Cell Therapy News 20.02 January 21, 2019 | |
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TOP STORYScientists treated three adults and six children with absent or severe reduced mutations in a Phase I/II trial with an intrabone administration of hematopoietic stem cells transduced with the lentiviral vector GLOBE. [Nat Med] Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)Development of a Gene-Editing Approach to Restore Vision Loss in Leber Congenital Amaurosis Type 10 Researchers developed EDIT-101, a candidate genome-editing therapeutic, to remove the aberrant splice donor created by the IVS26 mutation in the CEP290 gene and restore normal CEP290 expression. Key to this therapeutic, they identified a pair of Staphylococcus aureus Cas9 guide RNAs that were highly active and specific to the human CEP290 target sequence. [Nat Med] Abstract Scientists demonstrated a strategy for autologous hematopoietic stem cell (HSC) transplant and gene therapy utilizing a lentiviral vector to restore FoxP3 expression under the control of endogenous human FOXP3 regulatory elements. [Cell Stem Cell] Abstract | Press Release | Graphical Abstract Compared to retinal pigment epithelium (RPE) cells in suspension, a biodegradable scaffold approach improved integration and functionality of RPE patches in rats and in a porcine laser-induced RPE injury model that mimicked age-related macular degeneration-like eye conditions. [Sci Transl Med] Abstract | Press Release Timed GDNF Gene Therapy Using an Immune-Evasive Gene Switch Promotes Long Distance Axon Regeneration Investigators report that time-restricted glial cell line-derived neurotrophic factor (GDNF) expression using an immune-evasive doxycycline-inducible gene switch attenuated local axon entrapment in avulsed reimplanted ventral spinal roots, was sufficient to promote long-term motor neuron survival and facilitated the recovery of compound muscle action potentials by eight weeks. [Brain] Full Article | Press Release Gene Therapy Targeting SARM1 Blocks Pathological Axon Degeneration in Mice To assess efficacy in vivo, the authors used adeno-associated virus-mediated expression of the most potent SARM1 dominant-negative and nerve transection as a model of severe axonal degeneration. [J Exp Med] Abstract | Press Release Researchers showed that NKG2D.z-natural killer (NK) cells were cytotoxic against myeloid‑derived suppressor cells (MDSCs), but spared NKG2D ligand‑expressing normal tissues. [Cancer Immunol Res] Abstract Antagonism of IAPs Enhances CAR T Cell Efficacy Investigators demonstrated that antagonizing the inhibitor of apoptosis proteins (IAPs) with the clinical smac-mimetic, birinapant, significantly enhanced the antitumor activity of CAR T cells in a TNF-dependent manner. Enhanced tumor cell death occurred independently of the perforin-mediated granule exocytosis pathway, underscoring the cytotoxic potential of CAR T cell-derived TNF. [Cancer Immunol Res] Abstract Researchers investigated whether cell survival and synaptic transmission within an NT-3 and TRKC gene-overexpressing neural stem cell-derived neural network scaffold transplanted into transected spinal cord could be promoted by electroacupuncture through improving the microenvironment. [Stem Cell Reports] Full Article Investigators demonstrated that heparan sulfate mimetic injections that restore the extracellular matrix network and enhance the biological activity of growth factors, associated with local injection of mesenchymal stromal cell (MSC) protected in a hydrogel, that increased cell engraftment and cell survival, improved the therapeutic benefit of MSC treatment in two animal models relevant of the human pathology. [Sci Rep] Full Article Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWST-Cells “à la CAR-T(e)” – Genetically Engineering T-Cell Response against Cancer The authors give an overview of the most recent developments in the field of T-cell genetic engineering including TCR-gene transfer and CAR T-cells strategies. [Adv Drug Deliv Rev] 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 NEWSThe Center for Cell and Gene Therapy at Baylor College of Medicine to Present Marker Therapeutics, Inc. reported that data from four abstracts—including three oral presentations—were accepted for presentation. [Press release from Marker Therapeutics, Inc. discussing research presented at the Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research (ASBMT and CIBMTR), Houston] Press Release | |
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INDUSTRY NEWSKyn Therapeutics announced it has entered into a global strategic collaboration with Celgene Corporation. [Kyn Therapeutics] Press Release Sernova Corp. announced the enrollment of the first three of seven subjects in its Phase I/II trial of Sernova’s Cell Pouch™ for Clinical Islet Transplantation in type 1 diabetes. [Sernova Corp.] Press Release Cellular Biomedicine Group, Inc. announced that its off-the-shelf allogeneic adipose-derived mesenchymal progenitor cell AlloJoin® therapy for KOA has been approved as the first stem cell KOA drug application in China for a Phase II clinical trial. [Cellular Biomedicine Group, Inc.] Press Release AVROBIO, Inc. announced that its investigational gene therapy candidate for cystinosis, AVR-RD-04, is cleared to begin a Phase I/II clinical trial, following acceptance of the IND application by the FDA. [AVROBIO, Inc.] Press Release PharmaCyte Biotech Announces Encapsulation of Live Cells for Clinical Trial in Pancreatic Cancer PharmaCyte Biotech, Inc. announced PharmaCyte’s partner, Austrianova, has successfully encapsulated the live cells used in PharmaCyte’s therapy for its planned clinical trial in patients with locally advanced, non-metastatic, inoperable pancreatic cancer. [PharmaCyte Biotech, Inc.] Press Release Thyas Co. Ltd. signed a licensing agreement with iPS Academia Japan, Inc. Under the agreement, Thyas obtains exclusive rights on patents related to production and use of iPS cell-derived T cells, and non-exclusive rights on the patents related to iPS cell generation, for development and commercialization of autologous immunotherapy of cancers and infectious diseases. [Thyas Co. Ltd. (GlobeNewswire, Inc.)] Press Release Krystal Biotech Inc. announced that construction of Ancoris, a new state-of-the-art Good Manufacturing Practice (GMP) facility, is complete. [Krystal Biotech Inc.] Press Release FDA Planning New Guidance, Hires in Cell and Gene Therapies for 2019 The FDA plans to hire at least 50 new clinical reviewers tasked with assessing cell and gene therapies to prepare for what the agency describes as a surge of cutting-edge products currently entering early development. [FierceBiotech] Press Release | |
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POLICY NEWSSurprise! Shutdown also Disrupting U.S. Science Agencies that Aren’t Closed Many U.S. government scientists and federally funded researchers breathed a sigh of relief last month, after the partial shutdown of the U.S. government began. That’s because the budget impasse between Congress and President Donald Trump didn’t affect some of the largest federal research agencies, including the $39 billion National Institutes of Health (NIH) and the $35.6 billion Department of Energy (DOE). [Science Insider] Editorial How UK Scientists Are Planning for a Chaotic No-Deal Brexit British scientists are ramping up preparations for a ‘no deal’ Brexit on 29 March — a situation that would instantly affect travel, data collection, clinical trials and crucial laboratory supplies. [Nature News] Editorial
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REGULATORYFDAStatement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on New Policies to Advance Development of Safe and Effective Cell and Gene Therapies (FR Doc. No:PressAnnouncements) Notice
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EVENTSNEW 2nd London Stem Cell Network Symposium Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Cell Manufacturing Scientist (CSL Behring) NEW Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University) Postdoctoral Fellow – Inherited Retinal Degenerations (Ocular Genomics Institute) Postdoctoral Position – Pluripotent Stem Cells and Rare Diseases (I-STEM) Postdoctoral Fellow – Genome and Metabolic Engineering (Columbia University Medical Center) Postdoctoral Research Associate – Gene Therapy (University of Massachusetts) Research Fellow – Stem Cell Biology (Brigham & Women’s Hospital – Harvard Medical School) Faculty Position – Cancer Bioengineering (Ecole Polytechnique Fédérale de Lausanne) Assistant Project Scientist – Hematology and Oncology (University of California, Davis) 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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