Cell Therapy News Volume 23.16 | May 9 2022

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    2022-05-09 | CTN 23.16


    Cell Therapy News by STEMCELL Technologies
    Vol. 23.16 – 9 May, 2022
    TOP STORY

    Enhanced Safety and Efficacy of Protease-Regulated CAR-T Cell Receptors

    Investigators presented SNIP CARs, a protease-based platform for regulating CAR activity using an FDA-approved small molecule.
    [Cell]

    Full ArticleGraphical Abstract
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    PUBLICATIONSRanked by the impact factor of the journal

    Claudin18.2-Specific CAR T Cells in Gastrointestinal Cancers: Phase I Trial Interim Results

    Researchers reported the interim analysis results of an ongoing, open-label, single-arm, Phase I clinical trial of CLDN18.2-targeted CAR T cells in patients with previously treated, CLDN18.2-positive digestive system cancers.
    [Nature Medicine]

    Full Article

    Anti-CCR9 Chimeric Antigen Receptor T Cells for T Cell Acute Lymphoblastic Leukemia

    Scientists demonstrated that the chemokine receptor CCR9 is expressed in over 70% of cases of T cell acute lymphoblastic leukemia, including over 85% or relapsed/refractory disease, and only on a small fraction of normal T cells.
    [Blood]

    Abstract

    Risk Assessment with Low Pass Whole Genome Sequencing of Cell Free DNA before CD19 CAR T Cells for Large B Cell Lymphoma

    The authors evaluated low pass whole genome sequencing of cell free DNA prior to CART19 as a new approach for risk stratification.
    [Blood]

    Abstract

    Cell Surface-Tethered IL-12 Repolarizes the Tumor Immune Microenvironment to Enhance the Efficacy of Adoptive T Cell Therapy

    Researchers described an approach to safely harness cytokine biology for adoptive cell therapy through uniform and dose-controlled tethering onto the surface of the adoptively transferred cells.
    [Science Advances]

    Full Article

    CAR T Cell Manufacturing from Naive/Stem Memory T-Lymphocytes Enhances Antitumor Responses while Curtailing Cytokine Release Syndrome

    Scientists investigated the efficacy and safety profiles of CAR T cell products generated from pre-selected naive/stem memory T cells, as compared to unselected T cells.
    [Journal of Clinical Investigation]

    AbstractGraphical Abstract

    Highly Active CAR T Cells That Bind to a Juxtamembrane Region of Mesothelin and Are Not Blocked by Shed Mesothelin

    The authors identified the major protease sites in mesothelin (MSLN) and prepared a monoclonal antibody 15B6, that bound next to the cell membrane at the protease-sensitive region and inhibits MSLN shedding.
    [Proceedings of the National Academy of Sciences of the United States of America]

    Full Article

    Differential CXCR4 Expression on Hematopoietic Progenitor Cells versus Stem Cells Directs Homing and Engraftment

    Investigators found that intra-bone marrow hematopoietic stem and progenitor cell transplantation improved engraftment of hematopoietic progenitor cells but not of long-term repopulating hematopoietic stem cells.
    [JCI Insight]

    Full Article

    Hematopoietic Stem Cell Gene Therapy Ameliorates CNS Involvement in Murine Model of GM1-Gangliosidosis

    Researchers evaluated the therapeutic efficacy of ex vivo gene therapy targeting hematopoietic stem cells using a lentiviral vector to increase enzyme activity, reduce substrate accumulation, and improve astrocytosis and motor function.
    [Molecular Therapy-Methods & Clinical Development]

    AbstractFull ArticleGraphical Abstract
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    REVIEWS

    New Editing Tools for Gene Therapy in Inherited Retinal Dystrophies

    Scientists review gene-editing tools based on CRISPR-Cas9 technology that have been used in vivo and the recent first-in-human application of CRISPR-Cas9 in an inherited retinal dystrophy.
    [CRISPR Journal]

    Full Article

    Stem Cell Therapy for Vocal Fold Regeneration after Scarring: A Review of Experimental Approaches

    The authors cover the methodological aspects of preclinical studies such as experimental models, characterization of cell products, assessment of the study outcome using molecular, morphological, and immunohistochemical analyses, as well as measuring the tissue’s physical properties.
    [Stem Cell Research & Therapy]

    Press ReleaseGraphical Abstract
    INDUSTRY AND POLICY NEWS

    Achilles Therapeutics Doses First Patient with Higher-Dose cNeT in Phase I/IIa CHIRON Trial in Advanced NSCLC and Initiates Enrollment in Cohort B of the THETIS Trial (cNeT + PD-1 Checkpoint Inhibitor) in Metastatic Malignant Melanoma.

    Achilles Therapeutics plc announced that the first patient has been dosed with personalized clonal neoantigen-reactive T cells, or cNeT, manufactured with the company’s higher-dose VELOS™ Process II’ in the ongoing Phase I/IIa CHIRON clinical trial for the treatment of advanced non-small cell lung cancer
    [Achilles Therapeutics plc]

    Press Release

    Umoja Biopharma Announces Activation of First ENLIGHTen Phase I Trial Site in Study of UB-TT170, a TumorTag that Targets Folate Receptors to Mark Tumors for Clearance by CAR T Cells

    Umoja Biopharma, Inc. announced the Seattle Children’s activation of the Phase I ENLIGHTen clinical trial in patients with osteosarcoma to assess the safety and tolerability of autologous “universal” CAR T cells when administered with UB-TT170, the company’s proprietary small molecule fluorescein tag.
    [Umoja Biopharma, Inc.]

    Press Release
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    European Molecular Biology Laboratory – Barcelona, Spain

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