Cell Therapy News Volume 21.33 | Dec 7 2020

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    CTN 21.33 | Dec 7 2020


    Cell Therapy News by STEMCELL Technologies
    Vol. 21.33 – 7 December, 2020
    TOP STORY

    CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

    Scientists performed electroporation of CD34+ hematopoietic stem and progenitor cells obtained from healthy donors, with CRISPR-Cas9 targeting the BCL11A erythroid-specific enhancer.
    [New England Journal of Medicine]

    Abstract

    Dr. Jackie Damen discusses why the CFU assay should be a vital part of your cell therapy workflow in this on-demand webinar.
    PUBLICATIONSRanked by the impact factor of the journal

    CRISPR Screen Identifies Genes That Sensitize AML Cells to Double Negative T Cell Therapy

    Investigators used a targeted CRISPR/Cas9 screen to identify genes that confer susceptibility of acute myeloid leukemia cells (AML) to double negative T cell therapy.
    [Blood]

    Abstract

    Clinical Effects of Administering Leukemia-Specific Donor T Cells to Patients with AML/MDS Post-Allogeneic Transplant.

    Scientists selectively activated and expanded stem-cell donor-derived T cells that were reactive to multiple antigens expressed by acute myeloid leukemia/myelodysplastic syndrome cells
    [Blood]

    Abstract

    ATG5-Dependent
    Autophagy Uncouples T Cell Proliferative and Effector Functions and Separates Graft-versus-Host Disease from Graft-versus-Leukemia

    To assess the relevance of autophagy to T cell allo-immunity, the authors generated T cell specific ATG5 knock-out mice. Deficiency of ATG5 dependent autophagy reduced T cell proliferation, increased apoptosis following in vitro and in vivo allo-stimulation.
    [Cancer Research]

    Abstract

    Master
    Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens

    Using multiplexed gene regulation with orthogonal CRISPR systems, researchers demonstrated improved neuronal differentiation with concurrent activation and repression of target genes, underscoring the power of CRISPR-based gene regulation for programming complex cellular phenotypes.
    [Cell Reports]

    Full ArticlePress Release
    Graphical Abstract

    Autologous Stem Cell Transplantation for Multiple Myeloma Patients Aged ≥75 Treated With Novel Agents

    Investigators report on outcomes of outpatient autologous stem cell transplantation in a cohort of patients with multiple myeloma aged ≥75 years.
    [Bone Marrow Transplantation]

    Abstract

    Immune Imitation of Tumor Progression after Anti-CD19 Chimeric Antigen Receptor T Cells Treatment in Aggressive B-Cell Lymphoma

    Scientists present three patients with aggressive non-Hodgkin’s B-cell lymphoma who received anti-CD19 chimeric antigen receptor T cells therapy after failure of several lines of chemotherapy that developed pseudo-progression.
    [Bone Marrow Transplantation]

    Abstract

    Selective Cognitive Dysfunction and Physical Disability Improvement after Autologous Hematopoietic Stem Cell Transplantation in Highly Active Multiple Sclerosis

    Researchers explored the potential factors influencing disability regression after autologous hematopoietic stem cell transplantation and estimated the safety of low-dose immunosuppressive therapy in highly active Multiple Sclerosis patients.
    [Scientific Reports]

    Abstract

    Request your free copy of the 'Production of Chimeric Antigen Receptor T Cells' Wallchart
    REVIEWS

    Outlook
    for New CAR-Based Therapies with a Focus on CAR NK Cells: What Lies Beyond CAR-Engineered T Cells in the Race against Cancer

    Investigators review the advantages, challenges, and preclinical and clinical research advances in CAR NK cell engineering for cancer immunotherapy.
    [Cancer Discovery]

    Abstract

    The Clinical Role of the Gut Microbiome and Fecal Microbiota Transplantation in Allogeneic Stem Cell Transplantation

    The authors define the role of administrating fecal microbiota transplantation in the setting of allogeneic hematopoietic stem cell transplantation.
    [Haematologica]

    Abstract

    Regulatory T Cell Therapy following Liver Transplantation

    Scientists summarize the discovery and development of Tregs, and discuss the outstanding challenges of Treg cell therapy and its future prospects for routine use in liver transplantation.
    [Liver Transplantation]

    Abstract

    INDUSTRY AND POLICY NEWS

    Turnstone Biologics Announces First Patients Enrolled in Phase I/II Clinical Trial Evaluating RIVAL-01 for the Treatment of Solid Tumors

    Turnstone Biologics Corp., have enrolled the first patients in a Phase I/IIa clinical trial of its RIVAL-01/TAK-605 candidate in patients with solid tumors, conducted in collaboration with Takeda Pharmaceutical Company Limited. RIVAL-01 consists of Turnstone’s proprietary oncolytic vaccinia virus backbone encoding transgenes for Flt3 ligand, anti-CTLA-4 antibody and IL-12 cytokine, purposefully designed to work together to drive immune activity and re-program the microenvironment to be best suited for tumor eradication.
    [Turnstone Biologics Corp. (Businesswire, Inc.)]

    Press Release

    Shape Therapeutics Unveils AAVid™ Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery Challenge in Gene Therapy

    Shape Therapeutics, Inc. announced the unveiling of the AAVid™ capsid discovery platform and results from its first AAV5 variant library in a non-human primate selection campaign.
    [Shape Therapeutics, Inc. (BusinessWire, Inc.)]

    Press Release

    Government of Canada and JDRF Canada Announce New Research Funding to Accelerate Stem Cell-Based Therapies for Type 1 Diabetes

    To mark the end of Diabetes Awareness Month, Sonia Sidhu, Member of Parliament for Brampton South, on behalf of Patty Hajdu, Minister of Health, announced an investment of $6 million through the CIHR-JDRF Partnership to Defeat Diabetes for two Canadian research teams to accelerate the development of stem cell-based therapies for the treatment of type 1 diabetes.
    [Juvenile Diabetes Research Foundation]

    Press Release


    REGENXBIO Announces Dosing of First Patient in Phase I/II Trial of RGX-111 for the Treatment of Mucopolysaccharidosis Type I

    REGENXBIO, Inc. announced that the first patient has been dosed in the Phase I/II trial of RGX-111 for the treatment of Mucopolysaccharidosis Type I. RGX-111 is an investigational one-time gene therapy designed to deliver the α-l-iduronidase gene directly to the central nervous system using the NAV AAV9 vector.
    [REGENXBIO, Inc.]

    Press Release

    FEATURED EVENT

    Gordon Research Conferences: Stem Cells and Cancer

    May 16 – May 21, 2021
    Lucca, Italy


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    JOB OPPORTUNITIES


    Postdoctoral Research Scientist – Tissue Engineering of the Lung

    Columbia University – New York, New York, United States

    Technical Director – Cellular Therapies

    H. Lee Moffitt Cancer Center & Research Institute – Tampa, Florida, United States

    Research Manager – Gene Therapy

    Axovia Therapeutics – London, England, United Kingdom

    PhD Student – Adoptive T Cell Therapy of Cancer

    LMU University Hospital Munich – Munich, Germany

    Process
    Development Lead – Gene Transfer

    EnaraBio – Oxford, England, United Kingdom

    > See All Jobs

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