it has entered into a letter of intent with Pasteur Institute to exclusively negotiate a framework agreement to obtain the exclusive licenses to develop and commercialize gene therapy product candidates for restoration, treatment and prevention of hearing loss disorders. [Sensorion] Press Release

Orgenesis Announces Collaboration with the New York Blood Center for Tissue Collection, Processing and Biobanking U.S. Cell Therapy Development Program

Orgenesis Inc. announced that it has entered into a collaboration agreement with the New York Blood Center (NYBC). Under the agreement, NYBC will be responsible for establishing the infrastructure to collect, process, test, cryopreserve and biobank sampling materials, including liver biopsies. [Orgenesis Inc.] Press Release

Vineti™ to Partner with Tessa Therapeutics™ to Advance and Scale Cancer Immunotherapies World-Wide

Vineti, Inc. announced a new partnership with Tessa Therapeutics to advance and scale personalized cell therapies for solid tumors in Asia and world-wide. [Vineti, Inc. (Business Wire, Inc.)] Press Release

Kolon TissueGene Doses First Patient in US Phase III Clinical Trial

Kolon TissueGene, Inc. announced that it has dosed its first patient in its pivotal US Phase III clinical trials for Invossa, a cell and gene therapy in development for knee osteoarthritis. [Kolon TissueGene, Inc.] Press Release

Research Highlights the UK’s Growing Industry and Infrastructure for Manufacturing Cell and Gene Therapies

New research indicates that the UK’s cell and gene therapy industry continues to strengthen, as private and government investment has led to an increase in manufacturing space and jobs in the sector within the past year. [Cell and Gene Therapy Catapult] Press Release

Sernova Highlights Achievements from the HemAcure Consortium in Developing a Novel Cell-Based Therapy for Hemophilia A

Sernova Corp. highlighted the achievements in developing a novel cell-based therapy in combination with Sernova’s Cell Pouch™ for treatment of Hemophilia A. [Sernova Corp.] Press Release

NapaJen Pharma Announces First Patient Dosed in Phase I First-in-Human Clinical Trial of Novel RNA Interference (RNAi) Therapeutic

NapaJen Pharma Co., Ltd. announced dosing of the first patient in a Phase I first-in-human clinical trial of NJA-730 in healthy volunteers. [NapaJen Pharma Co., Ltd. (GlobeNewswire, Inc.)] Press Release

European Commission Approves Spark Therapeutics’ LUXTURNA^® (Voretigene Neparvovec), a One-Time Gene Therapy for Inherited Retinal Disease Caused by Confirmed Biallelic RPE65 Mutations

Spark Therapeutics announced that the European Commission has granted marketing authorization for LUXTURNA^®, a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. [Spark Therapeutics, Inc.] Press Release

BrainStorm Cell Therapeutics Announces Submission of IND for NurOwn^® in Progressive Multiple Sclerosis

BrainStorm Cell Therapeutics Inc. announced that it has submitted an Investigational New Drug (IND) application with the FDA to initiate a Phase II study of NurOwn^® in patients with progressive multiple sclerosis. [BrainStorm Cell Therapeutics Inc.] Press Release

Vir Biotechnology and Alnylam Pharmaceuticals Initiate Phase I/II Study of VIR-2218

Vir Biotechnology, Inc. and Alnylam Pharmaceuticals, Inc. announced the initiation of a Phase I/II study of VIR-2218, a novel, investigational RNA interference therapeutic for the treatment of chronic hepatitis B virus infection. [Vir Biotechnology, Inc.] Press Release

Rocket Pharmaceuticals Announces Preclinical Data for AAV-Based RP-A501, the First Investigational Gene Therapy Program for a Monogenic Heart Failure Syndrome

Rocket Pharmaceuticals, Inc. announced it is developing its first adeno-associated viral vector (AAV)-based gene therapy, RP-A501, that is designed to restore the lysosome-associated membrane glycoprotein 2 gene which is defective in patients afflicted with Danon disease. [Rocket Pharmaceuticals, Inc.] Press Release

Crashing the Boards: Neuroscientist Maureen Condic Brings a Different Voice to NSF Oversight Body

The National Science Foundation (NSF) will welcome the first cohort of members appointed by President Donald Trump to its oversight body, the National Science Board. Most of the seven fit the mold of senior academic leaders, prominent scientists, and corporate research managers who typically sit on the 24-member board. But Maureen Condic is somewhat different. [ScienceInsider] Editorial

A 100^th Birthday Wish: Uphold Academic Freedom in Dark Times

One hundred years ago this month, shortly after the guns of the First World War fell silent, a German-speaking Scottish lawyer-turned-politician sent an 80-page report to his prime minister. In it was an idea whose echo still shapes the way in which many nations fund research — an idea arguably as important to the soul of modern science as the secular state is to modern democracy. [Nature News] Editorial

Genome-Edited Baby Claim Provokes International Outcry

A Chinese scientist claims that he has helped make the world’s first genome-edited babies — twin girls who were born this month. The announcement has provoked shock, and some outrage, among scientists around the world. [Nature News] Editorial

NEW 2^nd Pan-Canadian Strategic Forum
March 13-15, 2019
Toronto, Ontario, Canada

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NEW Postdoctoral Associate – Gene Therapy (University of Massachusetts)

PhD Scholarship – iPS Cells and Neurodegenerative Disease (TU Dresden)

Research Scientist – Cellular Manufacturing (California Institute for Biomedical Research)

Assistant Project Scientist – Hematology and Oncology (University of California, Davis)

Senior Research Officer – Therapies for Neurodegenerative Disorders (University of Edinburgh)

Executive Director – Cell and Gene Medicine (Stanford University)

Faculty Positions – Hematology and Oncology (University of California Davis)

Lab Technologist – Cell Therapy (University of Maryland Medical Center)

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

Open Rank Professor – Biopharmaceutical Discovery (University of Delaware)

Assistant Professor – Biology (University of Delaware)

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