Cell Therapy News Volume 21.12 | Apr 6 2020

TOP STORY

Selective Organ Targeting (SORT) Nanoparticles for Tissue-Specific mRNA Delivery and CRISPR-Cas Gene Editing

Researchers report a strategy termed selective organ targeting (SORT) wherein multiple classes of lipid nanoparticles are systematically engineered to exclusively edit extrahepatic tissues via addition of a supplemental SORT molecule. [Nat Nanotechnol] Abstract

(Ranked by impact factor of the journal)

Human iPSC-Derived Mature Microglia Retain Their Identity and Functionally Integrate in the Chimeric Mouse Brain

Investigators developed a human pluripotent stem cell (hPSC)-based microglial chimeric mouse brain model by transplanting hPSC-derived primitive macrophage progenitors into neonatal mouse brains. [Nat Commun] Full Article

Designer, Injectable Gels to Prevent Transplanted Schwann Cell Loss during Spinal Cord Injury Therapy

In a cervical contusion model, hydrogel matrix resulted in a greater than 700% improvement in successful Schwann cell transplantation. The combination therapy of cells and gel significantly improved the spatial distribution of transplanted cells within the endogenous tissue. [Sci Adv] Full Article

In Vivo Priming of Human Mesenchymal Stem Cells with Hepatocyte Growth Factor-Engineered Mesenchymal Stem Cells Promotes Therapeutic Potential for Cardiac Repair

Primed bone marrow-derived mesenchymal stem cells (MSCs) through hepatocyte growth factor-expressing MSCs exhibited improved vasculogenic potential and cell viability, which ultimately enhanced vascular regeneration and restored cardiac function to myocardial infarction hearts. [Sci Adv] Full Article

Bispecific CAR-T Cells Targeting Both CD19 and CD22 for Therapy of Adults with Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia

The authors demonstrated that bispecific CD19/CD22 CAR T cells could trigger robust cytolytic activity against target cells. MRD-negative complete remission was achieved in six out of six enrolled patients. [J Hematol Oncol] Full Article

Genome Editing of Donor-Derived T-Cells to Generate Allogenic Chimeric Antigen Receptor-Modified T Cells: Optimizing Αβ T Cell-Depleted Haploidentical Hematopoietic Stem Cell Transplantation

In vivo, the gene modified T cells eliminated leukemia without causing xenogeneic graft-versus-host disease in a xenograft model. Gene editing was highly specific with no evidence of off-target effects. [Haematologica] Abstract

TWIST1-Reprogrammed Endothelial Cell Transplantation Potentiates Neovascularization-Mediated Diabetic Wound Tissue Regeneration

Transplantation of stable TWIST1-reprogrammed endothelial cells into full-thickness type 1 and 2 diabetic-splinted wound healing murine models enhanced the microcirculatory blood flow and accelerated wound tissue regeneration. [Diabetes] Abstract

Reversing Acute Kidney Injury Using Pulsed Focused Ultrasound and Mesenchymal Stromal Cell Therapy: A Role for Heat Shock Protein-Mediated PI3K/AKT Signaling

Researchers studied the effect of bone marrow-derived mesenchymal stromal cells (BM-MSCs), in conjunction with focused ultrasound, in a mouse model of cisplatin-induced acute kidney injury. BM-MSCs improved kidney function, reduced histological markers of kidney injury, decreased inflammation and apoptosis, and promoted cellular proliferation. [Mol Ther Methods Clin Dev] Abstract | Full Article | Graphical Abstract

Activated Mesenchymal Stem Cells Induce Recovery following Stroke via Regulation of Inflammation and Oligodendrogenesis

Interferon-γ-activated mesenchymal stem cell (aMSCγ) treatment resulted in significant functional recovery assessed by the modified neurological severity score and open-field analysis compared with vehicle-treated animals. aMSCγ‐treated animals showed significant reductions in infarct size and inhibition of microglial activation. [J Am Heart Assoc] Abstract

Engineered Cartilage Utilizing Fetal Cartilage-Derived Progenitor Cells for Cartilage Repair

In a nonhuman primate investigation, cartilage repair scores were significantly better in the gel group compared to defects alone after 24 weeks. Cell distribution analysis at 24 weeks showed that human cells remained within the transplanted defects only. [Sci Rep] Full Article

SIRT3 Transfection of Aged Human Bone Marrow-Derived Mesenchymal Stem Cells Improves Cell Therapy-Mediated Myocardial Repair

Following myocardial infarction and intramyocardial injections of older donors human mesenchymal stem cells (O-hMSCs) in rats in vivo, the survival rate of O-hMSCs increased by sirtuin 3 (SIRT3) transfection. The cardiac function of rats was improved after SIRT3-overexpressed O-hMSC transplantation. [Rejuvenation Res] Abstract

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Next-Generation Stem Cells — Ushering in a New Era of Cell-Based Therapies

Gene therapy and gene editing technologies are being used to create stem cell derivatives with improved functionality, specificity and responsiveness compared with their natural counterparts. Scientists review these engineering approaches and areas in which they will help broaden the utility and clinical applicability of stem cells. [Nat Rev Drug Discov] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Bristol Myers Squibb and bluebird bio Announce Submission of Biologics License Application (BLA) for Anti-BCMA CAR T Cell Therapy Idecabtagene Vicleucel (Ide-cel, bb2121) to FDA

Bristol Myers Squibb and bluebird bio, Inc. announced the submission of their BLA to the FDAfor idecabtagene vicleucel, the companies’ lead investigational B-cell maturation antigen-directed CAR-T cell immunotherapy, for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. [Bristol Myers Squibb Company] Press Release

Citius Signs Exclusive Option with Novellus to License Novel Stem-Cell Therapy for Acute Respiratory Distress Syndrome (ARDS) Associated with COVID-19

Citius Pharmaceuticals, Inc. signed an exclusive six-month option agreement to in-license a stem-cell therapy for ARDS from a subsidiary of Novellus, Inc. Novellus’s patented process uses its exclusive non-immunogenic synthetic messenger ribonucleic acid molecules to create induced pluripotent stem cells that, in turn, generate mesenchymal stem cells with superior immunomodulatory properties. [Citius Pharmaceuticals, Inc.] Press Release

Celularity Announces FDA Clearance of IND Application for CYNK-001 in Coronavirus, First in Cellular Therapy

Celularity Inc. announced the FDA has cleared the company’s Investigational New Drug (IND) application for the use of its proprietary CYNK-001 in adults with COVID-19. Celularity will commence a Phase I/II clinical study including up to 86 patients with COVID-19. [Celularity Inc.] Press Release

Gracell Announces China MNPA Acceptance of Investigational New Drug Application for GC007g Cell Therapy for CD19 Positive Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia

Gracell Biotechnologies Co., Ltd. announced that China National Medical Products Administration (MNPA) has accepted Gracell’s Investigational New Drug application for GC007g, a donor-derived anti-CD19 CAR-T cell therapy. [Gracell Biotechnologies Co., Ltd.] Press Release

Safely Conducting Essential Research in the Face of COVID-19

Some scientists can’t simply stop going to their laboratories — especially not those who are overseeing clinical trials that could offer life-saving vaccines and therapies, particularly against the new coronavirus. And some research activities must continue even in the face of a shutdown. [Nature News] Editorial

NIH’s Process for Removing Reviewers Remains a Mystery, Watchdog Finds

A new report by the Office of Inspector General of National Institute of Health’s (NIH’s) parent body, the Department of Health and Human Services, documents how NIH has begun to remove reviews who breached NIH standards on research integrity. [ScienceInsider] Editorial

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NEW Associate/Senior Editor – Nature Biotechnology (Nature Research)

NEW Senior Research Scientist – Orthopedic Surgery/Regenerative Medicine (NYU Grossman School of Medicine)

NEW Team Leaders – Stem Cell Biology (RIKEN Center for Biosystems Dynamics Research)

NEW Postdoctoral Research Associate – Induced Pluripotent Stem Cell Therapies (The University of Arizona College of Medicine)

Scientist/Senior Scientist – Cell Therapy (Zelluna Immunotherapy AS)

Professorship – Biological Therapies of the Inner Ear (Hannover Medical School)

Group leaders – Cancer Biology (Institute of Pharmacology and Structural Biology)

Scientist – Cancer Immunotherapy (Lund University)

Postdoctoral Position – Cancer Immunotherapy (The University of Texas MD Anderson Cancer Center)

Sr Associate Scientist – Autoimmunity and Tumor Immunotherapy (Amgen)

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