Cell Therapy News Volume 20.33 | Sep 30 2019

TOP STORY

Delivery of mRNA Vaccines with Heterocyclic Lipids Increases Anti-Tumor Efficacy by Sting-Mediated Immune Cell Activation

Researchers developed a combinatorial library of ionizable lipid-like materials to identify mRNA delivery vehicles that facilitate mRNA delivery in vivo and provide potent and specific immune activation. [Nat Biotechnol] Abstract | Press Release

(Ranked by impact factor of the journal)

CAR T Cells Targeting BAFF-R Can Overcome CD19 Antigen Loss in B Cell Malignancies

B cell activating factor receptor-CAR, but not CD19-CAR, T cells demonstrated antitumor effects against an additional CD19 antigen loss primary patient-derived xenograft in vivo. [Sci Transl Med] Full Article

Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements

Investigators used published genomic and epigenomic data available through ENCODE to redefine enhancer element boundaries of the β-globin locus control region to construct novel ENCODE core sequences. At low vector copy number, vectors containing the ENCODE core sequences were capable of reversing the sickle phenotype in a mouse model of sickle cell disease. [Mol Ther] Abstract

Efficient CRISPR/Cas9-Mediated Gene Knockin in Mouse Hematopoietic Stem and Progenitor Cells

The authors describe an efficient CRISPR/Cas9-based system to knock in and repair genes in mouse hematopoietic stem and progenitor cells (HSPCs). They corrected a neomycin-disrupted Rag2 gene. The Rag2-corrected HSPCs restored B and T cell development in vivo. [Cell Rep] Full Article | Graphical Abstract

An Anti-CD103 Antibody-Drug Conjugate Prolongs the Survival of Pancreatic Islet Allografts in Mice

Targeting CD103 with M290-MC-MMAF induced immunosuppression and prolonged the survival of pancreatic islet allografts in mice. [Cell Death Dis] Full Article

Regenerative Potential of Human Dental Pulp Stem Cells in the Treatment of Stress Urinary Incontinence: In Vitro and In Vivo Study

Human dental pulp stem cells (hDPSCs) were able to commit towards myogenic lineage in vitro and, four weeks after cell injection, hDPSCs engrafted in the external urethral sphincter whose thickness was almost recovered, committed towards myogenic lineage in vivo, promoted vascularization and an appreciable recovery of the continence. [Cell Prolif] Abstract

Therapeutic Effect of Mesenchymal Stem Cells Derived from Human Umbilical Cord in Rabbit Temporomandibular Joint Model of Osteoarthritis

Compared with the untreated control group, the potential regenerative result and anti-inflammatory effects of human umbilical cord matrix-mesenchymal stem cells were evident at all the tested concentrations in rabbits with induced temporomandibular joint osteoarthritis. [Sci Rep] Full Article

Human Umbilical Cord Perivascular Cells-Derived Extracellular Vesicles Mediate the Transfer of IGF-I to the Liver and Ameliorate Hepatic Fibrogenesis in Mice

Scientists evaluated the therapeutic potential of human umbilical cord perivascular cells engineered to produce insulin growth factor like-I (IGF-I) in experimental liver fibrosis and the role of extracellular vesicles in this effect. [Gene Ther] Abstract

Superior Human Hepatocyte Transduction with Adeno-Associated Virus Vector Serotype 7

Researchers evaluated adeno-associated virus (AAV) vector transduction from several serotypes in human liver hepatocytes xenografted into chimeric mice. After systemic administration of AAV vectors encoding a GFP transgene in humanized mice, the liver was harvested for either immunohistochemistry staining or flow cytometry assay for AAV human hepatocyte transduction analysis. [Gene Ther] Abstract

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T Cell Engineering for Adoptive T Cell Therapy: Safety and Receptor Avidity

The authors describe how ‘optimal’ T cell receptor/chimeric antigen receptor affinity can increase the safety/efficacy balance of engineered T cells, and discuss simultaneous or sequential infusion of high and low avidity receptors as further options for efficacious but safe T cell therapy. [Cancer Immunol Immunother] Abstract

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Neuralstem Announces Last Subject Enrolled in Phase II Clinical Trial Evaluating Human Neural Stem Cells in Chronic Stroke Patients

Neuralstem, Inc. announced completion of subject enrollment in its Phase II clinical trial of NSI-566 for the treatment of chronic ischemic stroke. [Neuralstem, Inc.] Press Release

Kadimastem Announces Promising Interim Results of Cohort A of its Phase I/IIa Clinical Trial in ALS

Kadimastem Ltd. announced promising interim results of cohort A of its Phase I/IIa clinical trial for the treatment of patients with amyotrophic lateral sclerosis (ALS). [Kadimastem Ltd.] Press Release

BrainStorm Cell Therapeutics Announces Support for FDA Guidelines on ALS Drug Development

BrainStorm Cell Therapeutics Inc. supports the US FDA’s publication on September 24, 2019 of “Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry.” This document outlines ALS drug development guidelines and potential opportunities to advance novel ALS therapies through innovative clinical trial design and validated clinical outcome measures. [BrainStorm Cell Therapeutics Inc.] Press Release

Capricor Therapeutics to Meet with FDA to Discuss CAP-1002 to Treat Duchenne Muscular Dystrophy

Capricor Therapeutics announced that it has been granted a Type B End-of-Phase II meeting with the FDA to discuss prespecified interim analysis of the company’s HOPE 2 clinical trial. [Capricor Therapeutics] Press Release

Major Error Undermines Study Suggesting Change Introduced in the CRISPR Babies Experiment Shortens Lives

A scientific study published this past spring came with damning implications for Chinese scientist He Jiankui, who created the world’s first gene-edited babies: People with the rare genetic variants that He tried to engineer into embryos, the study asserted, had an increased death rate. On Friday, the paper’s senior author said his study was wrong. [STAT News] Editorial

NIH Reveals Its Formula for Tracking Foreign Influences

The US National Institutes of Health (NIH) thinks it may have figured out how China’s foreign talents recruitment program is undermining its system for making awards and ensuring ethical behavior by its grantees. [ScienceInsider] Editorial

The Potent Effects of Japan’s Stem-Cell Policies

A five-year regulatory free-for-all in regenerative medicine has given the industry a boost. But patients might be paying the price. [Nature News] Editorial

NIH

Prospective Grant of Exclusive Patent License: Capsid-Free AAV Vectors, Compositions, and Methods for Vector Production and Gene Delivery (FR Doc. No:2019-20992) Notice

NEW Indonesia International Institute for Life Sciences (i3L) Conference 2019
November 5-7, 2019
East Jakarta, Indonesia

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NEW Postdoctoral Fellow – Cancer Research (BC Cancer)

Research Technologist (STEMCELL Technologies Inc.)

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

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 Fellowship – Cancer Immunotherapy (McGill University)

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

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