Cell Therapy News Volume 20.32 | Sep 23 2019

TOP STORY

In Vivo CRISPR Screening in CD8 T Cells with AAV-Sleeping Beauty Hybrid Vectors Identifies Membrane Targets for Improving Immunotherapy for Glioblastoma

Scientists performed in vivo adeno-associated virus (AAV)-Sleeping Beauty-CRISPR screens for membrane protein targets in CD8⁺ T cells in mouse models of glioblastoma (GBM). They validated screen hits by demonstrating that adoptive transfer of CD8⁺ T cells with Pdia3, Mgat5, Emp1 or Lag3 gene editing enhanced the survival of GBM-bearing mice in both syngeneic and T-cell receptor transgenic models. [Nat Biotechnol] Abstract

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NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia

Non-homologous end-joining (NHEJ)-mediated editing efficacy was initially verified in Fanconi anemia lymphoblastic cell lines and then in primary FA patient-derived CD34⁺ cells, which showed marked proliferative advantage and phenotypic correction both in vitro and after transplantation. [Cell Stem Cell] Abstract | Graphical Abstract

Prevalence and Relevance of Pre-Existing Anti-AAV Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome

Researchers assessed the prevalence of anti-adeno-associated virus (AAV) 8 neutralizing antibodies (NAb) in subjects affected by Crigler-Najjar syndrome and investigated the impact of low NAb titers on liver gene transfer efficacy in an in vivo passive immunization model. [Hum Gene Ther] Abstract | Full Article

β-Adrenergic Blocker, Carvedilol, Abolishes Ameliorating Actions of Adipose-Derived Stem Cell Sheets on Cardiac Dysfunction and Remodeling after Myocardial Infarction

In in vivo studies using a rat myocardial infarction (MI) model, transplanted adipose-derived stem cell sheets improved contractile functions of MI hearts; they also facilitated neovascularization and suppressed fibrosis after MI. [Circ J] Full Article

Cell Therapy: Effect of Locally Injected Mesenchymal Stromal Cells Derived from Bone Marrow or Adipose Tissue on Bone Regeneration of Rat Calvarial Defects

Cell therapy based on local injection of bone marrow-derived mesenchymal stromal cells (BM-MSCs) or adipose tissue-derived MSCs (AT-MSCs) was effective in delivering cells that induced a significant improvement in bone healing. Despite differences observed in molecular cues between BM-MSCs and AT-MSCs, both cells had the ability to induce bone tissue formation at comparable amounts and properties. [Sci Rep] Full Article

Pioglitazone Treatment Prior to Transplantation Improves the Efficacy of Human Mesenchymal Stem Cells after Traumatic Brain Injury in Rats

Reducing proinflammatory cytokine expression in the cerebral tissues after traumatic brain injury by pioglitazone administration and prior to human mesenchymal stem cell therapy improved the outcome of the therapy in which brain-derived neurotrophic factor could have a role. [Sci Rep] Full Article

Regenerative Potential of the Product “CardioCell” Derived from the Wharton’s Jelly Mesenchymal Stem Cells for Treating Hindlimb Ischemia

Researchers investigated the role of CardioCell in promoting angiogenesis and relieving hindlimb ischemia. [Int J Mol Sci] Full Article

hUC-MSCs Exert a Neuroprotective Effect via Anti-Apoptotic Mechanisms in a Neonatal HIE Rat Model

Scientists investigated how human umbilical cord mesenchymal stem cells (hUC-MSCs) exerted a neuroprotective effect via antiapoptotic mechanisms in a neonatal hypoxic-ischemic encephalopathy (HIE) rat model. [Cell Transplant] Full Article

Angiogenic Effects of Mesenchymal Stem Cells in Combination with Different Scaffold Materials

Investigators studied the angiogenic effects after implanting poly-l-lactide-co-glycolide and beta-tricalcium phosphate scaffolds seeded with isogeneic mesenchymal stem cells in vivo. Fifty-six BALB/c mice were equally divided into seven groups and underwent implantation of the dorsal skinfold chambers. [Microvasc Res] Abstract

The Neuroprotective Effect of Mesenchymal Stem Cells Is Mediated through Inhibition of Apoptosis in Hypoxic Ischemic Injury

In the animal model, transplanted umbilical cord-derived mesenchymal stem cells migrated toward the cerebral lesion site and decreased the lesion extent in hypoxic ischemic encephalopathy. [World J Pediatr] Abstract

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Next-Generation Regulatory T Cell Therapy

As regulatory T cells (T_regs) deploy more than a dozen molecular mechanisms to suppress immune responses, they have potential as multifaceted adaptable smart therapeutics for treating inflammatory disorders. Early-phase clinical trials of T_regs cell therapy have shown feasibility, tolerability and potential efficacy in these disease settings. [Nat Rev Drug Discov] Abstract

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AskBio Acquires Assets from RoverMed BioSciences, Adds Nanotechnology Precision to Delivery of AAV Gene Therapeutics

Asklepios BioPharmaceutical, Inc. (AskBio) has acquired the technology assets of RoverMed BioSciences. RoverMed developed nanotechnology cargo delivery of therapeutics into the nucleus of diseased cells without affecting healthy cells. [Asklepios BioPharmaceutical, Inc.] Press Release

Mallinckrodt Announces Positive Top-Line Results from Pivotal Phase III Clinical Trial of StrataGraft^® Regenerative Tissue in Patients with Deep Partial-Thickness Thermal Burns

Mallinckrodt plc announced positive top-line results from its pivotal Phase III clinical trial of its investigational StrataGraft^® regenerative tissue. [Mallinckrodt plc] Press Release

Abeona Therapeutics Provides Regulatory Update Ahead of Pivotal Phase III Clinical Trial for EB-101 in Recessive Dystrophic Epidermolysis Bullosa

Abeona Therapeutics Inc. announced that it has recently received a clinical hold letter from the FDA clarifying that the FDA will not provide approval for the company to begin its planned Phase III clinical trial for EB-101 until it submits to the FDA additional data points on transport stability of EB-101 to clinical sites. [Abeona Therapeutics Inc.] Press Release

City of Hope Opens Chimeric Antigen Receptor (CAR) T Cell Trial for Advanced Prostate Cancer

City of Hope has opened and is treating the first patients in its CAR T cell trial for prostate cancer that has spread to other parts of the body. The trial uses a CAR T cell – also developed at City of Hope – that targets the prostate stem cell antigen in prostate cancer. [City of Hope] Press Release

Avalon GloboCare Announces Initiation of First-in-Human Clinical Trial of its CAR-T Candidate, AVA-001, for Treatment of B-Cell Malignancies

Avalon GloboCare Corp. announced it has initiated its first-in-human, Phase I clinical trial of CD19 CAR-T candidate, AVA-001. [Avalon GloboCare Corp.] Press Release

SanBio Granted Regenerative Medicine Advanced Therapy Designation from the US FDA for SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury

The SanBio Group announced that the FDA has granted Regenerative Medicine Advanced Therapy Designation for SB623 cell therapy for the treatment of chronic neurological motor deficits secondary to traumatic brain injury. [The SanBio Group (Business Wire, Inc.)] Press Release

CARsgen Announces Investigational CAR-T Therapy CT053 Granted PRIME Eligibility by the European Medicines Agency

CARsgen Therapeutics Inc. announced the European Medicines Agency has granted PRIority MEdicines (PRIME) eligibility to its investigational CAR-T cell therapy fully human anti-B Cell Maturation Antigen autologous CAR T Cells for the treatment of relapsed or refractory multiple myeloma. [CARsgen Therapeutics Inc. (PR Newswire Association LLC.)] Press Release

Kashmir’s Communication Blackout Is a ‘Devastating Blow’ for Academics, Researchers Say

Six professors from India’s top science institutions have appealed to the government to lift the blockade of academic and research institutions in Indian-controlled Kashmir. The blockade has been a “devastating blow,” the six write in an open letter published September 18^th. [ScienceInsider] Editorial

Senate Bill Would Give NIH $3 Billion in 2020, or 7.7% Boost

A Senate spending panel released a draft 2020 spending bill containing a hefty $3 billion increase for the National Institutes of Health (NIH) that would bring the agency’s total budget to $42.1 billion. That 7.7% boost is $1 billion more than a House of Representatives committee approved in its version of the bill in April, and would amount to a 40% increase in NIH’s budget over the past five years. [ScienceInsider] Editorial

FDA

Interacting with the Food and Drug Administration on Complex Innovative Clinical Trial Designs for Drugs and Biological Products; Draft Guidance for Industry; Availability (FR Doc. No:2019-20494) Notice

MHRA

Clinical Trials for Medicines: Authorisation Assessment Performance Notice

NEW From Stem Cell Biology to New Therapies
November 6-8, 2019
Toronto, Ontario, Canada

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NEW Assistant Professor – Regenerative Medicine (Washington University School of Medicine)

Research Technologist (STEMCELL Technologies Inc.)

Research Assistant – Cancer Research (BC Cancer Agency)

Postdoctoral Fellow – Cancer Research (Baylor College of Medicine)

Postdoctoral Fellow – Molecular Genetic Basis of Behavioral Variation (University of California, San Francisco)

Postdoctoral Fellow – Cancer Therapy (Purdue University)

Postdoctoral Researcher – Cell Cycles in Stem Cells (University of California, Santa Cruz)

Postdoctoral Fellowship – Cancer Immunotherapy (McGill University)

Postdoctoral Position – Gene Therapy of Muscular Dystrophy (Ohio State University)

Postdoctoral Scholar – Gene and Cell Therapy (Stanford University)

Research Scientist – Cellular Electrophysiology (Duke University)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center)

Tenure Track Faculty Position – Cancer Biology & Therapeutics (Geisel School of Medicine at Dartmouth)

Assistant/Associate or Professor – Cancer Immunotherapy/Tumor Immunology (University of California, Irvine)

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