Cell Therapy News Volume 18.40 | Dec 4 2017

TOP STORY

Single-Cut Genome Editing Restores Dystrophin Expression in a New Mouse Model of Muscular Dystrophy

Using CRISPR/Cas9, the authors generated a Duchenne muscular dystrophy mouse model by deleting exon 50. These ΔEx50 mice displayed severe muscle dysfunction, which was corrected by systemic delivery of adeno-associated virus encoding CRISPR/Cas9 genome editing components. [Sci Transl Med] Full Article | Press Release

(Ranked by impact factor of the journal)

Rescue of Pompe Disease in Mice by AAV-Mediated Liver Delivery of Secretable Acid α-Glucosidase

Scientists used adeno-associated virus (AAV) vectors optimized for hepatic expression to deliver the α-glucosidase (GAA) transgenes to Gaa knockout mice, a model of Pompe disease. [Sci Transl Med] Abstract

TEM8/ANTXR1-Specific CAR T Cells as a Targeted Therapy for Triple-Negative Breast Cancer

Adoptive transfer of TEM8 CAR T cells induced regression of established, localized patient-derived xenograft tumors as well as lung metastatic triple-negative breast cancer (TNBC) cell line-derived xenograft tumors, by both killing TEM8⁺ TNBC tumor cells and targeting the tumor endothelium to block tumor neovascularization. [Cancer Res] Abstract

High-Affinity GD2-Specific CAR T Cells Induce Fatal Encephalitis in a Preclinical Neuroblastoma Model

Scientists included the E101K mutation of GD2 that has enhanced antitumor activity against a GD2⁺ human neuroblastoma xenografts in vivo. However, this enhanced antitumor efficacy in vivo was concomitantly associated with lethal central nervous system toxicity comprised of extensive CAR T-cell infiltration and proliferation within the brain and neuronal destruction. [Cancer Immunol Res] Abstract

Delivery of CR2-fH Using AAV Vector Therapy as Treatment Strategy in the Mouse Model of Choroidal Neovascularization

Investigators tested an alternative approach of adeno-associated virus (AAV)-mediated delivery using subretinal delivery in C57BL/6J mice. [Mol Ther Methods Clin Dev] Full Article

Formulation of Stable and Homogeneous Cell-Penetrating Peptide NF55 Nanoparticles for Efficient Gene Delivery In Vivo

The authors introduce a new formulation approach called cryo-concentration, where they acquired stable and homogeneous nanoparticles for administration in vivo. [Mol Ther Nucleic Acids] Full Article | Graphical Abstract

Preclinical Evaluation and Optimization of a Cell Therapy Using Human Cord Blood-Derived Endothelial Colony-Forming Cells for Ischemic Retinopathies

Using a well-defined subpopulation of human cord blood-derived endothelial progenitors known as endothelial colony-forming cells, scientists evaluated essential requirements for further development of this cell therapy targeting the ischemic retina, including dose response, delivery route, and toxicity. [Stem Cells Transl Med] Full Article

In Situ Regeneration of Retinal Pigment Epithelium by Gene Transfer of E2F2: A Potential Strategy for Treatment of Macular Degenerations

Researchers used E2F2 to induce retinal pigment epithelium (RPE) cell replication and thereby regeneration. In both young and old wildtype mice, subretinal injection of non-integrating lentiviral vector expressing E2F2 resulted in 47% of examined RPE cells becoming BrdU positive. [Gene Ther] Abstract

Airway Transplantation of Adipose Stem Cells Protects against Bleomycin-Induced Pulmonary Fibrosis

Airway transplantation of adipose-derived stem cell significantly decreased the fibrosis rate in both early and established pulmonary fibrosis, modulating the expression of Hsp27, Vegfa, Nfkβ, IL-6, Col4, and Tgfβ1. [J Investig Med] Abstract

CAR-T Cell Therapies in Glioblastoma: A First Look

Results from three first-in-man CAR-T cell trials targeting IL13Rα2, Her2/CMV and EGFRvIII have recently been reported. Each one of these trials addresses important questions, such as T cell trafficking to CNS, engraftment and persistence, tumor microenvironment remodeling, and monitoring of glioma response to CAR-T cells. [Clin Cancer Res] Abstract

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

RXi Pharmaceuticals and the Center for Cancer Immune Therapy at Herlev Hospital Announce Research Collaboration

RXi Pharmaceuticals Corporation and the Center for Cancer Immune Therapy at Herlev Hospital announced that they have entered into a collaborative research agreement to evaluate the potential of RXi’s novel self-delivering RNAi technology platform in tumor infiltrating lymphocytess for the use in treatment for a number of cancer types, including melanoma and ovarian cancer. [RXi Pharmaceuticals Corporation] Press Release

Synpromics Announces Collaboration with UCL to Develop Revolutionary Gene Therapy for Parkinson’s Disease

Synpromics Ltd announced a new collaboration with University College London (UCL) to generate a range of synthetic gene promoters for the central nervous system, to develop a gene therapy for Parkinson’s disease. [Synpromics Ltd] Press Release

First Patient Enrolled in Phase III Trial for ATIR101™ in Adult Patients with Blood Cancer

Kiadis Pharma N.V. announced that the first patient has been enrolled in the HATCY Phase III clinical trial for ATIR101™. [Kiadis Pharma N.V.] Press Release

Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy

Solid Biosciences announced that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne muscular dystrophy. [Solid Biosciences] Press Release

bluebird bio Acquires Manufacturing Facility in North Carolina and Executes Multiple Global Supply Agreements to Enhance Ability to Deliver Gene Therapies to Patients

bluebird bio, Inc. announced it has acquired a 125,000-square foot manufacturing facility in Durham, North Carolina. Once construction and validation is complete, the site will produce lentiviral vector for the company’s gene and cell therapies. [bluebird bio, Inc.] Press Release

Moffitt Cancer Center Treats First U.S. Patient with Newly FDA-Approved CAR-T Therapy

Moffitt Cancer Center announced it treated the first patient commercially in the United States with the recently FDA-approved Yescarta™, a CAR-T therapy for patients with diffuse large B cell lymphoma, an aggressive form of non-Hodgkin lymphoma. [Moffitt Cancer Center] Press Release

Gritstone Oncology Opens New State-of-the-Art Personalized Cancer Immunotherapy Manufacturing Facility

Gritstone Oncology announced the opening of a new 43,000 square-foot manufacturing facility in Pleasanton, California. [Gritstone Oncology] Press Release

Biohackers Disregard FDA Warning on DIY Gene Therapy

Despite a warning from the federal government about do-it-yourself (DIY) gene therapy, two companies say they’ll continue offering DNA-altering materials to the public. The companies, The Odin and Ascendance Biomedical, both recently posted videos online of people self-administering DNA molecules their labs had produced. [MIT Technology Review] Editorial

Health Agency Reveals Scourge of Fake Drugs in Developing World

One in ten medicines in developing countries is fake or substandard, data from the World Health Organization suggest. Malaria drugs and antibiotics are among the most commonly reported, the agency found. But the problem extends to a variety of medications, including those for cancer, heart disease and HIV, as well as contraceptives and painkillers. [Nature News] Editorial

EMA

Procedural Guidance to Help Pharma Companies Prepare for Brexit Notice

NIH

Prospective Grant of Exclusive Patent Commercialization License: N6, A Novel, Broad, Highly Potent HIV-Specific Antibody (FR Doc. No:2017-25745) Notice

Prospective Grant of Exclusive Patent License: T-Cells Transduced with HLA A11 Restricted CT-RCC HERV-E Reactive T-Cell Receptors for the Treatment of Renal Cell Carcinoma (FR Doc. No:2017-25743) Notice

Prospective Grant of an Exclusive Patent License: Concatenated L2 Peptide Based Human Papillomavirus Vaccines (FR Doc. No:2017-25744) Notice

NEW 13^th Annual Wisconsin Stem Cell Symposium
April 18, 2018
Fitchburg/Madison, Wisconsin, United States

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

NEW Scientist – Gene Therapy (Medicines and Healthcare Products Regulatory Agency)

Research Technologist – Immunotherapy (STEMCELL Technologies Inc.)

Postdoctoral Positions – Immunotherapy (University of Houston)

Research Scientist – Cellular Immunotherapy (Columbia Center for Translational Immunology)

Assistant Scientist – Cancer Therapy Research (University of Miami)

Postgraduate Positions – Translational Medicine (Qingdao University)

Postdoctoral Position – Cancer Research (Helmholtz Zentrum München)

Scientist – Immuno-Oncology (Regeneron Pharmaceuticals, Inc.)

Postdoctoral Fellows – ImmunoGenomics (San Raffaele-Telethon Institute for Gene Therapy)

Postdoctoral Fellow – Cardiac Regeneration (Kyoto University)

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

Postdoctoral Fellow – Various Projects (University of Oklahoma)

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