Cell Therapy News Volume 24.33 | Oct 16 2023

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    2023-10-17 | CTN 24.33


    Cell Therapy News by STEMCELL Technologies
    Vol. 24.33 – 16 October, 2023
    TOP STORY

    Probiotic-Guided CAR-T Cells for Solid Tumor Targeting

    The authors developed a platform of probiotic-guided CAR-T cells, in which tumor-colonizing probiotics released synthetic targets that labeled tumor tissue for CAR-mediated lysis in situ.
    [Science]

    AbstractPress Release
    PUBLICATIONSRanked by the impact factor of the journal

    Control of the Antitumor Activity and Specificity of CAR T Cells via Organic Adapters Covalently Tethering the CAR to Tumor Cells

    Investigators showed that the tumor-targeting specificity and activity of T cells with a CAR consisting of an antibody with a lysine residue that catalytically formed a reversible covalent bond with a 1,3-diketone hapten can be regulated by the concentration of a small-molecule adapter.
    [Nature Biomedical Engineering]

    Abstract

    Human Pallial MGE-Type GABAergic Interneuron Cell Therapy for Chronic Focal Epilepsy

    Scientists reported the development and characterization of a cell therapy alternative for drug-resistant mesial temporal lobe epilepsy, which was derived from a human embryonic stem cell line and comprises cryopreserved, post-mitotic, medial ganglionic eminence (MGE) pallial-type GABAergic interneurons.
    [Cell Stem Cell]

    Full ArticleGraphical Abstract

    Multiple Infusions of Ex Vivo-Expanded Regulatory T Cells Promote CD163+ Myeloid Cells and Kidney Allograft Survival in Non-lymphodepleted Non-Human Primates

    Researchers examined the influence of autologous ex vivo-expanded polyclonal Tregs on kidney graft survival in a clinically-relevant non-human primate model. Peripheral blood Tregs were isolated and expanded using artificial antigen presenting cells.
    [Kidney International]

    Abstract

    Impact of Targeting Moiety Type and Protein Corona Formation on the Uptake of Fn14-Targeted Nanoparticles by Cancer Cells

    Scientists investigated a full-length Fn14-specific monoclonal antibody, ITEM4, or an ITEM4-Fab fragment as a targeting moiety to guide the development of a clinical formulation.
    [ACS Nano]

    AbstractGraphical Abstract

    Fertility-Preserving Myeloablative Conditioning Using Single-Dose CD117 Antibody-Drug Conjugate in a Rhesus Gene Therapy Model

    Investigators tested the ability of an antibody-drug conjugate targeting CD117 to enable engraftment in a non-human primate lentiviral gene therapy model of hemoglobinopathies.
    [Nature Communications]

    Full ArticlePress Release

    An “Off-the-Shelf” CD2 Universal CAR-T Therapy for T Cell Malignancies

    To broaden the use of CAR-T cells in pan T cell malignancies, researchers developed an allogeneic “universal” CD2-targeting CAR-T cell, in which the CD2 antigen is deleted to prevent fratricide, and the T cell receptor is removed to prevent graft-versus-host-disease.
    [Leukemia]

    Full Article

    A Deregulated m6A Writer Complex Axis Driven by BRD4 Confers an Epitranscriptomic Vulnerability in Combined DNA Repair–Targeted Therapy

    BRD4 directly stimulated transcripts expression of seven methyltransferase complex subunits, allowing the maintenance of the nuclear writer complex integrity.
    [Proceedings Of The National Academy Of Sciences Of The United States Of America]

    Abstract

    Gene Therapy Restores the Transcriptional Program of Hematopoietic Stem Cells in Fanconi Anemia

    Investigators demonstrated that gene therapy reverted the transcriptional signature of Fanconi anemia hematopoietic stem and progenitor cells (HSPCs), which then resembles the transcriptional program of healthy donor HSPCs.
    [Haematologica]

    Abstract

    Sustained Long-Term Disease Correction in a Murine Model of MPSII Following Stem Cell Gene Therapy

    The authors observed sustained IDS enzyme activity in organs of long-term IDS. ApoEII-treated MPSII mice, with continued clearance of storage material in the brain and peripheral organs, maintained correction of astrogliosis, microgliosis and correction of altered cytokines and chemokines.
    [Molecular Therapy-Methods & Clinical Development]

    AbstractFull ArticleGraphical Abstract
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    REVIEWS

    Synthetic Manipulation of the Cancer-Immunity Cycle: CAR-T Cell Therapy

    Investigators reviewed CAR T cell design and the mechanisms that drive sustained CAR T cell effector activity and anti-tumor function. They discuss engineering approaches aimed at improving anti-tumor function through a variety of mechanistic interventions for both hematologic and solid tumors.
    [Immunity]

    Full Article

    INSPIRED Symposium Part 4A: Access to CAR T cell Therapy in Unique Populations with B Cell Acute Lymphoblastic Leukemia

    It is important to prospectively study patient-reported outcomes given the differential toxicity expected between CD19 CAR T cell therapy and the historic standard of care, hematopoietic cell transplant.
    [Transplantation And Cellular Therapy]

    AbstractGraphical Abstract

    Involving Stemness Factors to Improve CAR T-Cell-Based Cancer Immunotherapy

    In-depth identification of tumor microenvironment and development of more potent CAR platform targeting CSCs may overcome the raised challenges, as presented in this review.
    [Medical Oncology]

    Abstract
    INDUSTRY AND POLICY NEWS

    Monkey Survives for Two Years after Gene-Edited Pig-Kidney Transplant

    A kidney transplanted from a genetically engineered miniature pig kept a monkey alive for more than two years — one of the longest survival times for an interspecies organ transplant.
    [Nature News]

    Editorial

    Gene Therapies for Rare Diseases Are Under Threat. Scientists Hope to Save Them

    Strimvelis is a potent treatment for a devastating genetic disorder of the immune system and one of the first gene therapies to be approved in Europe. But in 2022, the company that sold it announced that it could no longer afford to do so: Strimvelis was highly effective at treating disease, but not at turning a profit.
    [Nature News]

    Full Article
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