Cell Therapy News Volume 20.10 | Mar 25 2019

TOP STORY

IL-15-Mediated Reduction of mTORC1 Activity Preserves the Stem Cell Memory Phenotype of CAR-T Cells and Confers Superior Antitumor Activity

Chimeric antigen receptor (CAR)-T/IL-15 promoted superior antitumor responses in vivo in comparison to CAR-T/IL-2. Inclusion of additional cytokines with IL-15, either IL-7 and/or IL-21, reduced the beneficial effects of IL-15 for CAR-T phenotype and antitumor potency. [Cancer Immunol Res] Abstract

(Ranked by impact factor of the journal)

Adoptive Transfer of NKG2D CAR mRNA-Engineered Natural Killer Cells in Colorectal Cancer Patients

Three patients with metastatic colorectal cancer were treated with local infusion of chimeric antigen receptor (CAR)-natural killer (NK) cells. Reduction of ascites generation and a marked decrease in number of tumor cells in ascites samples were observed in the first two patients treated with intraperitoneal infusion of low doses of the CAR-NK cells. [Mol Ther] Abstract

A Self-Assembled DNA-Nanoparticle with a Targeting Peptide for Hypoxia-Inducible Gene Therapy of Ischemic Stroke

In stroke animal models, nanoparticle with hypoxia-specific anti-RAGE peptide (HSAP-NP) reduced the levels of RAGE, inducible nitric oxide synthase, and inflammation. Additionally, HSAP-NP with heme oxygenase-1 plasmid (pHO1) increased HO1 expression in the ischemic brain. [Biomater Sci] Abstract

The Alpha-1-Antitrypsin Promoter Improves the Efficacy of an AAV Vector for the Treatment of MNGIE

The authors studied the effect of various promoters and DNA configuration on expression of the TYMP transgene in the adeno-associated vector 8 serotype in a murine model of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). [Hum Gene Ther] Abstract

Three-Year Follow-Up of Phase I and IIa rAAV.sFLT-1 Subretinal Gene Therapy Trials for Exudative Age Related Macular Degeneration

Scientists assessed the safety and the three-year results of combined Phase I and IIa randomized controlled trials of rAAV.sFLT-1 gene therapy for wet-AMD. [Am J Ophthalmol] Abstract

Enrichment of Vascular Endothelial Growth Factor Secreting Mesenchymal Stromal Cells Enhances Therapeutic Angiogenesis in a Mouse Model of Hind Limb Ischemia

Mesenchymal stromal cells were transfected to co-express vascular endothelial growth factor as an angiogenic factor and enhanced green fluorescent protein as a marker. Transfected cells were then collected using flow cytometry based on green fluorescence and transplanted into ischemic hind limbs in mice. [Cytotherapy] Abstract

Nano-Mediated Delivery of Double-Stranded RNA for Gene Therapy of Glioblastoma Multiforme

Investigators report new approach towards RNA interference therapy of glioblastoma multiforme based on the magnetic nanoparticles delivery of the double-stranded RNA with homological sequences to mRNA of tenascin-C, named ATN-RNA. [PLoS One] Full Article

Integration of C-Type Natriuretic Peptide Gene-Modified Bone Marrow Mesenchymal Stem Cells with Chitosan/Silk Fibroin Scaffolds as a Promising Strategy for Articular Cartilage Regeneration

Scientists successfully synthesized a chitosan/silk fibroin (CS/SF) porous scaffold, which had a suitable aperture size for chondrogenesis. They loaded C-type natriuretic peptide gene-modified bone marrow-derived mesenchymal stem cells onto CS/SF scaffolds and tested their effect on repairing full-thickness cartilage defects in rat joints. [Cell Tissue Bank] Abstract

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CRISPR/Cas9-Modified Hematopoietic Stem Cells—Present and Future Perspectives for Stem Cell Transplantation

The authors summarize the possibility of CRISPR/Cas9-mediated autologous hematopoietic stem cell transplantation as a potential treatment option for various diseases supported by preclinical gene-editing studies. [Bone Marrow Transplant] Abstract

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VBL Presents New Data on Potential of VB-111 to Stimulate the Immune System and Drive Immune Cells to Infiltrate Tumor Microenvironment

VBL Therapeutics announced the presentation of human data indicating that the viral anti-cancer investigational therapy VB-111 has potential to stimulate the immune system to induce a strong and durable response against ovarian tumors. [Press release from VBL Therapeutics discussing research presented at the SGO 50^th Annual Meeting on Women’s Cancer, Honolulu] Press Release

Humanigen Data Demonstrates Potential to Improve the Efficacy of CAR-T Therapy

Humanigen, Inc. announced that data from the study entitled “GM-CSF Blockade during Chimeric Antigen Receptor T-cell (CART) Therapy Reduces Cytokine Release Syndrome and Neurotoxicity and may Enhance CART Effector Function” were presented. [Press release from Humanigen, Inc. discussing research presented at the 2019 NCCN Annual Conference: Improving the Quality, Effectiveness, & Efficiency of Cancer Care™, Orlando] Press Release

Phio Pharmaceuticals to Present Poster on the Use of Self-Delivering RNAi in Immuno-Oncology

Phio Pharmaceuticals Corp. announced that the company will present a poster. [Press release from Phio Pharmaceuticals Corp. discussing research to be presented at the 2019 American Association for Cancer Research (AACR) Annual Meeting, Atlanta] Press Release

NJII and Pall Corporation Form Agreement to Advance Cell and Gene Therapy Manufacturing

New Jersey Innovation Institute (NJII) has signed an Industry Participation Agreement with Pall Corporation to support the development of its BioPharmaceutical Innovation iLab. The partnership will support two key Centers at NJII that will advance the manufacturing of new cell and gene therapies: the Cell and Gene Therapy Development Center and the Center of Advanced Biologic Manufacturing. [Pall Corporation] Press Release

Pfizer Secures Exclusive Option to Acquire Gene Therapy Company Vivet Therapeutics

Vivet Therapeutics and Pfizer Inc. announced that Pfizer has acquired a 15% equity interest in Vivet and secured an exclusive option to acquire all outstanding shares. Pfizer and Vivet will collaborate on the development of VTX-801, Vivet’s proprietary treatment for Wilson disease. [Vivet Therapeutics] Press Release

Trizell Ltd. Announces Phase III Pivotal Study of Interferon alfa-2b Gene Therapy in Malignant Pleural Mesothelioma

Trizell Ltd announced that it has opened a Phase III Study of its novel gene therapy TR002, an adenovirus-mediated interferon alfa 2b, in patients with malignant pleural mesothelioma who have failed first-line standard of care chemotherapy. [Trizell Ltd (PR Newswire Association LLC)] Press Release

Mogrify Awarded $555,000 USD (£420,000 GBP) Innovate UK Funding to Accelerate Regenerative Cell Therapies

Cell Mogrify Ltd announced that it has been awarded $555,000 USD funding from Innovate UK through the investment accelerator for innovation in precision medicine. [Cell Mogrify Ltd] Press Release

bluebird bio Opens State-of-the-Art Gene and Cell Therapy Manufacturing Facility in Durham, North Carolina

bluebird bio, Inc. announced the official opening of its first wholly owned manufacturing facility in Durham, N.C., that will produce lentiviral vector for the company’s investigational gene and cell therapies, including: bb2121 and bb21217 for the treatment of multiple myeloma and potentially LentiGlobin™ for the treatment of transfusion-dependent β-thalassemia and sickle cell disease. [bluebird bio, Inc.] Press Release

Orgenesis Subsidiary Masthercell Global Announces Plans to Establish New Facility for Cell and Gene Therapy Commercial Manufacturing

Orgenesis Inc. announced plans to establish a new, state-of-the-art production site for its Masthercell Global subsidiary, designed to manufacture late-stage and commercially-approved cell and gene therapy products. [Orgenesis Inc.] Press Release

Scientists Among Thousands Marching to Demand Say on Brexit

Scientists took to the streets of London on 23 March as part of a major demonstration calling for the terms of Brexit to be put to the British people in a vote. [Nature News] Editorial

Chinese-American Scientist Societies Fear Racial Profiling

In a strongly worded letter published in Science, a group of Chinese-American scientists voiced concern that recent proposals from the National Institutes of Health and FBI actions could lead to unjust targeting of ethnically Chinese scientists. [The Scientist] Editorial

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NEW Postdoctoral Position – Immunotherapy (McGill University)

Research Technologist – Immunology (STEMCELL Technologies Inc.)

Research Associate – Bioengineering (STEMCELL Technologies Inc.)

Postdoctoral Research Associate – Retinal Stem Cell Biology and Regeneration (University of Nebraska Medical Center)

Cell Biologist – Regenerative Medicine (Hepatx Corporation)

Stem Cell Podcast Co-Host (Stem Cell Podcast)

Associate Scientist – Immuno-Oncology (HMH Center for Discovery & Innovation)

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

Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma)

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

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

Cell Manufacturing Scientist (CSL Behring)

Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University)

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

Faculty Positions – Hematology and Oncology (University of California, Davis)

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