Cell Therapy News Volume 19.08 | Mar 12 2018

TOP STORY

Selective Targeting of Engineered T Cells Using Orthogonal IL-2 Cytokine-Receptor Complexes

Introduction of orthogonal (ortho) interleukin-2 (IL-2)Rβ into T cells enabled the selective cellular targeting of orthoIL-2 to engineered CD4⁺ and CD8⁺ T cells in vitro and in vivo, with limited off-target effects and negligible toxicity. [Science] Abstract | Press Release

(Ranked by impact factor of the journal)

BEST1 Gene Therapy Corrects a Diffuse Retina-Wide Microdetachment Modulated by Light Exposure

Scientists found that the loss of retinal pigment epithelium apical microvilli and resulting microdetachment of the retina represent the earliest features of canine bestrophinopathies. They showed that retinal light exposure expands, and dark adaptation contracts, the microdetachments. Subretinal adeno-associated virus-based gene therapy corrected both the vitelliform lesions and the light-modulated microdetachments. [Proc Natl Acad Sci USA] Full Article | Press Release

Neurturin Gene Therapy Protects Parasympathetic Function to Prevent Irradiation-Induced Murine Salivary Gland Hypofunction

Neurturin adenovirus was delivered to mouse salivary glands in vivo, 24 hours before irradiation, and compared with a control adenovirus. The control-treated glands had ~50% reduction in salivary flow 60 days post-irradiation, whereas neurturin-treated glands had similar flow to nonirradiated glands. [Mol Ther Methods Clin Dev] Full Article

Myeloid Conditioning with c-Kit-Targeted CAR-T Cells Enables Donor Stem Cell Engraftment

Pre-treatment of recipient mice with low-dose cyclophosphamide together with CXCR4 transduction in the CAR-T cells enhanced trafficking to and expansion in bone marrow (BM). This resulted in significant depletion of the BM c-kit⁺ population. [Mol Ther] Abstract

Embryonic Stem Cell-Derived Mesenchymal Stem Cells (MSCs) Have a Superior Neuroprotective Capacity over Fetal MSCs in the Hypoxic-Ischemic Mouse Brain

Scientists compared the therapeutic potential of pluripotent stem cell-derived-MSCs (ES-MSCs from embryonic stem cells) to fetal MSCs (AF-MSCs from the amniotic fluid), demonstrating that ES-MSCs have a superior neuroprotective potential over AF-MSCs in the mouse brain following hypoxia-ischemia. [Stem Cells Transl Med] Full Article

Human Umbilical Cord Matrix Stem Cells Reverse Oxidative Stress-Induced Cell Death and Ameliorate Motor Function and Striatal Atrophy in Rat Model of Huntington Disease

Researchers evaluated the in vitro and in vivo efficacy of umbilical cord matrix stem cells and their paracrine effect against oxidative stress with a specific focus on Huntington disease. [Neurotox Res] Abstract

In Vivo Selection with Lentiviral Expression of Bcl2^{T69A/S70A/S87A} Mutant in Hematopoietic Stem Cell-Transplanted Mice

The authors demonstrated in hematopoietic stem cell-transplanted mice that Bcl2^{T69A/S70A/S87A} facilitated efficient in vivo selection in the absence of any exogenously applied drugs under both myeloablative and non-myeloablative conditioning. [Gene Ther] Abstract

Improving Therapeutic Efficacy of IL-12 Intratumoral Gene Electrotransfer through Novel Plasmid Design and Modified Parameters

IL-12 expression from a plasmid incorporating a picornavirus-derived co-translational P2A site was the most effective in expressing IL-12p70. In addition, modifying the electroporation parameters improved transfection efficiency and expression of plasmid-derived IL-12p70, as well as its downstream effector IFN-γ in vivo. [Gene Ther] Abstract | Press Release

T-Bet Promotes Potent Antitumor Activity of CD4⁺ CAR T Cells

In vivo, CD4⁺ T cells co-expressing a B7H6-specific CAR and T-bet improved the survival of RMA-B7H6 lymphoma-bearing mice. [Cancer Gene Ther] Full Article

Feasibility Study of Human Corneal Endothelial Cell Transplantation Using an In Vitro Human Corneal Model

Researchers tested the feasibility of a cell therapy approach to treat corneal endothelial disorders using an in vitro model of human corneal decompensation. [Cornea] Abstract

Cerebrospinal Fluid-Stem Cell Interactions May Pave the Path for Cell-Based Therapy in Neurological Diseases

The authors review the literature on the influence of the cerebrospinal fluid on stem cells in order to provide a more comprehensive reference for the wide clinical application of neural stem cells in the future. [Stem Cell Res Ther] Full Article

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

STEMCELL Technologies Signs Exclusive License with IU Innovation and Commercialization Office for Inner Ear Organoid Technology

STEMCELL Technologies has signed an exclusive license with Indiana University Innovation and Commercialization Office for patented rights to groundbreaking inner ear organoid technologies. [STEMCELL Technologies Inc.] Press Release

MD Anderson and Berkeley Lights Launch Optera Therapeutics to Accelerate Development of Cell Therapies for Cancer

The University of Texas MD Anderson Cancer Center and Berkeley Lights, Inc. announced the launch of Optera Therapeutics Corp, a biopharmaceutical company developing cell therapies with scalable manufacturing solutions for cancer. [The University of Texas MD Anderson Cancer Center] Press Release

Voyager Therapeutics Announces Longer-Term Data from Ongoing Phase Ib Trial of VY-AADC for Advanced Parkinson’s Disease

Voyager Therapeutics, Inc. announced longer-term data from its ongoing dose-ranging Phase Ib trial of VY-aromatic L-amino acid decarboxylase (AADC) in advanced Parkinson’s disease. [Voyager Therapeutics, Inc.] Press Release

Alnylam Retains Global Rights to Lumasiran, an Investigational RNAi Therapeutic for the Treatment of Primary Hyperoxaluria Type 1 (PH1)

Alnylam Pharmaceuticals, Inc. announced that Sanofi Genzyme has declined its opt-in for the development and commercialization of lumasiran, an investigational RNAi therapeutic for the treatment of PH1. [Alnylam Pharmaceuticals, Inc.] Press Release

Arrowhead Begins Dosing in Phase I Study of ARO-AAT for Treatment of Alpha-1 Liver Disease

Arrowhead Pharmaceuticals Inc. announced that it has dosed the first subjects in a Phase I clinical study of ARO-AAT, which is being developed as treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency. [Arrowhead Pharmaceuticals Inc.] Press Release

Celgene Completes Acquisition of Juno Therapeutics, Inc., Advancing Global Leadership in Cellular Immunotherapy

Celgene Corporation announced that it has completed the acquisition of Juno Therapeutics, Inc. [Celgene Corporation] Press Release

Caladrius Biosciences Acquires an Exclusive License to a Late Stage CD34⁺ Cell Therapy Program for the Treatment of Refractory Angina

Caladrius Biosciences, Inc. announces that the company acquired from Shire plc an exclusive worldwide license to data from a late stage CD34⁺ cell therapy program for the treatment of chronic myocardial ischemia targeting refractory angina. [Caladrius Biosciences, Inc.] Press Release

Fibrocell Announces FDA Allowance of Investigational New Drug Application for FCX-013 for the Treatment of Moderate to Severe Localized Scleroderma

Fibrocell Science, Inc. announced that the FDA has granted allowance of its Investigational New Drug Application for FCX-013—one of the company’s gene therapy candidates—to begin clinical trials for the treatment of moderate to severe localized scleroderma. [Fibrocell Science, Inc.] Press Release

Balancing Safety and Innovation for Cell-Based Regenerative Medicine

Regenerative medicine is a field that involves replacing, engineering, or regenerating human cells, tissues, or organs to establish, restore, or enhance normal function. It is an area with great promise that goes directly to the role of the FDA in helping to facilitate the availability of safe and effective treatments. [N Engl J Med] Editorial

Key Republican Lawmaker Releases New ‘Right-to-Try’ Legislation

A key Republican lawmaker released a new version of so-called “right to try” legislation, a pivotal next step in the effort to help dying patients get access to experimental therapies. [STAT News] Editorial

Scientists Win and Lose in Texas Primary Contests

Texas primary was the first test for scientists seeking seats in the U.S. House of Representatives, and the results were mixed. [ScienceInsider] Editorial

Behavioral ‘Violations’ Cost Prominent Neuroscientist Positions at Columbia, HHMI

Prominent Columbia University neuroscientist Tom Jessell, 66, has been fired for “serious [behavioral] violations” and the university is closing his lab, the New York City institution said in a statement. The Howard Hughes Medical Institute (HHMI) in Chevy Chase, Maryland, which effectively employed Jessell, a leader in understanding neural development, also terminated him as an investigator because he “violated HHMI policy,” according to a statement from the institute. [ScienceInsider] Editorial

MHRA

Clinical Trials for Medicines: Apply for Authorization in the UK Notice

NEW International Society for Stem Cell Research (ISSCR) 2018 Annual Meeting
June 20-23, 2018
Melbourne, Australia

Visit our events page to see a complete list of events in the community.

NEW Assistant Professor – Translation of Preclinical Regenerative Medicine into Advanced Therapy Medicinal Products (Lund University)

Postdoctoral Research Fellow – Hematopoiesis (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

Postdoctoral Fellowship – Functional Genomics of Human Hematopoiesis (Boston Children’s Hospital)

Postdoctoral Researcher – Molecular Cardiology (National Centre for Cardiovascular Research)

Postdoctoral Fellow – Cancer Research (Indiana University School of Medicine)

Postdoctoral Associate – Cardiovascular Science (Duke University)

Postdoctoral Position – Pancreas Development and Stem Cells (Helmholtz Zentrum München)

Postdoctoral Fellowships – Cancer Research (University of Miami)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

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