Cell Therapy News Volume 19.34 | Oct 15 2018

TOP STORY

Treatment of a Metabolic Liver Disease by In Vivo Genome Base Editing in Adult Mice

Researchers corrected the disease phenotype of adult phenylalanine hydroxylase Pah^enu2 mice, a model for the human autosomal recessive liver disease phenylketonuria 1, using recently developed CRISPR-Cas-associated base editors. [Nat Med] Abstract | Press Release

(Ranked by impact factor of the journal)

In Utero CRISPR-Mediated Therapeutic Editing of Metabolic Genes

Scientists assessed the viral vector-mediated delivery of CRISPR-Cas9 or base editor 3 in utero, seeking therapeutic modification of Pcsk9 or Hpd in wild-type mice or the murine model of hereditary tyrosinemia type 1, respectively. [Nat Med] Abstract | Press Release

Huntingtin Suppression Restores Cognitive Function in a Mouse Model of Huntington’s Disease

Investigators tested allele-specific antisense oligonucleotides in a mouse model of Huntington’s disease. Both early and late treatment reduced cognitive and behavioral impairments in mice. [Sci Transl Med] Abstract | Editorial

Antigen-Selective Modulation of AAV Immunogenicity with Tolerogenic Rapamycin Nanoparticles Enables Successful Vector Re-Administration

The authors demonstrated that synthetic vaccine particles encapsulating rapamycin, co-administered with adeno-associated virus (AAV) vectors, prevented the induction of anti-capsid humoral and cell-mediated responses. [Nat Commun] Full Article | Press Release

Intratumoral Immunotherapy with XCL1 and sFlt3L Encoded in Recombinant Semliki Forest Virus-Derived Vectors Fosters Dendritic Cell-Mediated T Cell Cross-Priming

In syngeneic mice, intratumoral injection of Semliki Forest virus-XCL1-sFlt3L delayed progression of MC38- and B16-derived tumors. Therapeutic activity was observed and exerted additive effects in combination with anti-PD-1, anti-CD137, or CTLA-4 immunostimulatory monoclonal antibodies. [Cancer Res] Abstract

Dose-Dependent Effects of FKRP Gene Replacement Therapy on Functional Rescue and Longevity in Dystrophic Mice

Investigators examined the longitudinal effects of a dose-escalation study to evaluate the safety and therapeutic potential of fukutin-related protein (FKRP) gene replacement therapy in a p.P448L mouse model of muscular dystrophy-dystroglycanopathy. [Mol Ther Methods Clin Dev] Abstract | Full Article

Human Neural Stem Cell Transplantation Improves Cognition in a Murine Model of Alzheimer’s Disease

Researchers completed a preclinical proof-of-concept study in the APP/PS1 murine model of Alzheimer’s disease. Human neural stem cell transplantation targeted to the fimbria fornix significantly improved cognition in two hippocampal-dependent memory tasks at 4 and 16 weeks post-transplantation. [Sci Rep] Full Article

Kidney-Derived c-kit⁺ Progenitor/Stem Cells Contribute to Podocyte Recovery in a Model of Acute Proteinuria

Scientists investigated whether c-kit⁺ progenitor/stem cells contributed to podocyte repair in a rat model of acute proteinuria induced by puromycin aminonucleoside, the experimental prototype of human minimal change disease and early stages of focal and segmental glomerulosclerosis. [Sci Rep] Full Article

Characterization of Abscopal Effects of Intratumoral Electroporation-Mediated IL-12 Gene Therapy

Investigators analyzed the immunological changes induced with the new intratumoral electroporation-mediated IL-12 gene therapy (IT-pIL12/EP) platform in both electroporated and distant, non-electroporated lesions. IT-pIL12/EP-treated tumors demonstrated rapid induction of IL-12-regulated pathways, as well as other cytokines and chemokines pathways, and upregulation of antigen presentation machinery. [Gene Ther] Full Article

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Stem Cells under the Influence of Alcohol: Effects of Ethanol Consumption on Stem/Progenitor Cells

The authors review the main studies describing the effects of alcohol on different types of progenitor/stem cells including neuronal, hepatic, intestinal and adventitial progenitor cells, bone-marrow-derived stromal cell, dental pulp, embryonic and hematopoietic stem cells, and tumor-initiating cells. [Cell Mol Life Sci] Full Article

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Alnylam Reports New Platform Innovations for RNAi Therapeutics

Alnylam Pharmaceuticals, Inc. announced further progress on the company’s platform efforts in extrahepatic delivery of novel siRNA conjugates, including central nervous system and ocular delivery in rat and non-human primates. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the Oligonucleotide Therapeutics Society (OTS) 2018 Annual Meeting, Seattle] Press Release

Alnylam Reports Updated Positive Results from Phase I/II Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals, Inc. announced updated positive results from its Phase I/II clinical study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase for the treatment of primary hyperoxaluria type 1. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the 2018 European Society for Paediatric Nephrology (ESPN) Annual Meeting, Antalya] Press Release

Themis Bioscience Announces Exclusive Licensing Agreement with Max Planck Innovation to Develop and Commercialize Oncolytic Virotherapies

Themis announced that it has entered into a license agreement with Max-Planck-Innovation GmbH granting it exclusive worldwide license to develop, manufacture and commercialize therapies based on an oncolytic measles virus platform that was jointly developed by the Eberhard-Karls-University Tübingen and the Max Planck Institute for Biochemistry. [Themis Bioscience GmbH] Press Release

Be The Match BioTherapies^® and Cryoport Announce Strategic Partnership to Deliver Integrated End-to-End Supply Chain Services to the Cell and Gene Therapy Industry

Be The Match BioTherapies and Cryoport, Inc. announced a strategic partnership to deliver end-to-end supply chain services to the cell and gene therapy industry. [Be The Match BioTherapies] Press Release

Platelet Biogenesis and Scaled Biolabs to Collaborate to Optimize Platelet Production Platform

Platelet BioGenesis (PBG) and Scaled Biolabs announced the two firms are collaborating to optimize PBG’s unique production process for making human platelets, or PLTs+™. [Platelet BioGenesis (Business Wire Ltd.)] Press Release

Celyad Announces Exclusive Agreement for Horizon Discovery’s shRNA Platform to Develop Next-Generation Allogeneic CAR-T Therapies

Celyad announced an exclusive agreement with Horizon Discovery Group plc for the use of its shRNA technology to generate Celyad’s second non-gene-edited allogeneic platform. [Celyad] Press Release

Sarepta and Lysogene Announce Exclusive License Agreement for LYS-SAF302, a Late-Stage Gene Therapy for the Treatment of MPS IIIA, and Grant of Option Rights to an Additional CNS Gene Therapy Candidate

Sarepta Therapeutics, Inc. announced it has signed a license agreement with Lysogene for the development of a gene therapy, LYS-SAF302, to treat mucopolysaccharidosis type IIIA (MPS IIIA). [Sarepta Therapeutics, Inc.] Press Release

Qatar Biomedical Research Institute Forms New Partnership with Harvard Stem Cell Institute

The Qatar Biomedical Research Institute has signed a collaborative research and training agreement with the Harvard Stem Cell Institute. [Hamad Bin Khalifa University] Press Release

Athenex and Xiangxue Life Sciences Announce Preliminary Results of Patients Receiving T-Cell Receptor Affinity Enhancing Specific T-Cell Therapy (TAEST) Showed Encouraging Positive Clinical Signals

Athenex, Inc. announced that preliminary results of pilot studies in China in which patients received T-cell receptor affinity enhancing specific T-cell therapy showed encouraging positive clinical signals in terms of efficacy and safety. [Athenex, Inc.] Press Release

Orchard Therapeutics Receives EMA PRIME Designation for OTL-300, an Investigational Lentiviral Gene Therapy for the Treatment of Transfusion-Dependent Beta-Thalassemia

Orchard Therapeutics announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-300, an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta-thalassemia, the most severe form of beta-thalassemia. [Orchard Therapeutics] Press Release

UIC Receives Department of Defense Grant to Lead Clinical Trial of Stem Cell Therapy for Eye Injuries

Researchers at the University of Illinois at Chicago (UIC) have received a four-year, $5.25 million grant from the U.S. Department of Defense to lead a multi-site clinical trial to test the efficacy of a stem cell-based treatment for eye injuries. [University of Illinois at Chicago] Press Release

HSS Researchers Launch Clinical Trial to Determine if Popular Stem Cell Therapy Can Protect Damaged Knees from Osteoarthritis

Led by Andreas H. Gomoll, MD the study is looking at the effects on knee function of ReNu, an allograft product composed of human amniotic membrane and cells from the amniotic fluid. Organogenesis, who makes the product, is studying ReNu in a number of orthopedic applications. [Hospital for Special Surgery] Press Release

bluebird bio Announces European Medicines Agency’s Acceptance of Marketing Authorization Application for LentiGlobin™ Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

bluebird bio, Inc. announced that the European Medicines Agency accepted the company’s marketing authorization application for its investigational LentiGlobin™ gene therapy for the treatment of adolescents and adults with transfusion-dependent β-thalassemia and a non-β⁰/β⁰ genotype. [bluebird bio, Inc.] Press Release

Japan Set to Allow Gene Editing in Human Embryos

Japan has issued draft guidelines that allow the use of gene-editing tools in human embryos. Although the country regulates the use of human embryos for research, there have been no specific guidelines on using tools such as CRISPR-Cas9 to make precise modifications in their DNA until now. [Nature News] Editorial

The UK Pharmaceutical Industry Braces for Brexit

As Britain’s departure date from the European Union approaches, drugmakers are preparing for potential changes to regulatory procedures and border controls. [The Scientist] Editorial

Argentina’s Scientists Struggle amid Slipping Peso and Rising Inflation

Researchers have struggled under austerity measures adopted by the government in 2014 and intensified in June by a financing agreement that Argentina signed with the International Monetary Fund. [Nature News] Editorial

NEW BIT’s 12^th World Congress of Regenerative Medicine & Stem Cell
December 7-9, 2018
Xi’an, China

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NEW PhD and Posdoctoral Research Positions – Innate Immunity (Universitatsklinikum Tubingen)

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Postdoctoral Fellow – Stem Cell Biology/Stroke, and Alzheimer’s Disease Research (Tulane University)

Open Rank Professor – Biopharmaceutical Discovery (University of Delaware)

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Research Associate – Immunotherapy (Baylor College of Medicine)

Postdoctoral Position – Cancer, Immunology, and/or Single-Cell Epigenomics (CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences)

Director – Clinical Development (California Institute for Biomedical Research)

Faculty Position – Adoptive T Cell Therapy (Dana-Farber Cancer Institute)

Professorship – Somatic Gene Therapy (University of Zurich)

Postdoctoral Staff Fellow – Biologist/Biomedical Engineer (Food and Drug Administration)

Associate Professor – Cancer Research (University of Maryland)

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