Cell Therapy News 20.21 June 17, 2019 | |
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TOP STORYNew Gene Editor Harnesses Jumping Genes for Precise DNA Integration A new discovery could fix one of the major shortcomings of current gene-editing tools, including CRISPR, and offer a powerful new approach for genetic engineering and gene therapy. [Press release from Columbia University Irving Medical Center discussing online prepublication in Nature] Press Release | Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)Increasing the Efficacy of Stem Cell Therapy via Triple-Function Inorganic Nanoparticles In vivo rodent studies showed that labeling stem cells with a nanoparticle increased the efficacy of stem cell therapy in a ligation/reperfusion model. [ACS Nano] Abstract | Graphical Abstract In a gastric cancer NSG mice model, meso3 CAR T cells mediated stronger antitumor responses than meso1 CAR T cells did. Scientists identified that meso3 CAR T cells could effectively inhibit the growth of large ovarian tumors in vivo. [Cell Death Dis] Full Article Researchers report the development of immunotherapy using human lymphocytes transduced with a codon-optimized melanoma-associated antigen-A4 and HLA-A*2402-restricted T-cell receptor (TCR), which specifically downregulated endogenous TCR by small interfering RNA. [Cell Death Dis] Full Article Scientists tested synthetic vectors based on an adeno-associated virus platform for their potential anti-tumorigenic effect in human hepatocellular carcinoma (HCC) cells in vitro and in a HCC tumor model developed in nude mice. [Bioconjug Chem] Abstract Traceless Aptamer-Mediated Isolation of CD8+ T Cells for Chimeric Antigen Receptor T-Cell Therapy Captured CD8+ T cells were released label-free by complementary oligonucleotides that underwent toehold-mediated strand displacement with the aptamer. The authors showed that chimeric antigen receptor T cells manufactured from these cells were comparable to antibody-isolated chimeric antigen receptor T cells in proliferation, phenotype, effector function and antitumor activity in a mouse model of B-cell lymphoma. [Nat Biomed Eng] Abstract Human umbilical cord-derived mesenchymal stem cells pretreated with or without angiotensin-II were delivered through the tail vein of rats 12 hours after induction of severe acute pancreatitis. Pancreatitis severity scores and serum lipase levels, as well as the levels of VEGF and VEGFR2 were evaluated. [Biomed Pharmacother] Full Article Mesenchymal Stem Cells Decrease Oxidative Stress in the Bowels of Interleukin-10 Knockout Mice Human bone marrow-derived mesenchymal stem cells were injected into IL-10 KO mice. Oxidative stress and inflammation levels were evaluated in the large intestine and compared with those in control IL-10 KO mice and normal wild-type control mice. [Gut Liver] Abstract | Download Full Article Scientists investigated the use of mesenchymal stem cells in four dogs with neurological lesions caused by the distemper virus. [Heliyon] Full Article Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSAutophagy: A Potential Key Contributor to the Therapeutic Action of Mesenchymal Stem Cells The authors provide an overview of studies which highlight a possible link between the therapeutic properties of mesenchymal stem cells and their ability to modulate autophagy, and they summarize examples of disorders where these therapeutic properties have been correlated with such modulation. [Autophagy] Abstract | Graphical Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSbluebird bio Presents New Data for LentiGlobin® Gene Therapy for Sickle Cell Disease (SCD) bluebird bio, Inc. announced new data from patients in Group C of its ongoing Phase I/II HGB-206 study of the company’s investigational LentiGlobin® gene therapy for sickle cell disease. [Press release from bluebird bio, Inc. discussing research presented at the 24th European Hematology Association (EHA) Congress, Amsterdam] Press Release | |
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INDUSTRY NEWSNew CRISPR Lab to Accelerate Drug Discovery, Advance Genomic Research GlaxoSmithKline plc has launched a five-year, $67 million collaboration with the San Francisco and Berkeley campuses of the University of California to build a state-of-the-art laboratory that will use CRISPR technologies to explore how genes cause disease and to rapidly accelerate the discovery of new medicines. [University of California, San Francisco] Press Release SIRION Biotech GmbH announced the entry into a license agreement with the National Institute of Allergy and Infectious Diseases (NIAID) pursuant to which NIAID has licensed SIRION’s LentiBOOST™ technology to improve clinical efficacy of its hematopoietic stem cell gene therapy programs. [SIRION Biotech GmbH] Press Release Voyager Therapeutics, Inc. announced a restructuring of its gene therapy relationship with Sanofi Genzyme. [Voyager Therapeutics, Inc.] Press Release IVERIC bio, Inc. and Catalent Biologics announced that they have entered into an agreement for production and manufacturing of GMP-grade adeno-associated virus vector for IVERIC bio’s gene therapy product candidates, IC-100 for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa and IC-200 for the treatment of BEST1 related retinal diseases. [IVERIC bio, Inc.] Press Release Bone Therapeutics announced that its allogeneic cell therapy product, ALLOB, successfully completed the 12 months follow-up period of a Phase IIa study in patients undergoing a lumbar spinal fusion procedure. [Bone Therapeutics S.A.] Press Release AlloVir announced that it has received RMAT designation from the FDA for Viralym-M, its lead allogeneic, off-the-shelf, multi-virus specific T-cell therapy, for the treatment of hemorrhagic cystitis caused by BK virus in adults and children following allogeneic hematopoietic stem cell transplantation (HSCT). [AlloVir] Press Release Krystal Biotech Inc. announced that it has submitted an IND application with the FDA to initiate a Phase I/II, first in-human trial of KB105, an HSV-1 based gene therapy engineered to deliver a human transglutaminase-1 (TGM1) gene to patients with TGM1-deficient autosomal recessive congenital ichthyosis. [Krystal Biotech Inc.] Press Release Prevail Therapeutics Inc. announced the FDA has accepted the company’s Investigational New Drug (IND) application for its lead gene therapy program, PR001. [Prevail Therapeutics Inc.] Press Release First Patient Dosing in Phase I Clinical Trial of Anti-CD20 CAR-T for Lymphoma in China Cellular Biomedicine Group Inc. announced that it has initiated its Phase I clinical trial of anti-CD20 CAR-T targeting anti-CD19 treated, relapsed diffuse large B-cell lymphoma (DLBCL) and small B-cell lymphoma patients in China, and dosed the first CD19 CAR-T relapsed DLBCL patient. [Cellular Biomedicine Group Inc.] Press Release Ziopharm Oncology Announces FDA Clearance of IND for Sleeping Beauty TCR-T Cell Therapy Trial at NCI Ziopharm Oncology, Inc. announced that the investigational new drug (IND) application submitted by the National Cancer Institute (NCI) has received clearance from the FDA for a clinical trial in solid tumors to evaluate T-cell receptor (TCR) T-cell therapy utilizing Ziopharm’s Sleeping Beauty platform. [Ziopharm Oncology, Inc.] Press Release Orgenesis Inc. announced that the FDA has granted Orphan Drug designation for its Autologous Insulin Producing cells as a cell replacement therapy for the treatment of severe hypoglycemia-prone diabetes resulting from total pancreatectomy due to chronic pancreatitis. [Orgenesis Inc.] Press Release CASI Pharmaceuticals Acquires Worldwide Rights to Commercialize Anti-CD19 T-Cell Therapy CASI Pharmaceuticals, Inc. announced the signing of a license agreement for exclusive worldwide license and commercialization rights to an autologous anti-CD19 T-cell therapy product from Juventas Cell Therapy Ltd. [CASI Pharmaceuticals, Inc.] Press Release MDA Awards Venture Philanthropy Grant to Locana to Develop Novel Treatment for Muscular Dystrophy The Muscular Dystrophy Association (MDA) and Locana announced the award of an MDA Venture Philanthropy grant totaling $550,000 to advance Locana’s development program for myotonic dystrophy. [The Muscular Dystrophy Association] Press Release | |
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POLICY NEWSStem Cell Clinics Co-Opt Clinical-Trials Registry to Market Unproven Therapies, Critics Say A few weeks ago, if you’d been scouring the ever-expanding mass of digitized federal paperwork, you might have noticed a contradiction. On the one hand, the FDA issued a letter stating that what an Arizona distributor was selling as stem cell therapies were “unapproved” and posed “safety concerns.” On the other, a National Institutes of Health database — clinicaltrials.gov — went right on listing the same merchant’s studies, with a link to the company’s website and the word “Recruiting” displayed invitingly in green. [STAT News] Editorial Trump Issues Executive Order that Could Reduce Scientific Advice President Donald Trump issued an executive order on June 14 to cut back the number of advisory committees by at least a third by the end of September, according to a statement from the White House. The federal advisory committee system is the formal structure for government agencies to seek advice from experts including scientists. [The Scientist] Editorial China Clamps Down on Foreign Use of Chinese Genetic Material and Data China has announced new regulations for the use of human genetic resources that come from Chinese participants. The rules, announced on June 10, require international scientists using biomaterials from China to have a Chinese collaborator and go into effect on July 1. [The Scientist] Editorial
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REGULATORYFDARequest for Nominations from Industry Organizations Interested in Participating in the Selection Process for Nonvoting Industry Representatives and Request for Nominations for Nonvoting Industry Representatives on the Cellular, Tissue, and Gene Therapies Advisory Committee (FR Doc. No:2019-12565) Notice NIHProspective Grant of an Exclusive Patent License: Development and Commercialization of Cell Therapies for Cancer (FR Doc. No:2019-12707) Notice
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EVENTSNEW 7th International Stem Cell Meeting Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Postdoctoral Scientist – Computational Biologist (Cedars-Sinai Medical Center) Stem Cell Podcast Co-Host (Stem Cell Podcast) Senior Scientist – Antigen Presentation (Astellas Pharma) Postdoctoral Research Fellowships – Kidney Disease (Vanderbilt University Medical Center) Postdoctoral Scholarship – Gene Therapy for Osteopetrosis (Lund University) Postdoctoral Fellow – Hematopoietic Stem Cells and Gene Therapy (UC San Diego) Postdoctoral Position – Biomedical Engineering (US Food and Drug Administration) Postdoctoral Research Fellow – Translational Gene Therapy (Fred Hutchinson Cancer Research Center) 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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