Cell Therapy News Volume 21.10 | Mar 23 2020

TOP STORY

Human Chimeric Antigen Receptor Macrophages for Cancer Immunotherapy

In two solid tumor xenograft mouse models, a single infusion of human chimeric antigen receptor-macrophages decreased tumor burden and prolonged overall survival. [Nat Biotechnol] Abstract | Press Release

(Ranked by impact factor of the journal)

RecV Recombinase System for In Vivo Targeted Optogenomic Modifications of Single Cells or Cell Populations

To allow targeting of individual neurons for structural and functional studies, scientists created light-inducible site-specific DNA recombinases based on Cre, Dre and Flp (RecVs). RecVs could induce genomic modifications by one-photon or two-photon light induction in vivo. [Nat Methods] Abstract

Systemic AAV9.LAMP2B Injection Reverses Metabolic and Physiologic Multiorgan Dysfunction in a Murine Model of Danon Disease

Lysosomal-associated membrane protein 2 (Lamp2) KO mice receiving LAMP2B gene transfer using a recombinant adeno-associated virus 9 carrying human LAMP2B (AAV9.LAMP2B) demonstrated dose-dependent restoration of human LAMP2B protein in the heart, liver, and skeletal muscle tissue. [Sci Transl Med] Abstract

Development of a CRISPR-SaCas9 System for Projection- and Function-Specific Gene Editing in the Rat Brain

Investigators developed a CRISPR-SaCas9 system-based technique, and its combined application with anterograde/retrograde adeno-associated virus vectors and activity-dependent cell-labeling techniques achieved projection- and function-specific gene editing in the rat brain. [Sci Adv] Full Article

AAV Capsid-Promoter Interactions Determine CNS Cell Selective Gene Expression In Vivo

For adeno-associated virus (AAV)9 vectors CBA promoter driven gene expression exhibited a dominant neuronal gene expression in the rat striatum. For otherwise identical AAV9 vectors the truncated CBh promoter shifted gene expression towards striatal oligodendrocytes. In contrast AAV2 vector gene expression was restricted to striatal neurons, regardless of the constitutive promoter used. [Mol Ther] Abstract | Graphical Abstract

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy

Scientists provided preclinical data to support initiation of a first-in-human trial for sickle cell disease using an approach which relied on reversal of the developmental fetal-to-adult hemoglobin switch. [Mol Ther Methods Clin Dev] Abstract | Full Article

Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice

The authors evaluated a combination of peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) and adeno-associated virus (AAV)-based therapy in a mouse model of severe Duchenne muscular dystrophy (DMD). Striking benefits of this combined therapy were observed in striated muscles, with marked improvements in heart and diaphragm structure and function with unrivalled extend of survival, opening novel therapeutic perspectives for patients. [Mol Ther Methods Clin Dev] Abstract | Graphical Abstract

Assessing the Impact of AGS-004, a Dendritic Cell-Based Immunotherapy, and Vorinostat on Persistent HIV-1 Infection

Scientists investigated the combined impact of the latency reversing agent vorinostat and AGS-004, an autologous dendritic cell immunotherapeutic, on the HIV reservoir. [Sci Rep] Full Article

Targeted DNA Demethylation of the Fgf21 Promoter by CRISPR/dCas9-Mediated Epigenome Editing

Researchers employed the dCas9-SunTag and single-chain variable fragment-TET1 catalytic domain system to induce targeted DNA methylation of the Fgf21 promoter both in vitro and in vivo. [Sci Rep] Full Article

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Non-Invasive Reporter Gene Imaging of Cell Therapies, Including T Cells and Stem Cells

The authors explain how in vivo imaging can aid the development and clinical translation of cell-based therapeutics. They describe the underlying principles governing non-invasive in vivo long-term cell tracking in the preclinical and clinical settings including available imaging technologies, reporter genes and imaging agents as well as pitfalls related to experimental design. [Mol Ther] Abstract | Graphical Abstract

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

Dragonfly Therapeutics Announces Multi-Target Expansion of Its Collaboration with Merck to Use Dragonfly’s Proprietary TriNKET^™ Platform to Develop Novel Therapeutic Candidates in the Fields of Oncology, Infectious Disease, and Immune Disorders

Dragonfly Therapeutics, Inc. announced an expansion of its strategic collaboration with Merck to discover, develop and commercialize a number of Dragonfly’s candidate natural killer cell engager immunotherapies for oncology, infectious disease, and immune disorders. [Dragonfly Therapeutics, Inc. (PR Newswire Association LLC)] Press Release

Abeona Therapeutics Announces First Patient Treated in Pivotal Phase III Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

Abeona Therapeutics Inc. announced that investigators at Stanford University Medical Center have treated the first patient in the pivotal Phase III VIITAL^™ study evaluating EB-101, the company’s gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa. [Abeona Therapeutics Inc.] Press Release

NantKwest Doses First Patient in Phase II Single-Arm Trial of Metastatic Merkel Cell Carcinoma Therapy with Off-the-Shelf CD16 Natural Killer Cells

NantKwest, Inc. announced dosing of the first patient in a Phase II trial for second- and third-line Merkel cell carcinoma in patients who are refractory to immune checkpoint inhibitors. [NantKwest, Inc.] Press Release

Cellenkos Inc. Focuses on the Development of Cell-Therapy for Treatment of COVID-19 Mediated Acute Respiratory Distress Syndrome (CoV-ARDS)

Cellenkos, Inc. announced that the company has submitted a clinical development proposal to the Biomedical Advanced Research and Development Authority to start a Phase I/II clinical trial with CK0802 for treatment of CoV-ARDS. [Cellenkos, Inc. (PR Newswire Association LLC)] Press Release

AgeX Therapeutics Licensee ImStem Biotechnology Announces FDA Has Lifted the Hold and Cleared the Investigational New Drug Application for IMS001 for the Treatment of Multiple Sclerosis

AgeX Therapeutics, Inc. announced that ImStem Biotechnology, Inc. has received notification from the FDA that it has lifted the hold and cleared the Investigational New Drug application to evaluate IMS001 for the treatment of multiple sclerosis. [AgeX Therapeutics, Inc.] Press Release

Amid Coronavirus Shutdowns, Some Grad Students Feel Pressure to Report to Their Labs

As the coronavirus outbreak roils university campuses across the world, early-career scientists are facing several dilemmas. Many are worrying about the survival of cell cultures, laboratory animals, and other projects critical to their career success. And some are reporting feeling unwelcome pressure to report to their laboratories. [ScienceInsider] Editorial

Universities Urge US Leaders to Boost Science Budgets by 15%, Ease Rules to Cope with Pandemic

The US research community is urgently asking the White House and Congress to take steps aimed at keeping academic research—as well as museums, botanic gardens, and zoos—afloat during the coronavirus pandemic. [ScienceInsider] Editorial

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NEW Professorship – Biological Therapies of the Inner Ear (Hannover Medical School)

NEW Group leaders – Cancer Biology (Institute of Pharmacology and Structural Biology)

Scientist – Cancer Immunotherapy (Lund University)

Postdoctoral Position – Cancer Immunotherapy (The University of Texas MD Anderson Cancer Center)

Sr Associate Scientist – Autoimmunity and Tumor Immunotherapy (Amgen)

Research Associate – Stem Cell and Gene Therapy Program (Fred Hutchinson Cancer Research Center)

Lecturer – Clinical Pharmacology (University of Bristol)

Postdoctoral Fellow – Cancer Research (BC Cancer)

Research Assistant – Cancer Research (BC Cancer)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer)

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