Cell Therapy News 20.12 April 8, 2019 | |
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TOP STORYTen week old Abcb4-/- mice received a single dose of adeno-associated virus (AAV)8-hABCB4 or AAV8-GFP under control of a liver specific promoter via tail vein injection. [J Hepatol] Full Article | Graphical Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)Lentiviral Gene Therapy Corrects Platelet Phenotype and Function in Wiskott-Aldrich Patients The authors carefully investigated platelet (PLT) defects in terms of phenotype and function in untreated Wiskott-Aldrich Syndrome patients and assessed the effect of gene therapy treatment on PLT dysfunction. [J Allergy Clin Immunol] Abstract | Full Article Engraftment of Human Stem Cell Derived Otic Progenitors in the Damaged Cochlea Researchers generated human otic progenitor cells (hOPCs) from induced pluripotent stem cells (iPSCs) in vitro and identified these cells by the expression of known otic markers. They showed successful cell transplantation of iPSC-derived-hOPCs in an in vivo adult guinea pig model of ototoxicity. [Mol Ther] Abstract Scientists investigated the effect of tumor necrosis factor α (Tnfα) RNA interference by introducing a functional and highly safe carbon dot (CD)‐SMCC nanovector synthesized by bioconjugation of CDs with a protein crosslinker, sulfosuccinimidyl‐4‐(N‐maleimidomethyl) cyclohexane‐1‐carboxylate, as the vehicle of the silenced TNFα on chondrogenesis of mesenchymal stem cells. [Stem Cells Transl Med] Full Article Researchers aimed to establish the effects of human bone marrow-derived endothelial progenitor cells systemically transplanted into G93A mice at symptomatic disease stage. [Sci Rep] Full Article Investigators developed a suicide approach using a dual gene expression two step transcriptional amplification lentiviral vector encoding BMP-2 and an inducible caspase 9 system that selectively induced apoptosis upon activation with a chemical inducer of dimerization. [Gene Ther] Abstract Transduction efficiency, transgene expression and cellular tropism were compared for the adeno-associated viral (AAV) serotypes AAV5, AAV9 and AAVRec2 whereby destabilized yellow fluorescent protein was controlled by the GFAP or the truncated GfaABC1D promoter. The vectors were tested in primary spinal cord astrocyte cell culture, spinal cord slice culture and an in vivo model of spinal cord injury contusion. [Gene Ther] Full Article In Situ Cross-Linking Hydrogel as a Vehicle for Retinal Progenitor Cell Transplantation The authors tested the hypothesis that gelatin-hydroxyphenyl propionic acid (Gtn-HPA) hydrogel supports survival and integration of retinal progenitor cells (RPCs) post-transplantation. In vitro compatibility and in vivo graft survival were assessed by mixing an equal volume of Gtn-HPA conjugate and RPC cell suspension and triggering enzyme-mediated gelation, using minute amounts of horseradish peroxidase and peroxide. [Cell Transplant] Full Article Mesenchymal Stem Cell Therapy Improves Spatial Memory and Hippocampal Structure in Aging Rats Investigators assessed the effect of intracerebroventricular administration of human bone marrow-derived mesenchymal stem cells on spatial memory and hippocampal morphology of senile female rats, using three-months-old counterparts as young controls. [Behav Brain Res] Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSCurrent Status of Autologous Stem Cell Transplantation for Multiple Myeloma More than 30 years after its introduction, autologous stem cell transplantation remains the standard of care for young patients with newly diagnosed multiple myeloma. [Blood Cancer J] Full Article Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSSarepta Therapeutics, Inc. stated that its previously announced positive clinical data from the company’s Limb-girdle muscular dystrophy Type 2E gene therapy program has been accepted for a late-breaker oral presentation. [Press release from Sarepta Therapeutics, Inc. discussing research to be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, Orlando] Press Release Nkarta Therapeutics announced preclinical data were presented in a poster session. [Press release from Nkarta Therapeutics discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release Rubius Therapeutics, Inc. announced that the company presented preclinical data from its emerging oncology pipeline of Red Cell Therapeutics™ [Press release from Rubius Therapeutics, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release Homology Medicines, Inc. announced preclinical data demonstrating that HMI-102, its gene therapy candidate for the treatment of phenylketonuria (PKU), restored the normal phenylalanine metabolic pathway as evidenced by measurements of key biomarkers in the disease model. [Press release from Homology Medicines, Inc. discussing research presented at the 2019 ACMG Annual Clinical Genetics Meeting, Seattle] Press Release | |
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INDUSTRY NEWSRegeneron Pharmaceuticals, Inc. and Alnylam Pharmaceuticals, Inc. announced a collaboration to discover, develop and commercialize new RNAi therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and CNS, in addition to a select number of targets expressed in the liver. [Regeneron Pharmaceuticals, Inc.] Press Release Orgenesis Inc. announced that it has entered into a collaboration agreement with Digilab Inc. to develop a live cell printing process and systems designed to automate the production of three-dimensional live cellular structures and tissues. [Orgenesis Inc.] Press Release Sangamo Therapeutics, Inc. announced recent progress across its clinical portfolio, including early data for ST-400, an ex vivo gene-edited beta thalassemia cell therapy developed in partnership with Sanofi, and next steps for its in vivo genome editing programs. [Sangamo Therapeutics, Inc.] Press Release University of Birmingham Trials a New Stromal Cell Immunotherapy for Chronic Liver Disease Up to 56 patients are being recruited to take part in the MERLIN trial, which will investigate the safety and efficacy of a new cellular immunotherapy in patients with either primary sclerosing cholangitis or autoimmune hepatitis. [University of Birmingham] Press Release UC San Diego Health Treats 1st Cancer Patient with Stem-Cell Derived Natural Killer Cells In February 2019, as part of a Phase I clinical trial at Moores Cancer Center at UC San Diego Health, Ruff became the first patient in the world to be treated for cancer with a human-induced pluripotent stem cell-derived cell therapy called FT500. [UC San Diego Health] Press Release Applied DNA Sciences, Inc. announced that LineaRx has achieved anti-CD-19 CAR expression in human T cells via its proprietary, non-viral, plasmid-free manufacturing platform. [Applied DNA Sciences, Inc.] Press Release Homology Medicines, Inc. announced that the FDA has provided clearance for Homology to begin its Phase I/II pheNIX clinical trial with HMI-102, a one-time gene therapy candidate for adults with the rare genetic disease phenylketonuria (PKU), an inborn error of metabolism. [Homology Medicines, Inc.] Press Release Abeona Therapeutics Inc. announced that the FDA has granted Fast Track designation to ABO-101, the company’s novel one-time gene therapy for MPS IIIB. [Abeona Therapeutics Inc.] Press Release The Janssen Pharmaceutical Companies of Johnson & Johnson announced that the European Medicines Agency has granted a PRIME designation for the company’s investigational B-cell maturation antigen chimeric antigen receptor (CAR) T-cell therapy, JNJ-68284528. [The Janssen Pharmaceutical Companies (Business Wire, Inc.)] Press Release FDA Clears the IND for UCARTCS1, the First Allogeneic CAR-T to Treat Multiple Myeloma Patients Cellectis announced that the FDA has approved the company’s Investigational New Drug (IND) application to initiate a Phase I clinical trial for UCARTCS1, in patients with multiple myeloma. [Cellectis] Press Release Audentes Therapeutics, Inc. announced it has expanded its scientific platform and pipeline to advance vectorized antisense treatments for the treatment of Duchenne muscular dystrophy and myotonic dystrophy type 1. [Audentes Therapeutics, Inc.] Press Release uniQure N.V. announced that the FDA has granted Fast Track designation for AMT-130, the company’s gene therapy candidate for the treatment of Huntington’s disease. [uniQure N.V.] Press Release | |
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POLICY NEWSBrazil’s Government Freezes Nearly Half of Its Science Spending Researchers in Brazil are up in arms after President Jair Bolsonaro’s government announced late March that it had frozen 42% of the budget for the country’s science and communications ministry. [Nature News] Editorial FDA, New York Attorney General Go After Stem Cell Clinics On April 3 the FDA sent letters to 20 companies providing stem cell treatments, reports The New York Times, reminding them that they may require FDA approval and should take action to comply. [The Scientist] Editorial FTC Hits Predatory Scientific Publisher with a $50 Million Fine The Federal Trade Commission (FTC) has won a summary judgement that just might cause some predatory publishers to step back from their business model. An India-based predatory publisher has been hit with a $50 million dollar judgement for deceptive business practices, along with permanent injunctions against most of the activities that made it money. [Ars Technica] Editorial Bioethicists Concerned over Japan’s Chimera Embryo Regulations Japanese regulators have effectively given the green light to research involving human-animal chimera embryos. Many researchers see the move to relax the rules as a welcome change, yet some are worried the revisions don’t take public concerns enough into consideration. [The Scientist] Editorial
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REGULATORYFDAStatement by FDA Commissioner Scott Gottlieb, M.D., and Biologics Center Director Peter Marks, M.D., Ph.D. on FDA’s Continued Efforts to Stop Stem Cell Clinics and Manufacturers from Marketing Unapproved Products that Put Patients at Risk, While Progressing the Agency’s Commitment to Help Advance Legitimate Stem Cell Product Development under Existing Agency Regulations (FR Doc. No:PressAnnouncements) Notice
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EVENTSNEW 17th World Congress of the International Pancreas & Islet Transplant Association (IPITA 2019) Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Gene Editing Specialist – Rare Genetic Disorders (Novo Nordisk) Research Technologist – Immunology (STEMCELL Technologies Inc.) Staff Scientist – Development of Mechanism-Based Tumor Therapies (Frankfurt Cancer Institute) Cell Biologist – Regenerative Medicine (Hepatx Corporation) Stem Cell Podcast Co-Host (Stem Cell Podcast) Associate Scientist – Immuno-Oncology (HMH Center for Discovery & Innovation) Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma) Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center) Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University) Faculty Positions – Hematology and Oncology (University of California, Davis) Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.
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