Cell Therapy News 18.14 April 24, 2017 | |
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TOP STORYInvestigators report a gene therapy strategy using CRISPR/Cas9-mediated cellular reprogramming by switching a mutation-venerable/sensitive cell type to a mutation-insensitive/resistant cell type, therefore restoring tissue architecture and function. They applied this strategy to retinitis pigmentosa, a major cause of blindness characterized by retinal rod photoreceptor degeneration caused by numerous mutations in many genes. [Cell Res] Full Article | Press Release | |
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PUBLICATIONS(Ranked by impact factor of the journal)A 3D-Printed Scaffold with MoS2 Nanosheets for Tumor Therapy and Tissue Regeneration Novel bifunctional scaffolds (MS-AKT scaffolds) were successfully fabricated by combining a 3D printing technique with a hydrothermal method. During the hydrothermal process, MoS2 nanosheets were grown in situ on the strut surface of bioceramic scaffolds, endowing them with photothermal therapeutic potential. The MS-AKT scaffolds supported the attachment, proliferation and osteogenic differentiation of bone mesenchymal stem cells and induced bone regeneration in vivo. [NPG Asia Mater] Full Article Scientists hypothesized that induced pluripotent stem cell (iPSC)-regulatory dendritic cells (DCregs) worked as a negative vaccine to generate regulatory T cells (Tregs), and induced donor-specific allograft acceptance. They immunized naive CBA mice with B6 iPS-DCregs and found that Tregs significantly increased in CBA splenocytes. [Stem Cell Reports] Full Article | Graphical Abstract Graphene oxide was introduced as an interface phase to improve the interfacial bonding between polyetheretherketone and hydroxyapatite for tissue engineering scaffolds. In vivo bone defect repair experiments showed that new bone formed throughout the scaffolds at 60 days after implantation. [Sci Rep] Full Article Researchers investigated the potential of a population of nonadherent muscle-derived stem cells (MDSC) from adult mouse muscle to differentiate in vitro into beta cells when transplanted as undifferentiated stem cells in vivo to compensate for beta-cell deficiency. In vivo, undifferentiated MDSC injected into streptozotocin-treated mice engrafted within 48 hours specifically to damaged pancreatic islets and were shown to differentiate and express insulin 10-12 days after injection. [Stem Cell Res Ther] Full Article Scientists tested six different preculture media formulations for their ability to preserve disc-like angle-ply structures composition and metabolic activity during the transition from in vitro culture to in vivo implantation in a subcutaneous athymic rat model. They assayed implants before and after implantation to determine collagen content, glycosaminoglycan content, metabolic activity, and magnetic resonance imaging characteristics. [Tissue Eng Part A] Abstract The authors investigated the outcomes of elderly primary central nervous system lymphoma patients who underwent high-dose chemotherapy followed by autologous stem cell transplantation at 11 centers between 2003 and 2016. End points included remission, progression-free survival, overall survival and treatment-related mortality. [Bone Marrow Transplant] Abstract Investigators established endothelin-1 (EDN-1) overexpressed bone marrow-derived mesenchymal stem cells (BMSCs), proliferation ability and osteogenesis differentiation were detected respectively. Transduced BMSCs were then combined with CPC-scaffold to repair calvarial defects in rats to evaluate the in vivo osteogenic potential of EDN-1. [Exp Cell Res] Abstract An innovative method was conceived to obtain acellular nerve allografts (ANAs), so as to speed up nerve decellularization, without compromising nerve architecture, and without breaking the asepsis chain. A pilot in vivo study was performed: ANAs were implanted into tibial nerve defects of three rabbits, and autografts, representing the gold standard, in other three animals. [J Biomed Mater Res A] Abstract Scientists investigated the efficacy of intra-cerebrospinal fluid delivery of a single-stranded AAV9-CAG-GFP vector in adult mice via intracisternal or intralumbar (it-Lumb) administration. It-Lumb ssAAV9 delivery resulted in greater diffusion throughout the entire spinal cord and green fluorescent protein (GFP) expression mainly in the cerebellum, cortex and olfactory bulb. [Gene Ther] Abstract Preclinical Study of rAAV2-STRAIL: Pharmaceutical Efficacy, Biodistribution and Safety in Animals Researchers evaluated the clinical potency for cancer gene therapy and the safety in mouse and non-human primates. Mouse models with HCT-116, NCI-H460 and BEL-7402 cancers were injected intraperitoneally with a single dose of 1.0×1011, 1.0×1010 and 1.0×109 vg of rAAV2-sTRAIL95–281 virus, respectively. Cynomolgus monkeys were injected with a single dose of rAAV2-sTRAIL95–281 of 1×1011, 3×1011 and 1×1012 vg, corresponding to 6-, 20- and 60-fold of intended use dosage for humans, respectively. [Cancer Gene Ther] Abstract | |
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REVIEWSGenetic Engineering and Manufacturing of Hematopoietic Stem Cells The current state-of-the-art manufacturing platforms to genetically engineer hematopoietic stem cells as well as the challenges pertaining to production standardization and product characterization are addressed in the context of primary immunodeficiency diseases and other monogenic disorders. [Mol Ther Methods Clin Dev] Full Article | Graphical Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSBiogen will present Phase III end of study SPINRAZA® data from CHERISH, which demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset spinal muscular atrophy compared to untreated children. The overall findings continue to support the robust efficacy and favorable safety profile of SPINRAZA across a broad range of individuals with SMA. [Press release from Biogen discussing research to be presented at the 2017 American Academy of Neurology (AAN) Annual Meeting, Boston] Press Release Precision Intratumoral Delivery of DNX-2401 with the Alcyone MEMS Cannula DNAtrix and Alcyone Lifesciences will reveal clinical data demonstrating the precise administration of DNX-2401, an oncolytic adenovirus, into brain tumors using Alcyone’s MEMS Cannula (AMCTM). Neurosurgeon Frederick Lang, MD, FACS, FAANS, will present results from a Phase Ib study confirming reproducible delivery of DNX-2401 by the AMC into recurrent glioblastoma tumors. [Press release from DNAtrix discussing research presented at the 2017 Annual Meeting of the American Association of Neurological Surgeons (AANS), Los Angeles] Press Release Promethera Biosciences Presents Preclinical Data on Novel H2Stem Cell Therapy Promethera Biosciences SA presented novel in vitro and in vivo data on its preclinical pipeline program H2Stem. The poster presentation highlighted H2Stem’s therapeutic potential as a valuable addition to Promethera’s pipeline in liver-directed cell therapies. [Press release from Promethera Biosciences SA discussing research presented at the European Association for the Study of the Liver (EASL) International Liver Congress 2017, Amsterdam] Press Release | |
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INDUSTRY NEWSCellular Dynamics International Signs Distribution Deal with STEMCELL Technologies Cellular Dynamics International (CDI) announced it has signed a distribution agreement with STEMCELL Technologies. Under the terms of the agreement, STEMCELL Technologies will distribute CDI’s iCell® catalog of products in North America, Europe, and Singapore, with other countries under consideration. CDI’s iCell products are differentiated human induced pluripotent stem cell-derived cells, which include cardiomyocytes, hepatocytes, and others, totaling up to 12 cell types. [STEMCELL Technologies Inc.] Press Release Cellular Dynamics International Signs Collaboration Agreement with Harvard Stem Cell Institute Cellular Dynamics International announced it has signed a collaboration agreement with the Harvard University Stem Cell Institute (HSCI), a novel network of stem cell scientists that extends from the University to its affiliated hospitals and partners with the biomedical industry. The objective of the new partnership is to increase the availability of iPS cells and services to the HSCI network and the Boston area research community at large. [Cellular Dynamics International] Press Release Plasticell and Kings College London to Collaborate in Trials of Blood Platelet Substitute Plasticell announced it has partnered with Kings College London to progress preclinical trials of its artificial blood platelet product, manufactured from pluripotent stem cells. The work is supported by a MedCity research grant which funds collaboration between leading SMEs and academics from London universities. [Plasticell] Press Release CRISPR Therapeutics and Casebia Therapeutics announced they have signed a collaboration agreement with StrideBio, LLC, a US-based company developing novel AAV vectors for in vivo gene delivery applications. Under the terms of the agreement, StrideBio will use its proprietary platform to develop AAV vectors with improved properties such as tissue specificity and reduced susceptibility to immune responses. [StrideBio, LLC] Press Release PolarityTEâ„¢, Inc. announced the signing of a manufacturing agreement with Cell Therapy and Regenerative Medicine (CTRM) at the University of Utah School of Medicine. CTRM is the established manufacturer of hematopoietic stem cell transplants for renowned institutions of the Salt Lake region such as the Huntsman Cancer Institute and Primary Children’s Hospital. [PolarityTEâ„¢, Inc.] Press Release Powered by CIRM Grant, jCyte Launches New Clinical Trial jCyte is launching a Phase IIb clinical trial to test the safety and efficacy of its developmental retinitis pigmentosa treatment, jCell. The company is recruiting 70 patients for the single-masked study, which will begin enrollment this month. The multicenter trial is being funded by jCyte, which recently received an $8.3 million matching grant from the California Institute for Regenerative Medicine. [jCyte] Press Release Enzyvant announced that the U.S. Food and Drug Administration (FDA) has granted RVT-802, Enzyvant’s investigational tissue-based therapy for the treatment of complete DiGeorge Syndrome, Breakthrough Therapy designation as well as Regenerative Medicine Advanced Therapy designation. Both designations confer special access to the Office of Tissues and Advanced Therapies for development guidance as well as expedited review pathways. [Enzyvant (PR Newswire Association LLC.)] Press Release Novartis announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies. [Novartis] Press Release BioMarin Pharmaceutical Inc. announced that the Committee for Medicinal Products for Human Use, the scientific committee of the European Medicines Agency, has adopted a positive opinion for the company’s Marketing Authorization Application for Brineuraâ„¢ to treat children with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease, a form of Batten disease, which is also known as tripeptidyl peptidase 1 deficiency. [BioMarin Pharmaceutical Inc.] Press Release uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe uniQure N.V. announced that it will not pursue the renewal of the Glybera® (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017. [uniQure N.V.] Press Release | Editorial Dr. John Lahey, President of Quinnipiac University, Awarded First-Ever ACGT Edward Netter Award Dr. John Lahey, president of Quinnipiac University, was honored at the event with the first ever Edward Netter Award for Business and Industry for his contributions to Alliance for Cancer Gene Therapy (ACGT) and the community. [Alliance for Cancer Gene Therapy (EurekAlert!)] Press Release | |
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POLICY NEWSWhat Happened at March for Science Events around the World Tens of thousands of people gathered in Washington DC, and at least 600 other cities around the world, in what may have been one of the largest-ever demonstrations in support of scientific research and evidence-based policymaking. [Nature News] Editorial French Scientists Focus on the Big Political Picture Researchers in France overwhelmingly oppose the far right and can stamp out its rise by turning out to register their disapproval. [Nature News] Editorial
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REGULATORYFDAOncologic Drugs Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments (FR Doc. No:2017-07772) Notice Medical Imaging Drugs Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments (FR Doc. No:2017-07767) Notice Gastroenterology and Urology Devices Panel of the Medical Devices Advisory Committee; Notice of Meeting (FR Doc. No:2017-08189) Notice
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EVENTSNEW ISSCR Annual Meeting 2017 NEW Gordon Research Conferences: Germinal Stem Cell Biology Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center) Program Director – CART Cell (Servier) Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic) Executive Director – Global CAR-T Supply (Celgene Corporation) Postdoctoral Associate – Gene Therapy (University of Massachusetts Medical School) Postdoctoral Research Fellow – Preclinical Models of Cancer Immunotherapy (Providence Cancer Center) Postdoctoral Fellow – Various Projects (University of Oklahoma) GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center) Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center) 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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Home Cell Therapy News Volume 18.14 | Apr 24 2017