Cell Therapy News Volume 21.19 | Jun 1 2020

TOP STORY

IgG-Cleaving Endopeptidase Enables In Vivo Gene Therapy in the Presence of Anti-AAV Neutralizing Antibodies

In mice passively immunized with intravenous Ig, imlifidase administration decreased anti-AAV antibodies and enabled efficient liver gene transfer. The approach was scaled up to nonhuman primates, a natural host for wild-type AAV. [Nat Med] Abstract

(Ranked by impact factor of the journal)

The Rational Development of CD133-Targeting Immunotherapies for Glioblastoma

Scientists report three immunotherapeutic modalities based on a human anti-CD133 antibody fragment that targeted a unique epitope present in glycosylated and non-glycosylated CD133 and studied their effects on targeting CD133⁺ cells in patient-derived models of glioblastoma. [Cell Stem Cell] Abstract | Graphical Abstract

Functional Reconstruction of Injured Corpus Cavernosa Using 3D-Printed Hydrogel Scaffolds Seeded with HIF-1α-Expressing Stem Cells

The biodegradable scaffolds, along with muscle-derived stem cells, were implanted into corpus cavernosa defects in a rabbit model to show good histocompatibility with no immunological rejection, support vascularized tissue ingrowth, and promote neovascularisation to repair the defects. [Nat Commun] Full Article

Ad-CD40L Mobilizes CD4 T Cells for the Treatment of Brainstem Tumors

The authors investigated whether direct delivery of adenovirus expressing CD40L (Ad-CD40L) to brainstem tumors would induce immune-mediated tumor clearance and, if so, whether therapy would be associated with a manageable toxicity due to immune-mediated inflammation in the brainstem. [Neuro Oncol] Full Article

Lentiviral Vector-Based Dendritic Cell Vaccine Synergizes with Checkpoint Blockade to Clear Chronic Viral Infection

The authors report on a lentiviral vector-based dendritic cell vaccine strategy that generated a CD8 T cell response that was much stronger than that achieved by standard peptide-pulsing approaches. The strategy was tested in the mouse lymphocytic choriomeningitis virus model. [Mol Ther] Abstract | Graphical Abstract

Therapeutic Efficacy of Anti-CD19 CAR-T Cells in a Mouse Model of Systemic Lupus Erythematosus

The transfer of syngeneic anti-CD19 CAR-T cells not only prevented disease pathogenesis before the onset of disease symptoms but also displayed therapeutic benefits at a later stage after disease progression. [Cell Mol Immunol] Abstract

Treating Bietti Crystalline Dystrophy in a High-Fat Diet-Exacerbated Murine Model Using Gene Therapy

Scientists revealed that mouse models lacking Cyp4v3 had less physiological and functional changes than those of Bietti crystalline dystrophy (BCD) patients with this gene defect. [Gene Ther] Abstract

Dendritic Cells with METTL3 Gene Knockdown Exhibit Immature Properties and Prolong Allograft Survival

In vivo, researchers found that methyltransferase-like protein 3 (METTL3)-silencing donor dendritic cells induced immune tolerance after mouse heart transplantation and prolonged the allograft survival. [Genes Immun] Abstract

Mesenchymal Stromal Cells Induce Distinct Myeloid-Derived Suppressor Cells in Inflammation

Mesenchymal stem/stromal cells (MSCs) directed the differentiation of Ly6G^lo BM cells from CD11b^hiLy6C^hi to CD11b^midLy6C^mid cells both in cell contact-independent and -dependent manners upon GM-CSF stimulation in vitro and in mice with experimental autoimmune uveoretinitis. [JCI Insight] Abstract | Full Article | Graphical Abstract

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The Future of Cancer Immunotherapy: Microenvironment-Targeting Combinations

In addressing frontiers in clinical immunotherapy, the authors describe two categories of approaches to the design of novel drugs and combination therapies: the first involves direct modification of the tumor, while the second indirectly enhances immunogenicity through alteration of the microenvironment. [Cell Res] Full Article

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AgeX Therapeutics and Sernova to Collaborate to Engineer Universal Locally Immune Protected Cell Therapies for Type I Diabetes and Hemophilia A

AgeX Therapeutics, Inc. and Sernova Corp. announced a research collaboration where Sernova will utilize AgeX’s UniverCyte^™ gene technology to generate immune-protected universal therapeutic cells for use in combination with Sernova’s Cell Pouch^™ for the treatment of type I diabetes and hemophilia A. [AgeX Therapeutics, Inc.] Press Release

Regeneron and Intellia Therapeutics Expand Collaboration to Develop CRISPR/Cas9-Based Treatments

Regeneron Pharmaceuticals, Inc. and Intellia Therapeutics, Inc. announced an expansion of their existing collaboration to provide Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic targets and for the companies to jointly develop potential products for the treatment of hemophilia A and B. [Regeneron Pharmaceuticals, Inc.] Press Release

Longeveron Announces Japanese Approval of Clinical Trial for Treatment of Aging Frailty with Longeveron’s Stem Cells

Longeveron LLC announced that Japan’s Pharmaceutical and Medical Devices Agency approved a Clinical Trial Notification application, approving the initiation of a Phase II clinical trial evaluating the safety and efficacy of Longeveron’s mesenchymal stem cells for the treatment of Aging Frailty in Japanese patients. [Longeveron LLC (PR Newswire Association LLC)] Press Release

Vivet’s Second Gene Therapy Product, VTX-803 for PFIC3, Receives US and European Orphan Drug Designation

Vivet Therapeutics announced that both the FDA and the European Commission have granted Orphan Drug Designation for Vivet’s second gene therapy product, VTX-803, for the treatment of progressive familial intrahepatic cholestasis type 3. [Vivet Therapeutics] Press Release

Big Gene Therapy Names Line Up behind Experimental COVID-19 Vaccine

An early stage vaccine against Covid-19 based on the same basic technology used in gene therapy is gaining some support from some of that field’s biggest names. [STAT News] Editorial

What the Growing Rift between the US and WHO Means for COVID-19 and Global Health

Experts in health policy are contending with the real possibility that the United States will pull away from the World Health Organization (WHO), fracturing a relationship that began in the wake of the Second World War. [Nature News] Editorial

US Looks to Block Chinese Grad Students’ and Researchers’ Visas

Administration officials and lawmakers say the move is to shore up national security threats, but university professors argue such cancellations represent targeted discrimination. [The Scientist] Editorial

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NEW Postdoctoral Position – CRISPR/Cas Genetic Engineering (Aarhus University)

Postdoctoral Fellow – Cancer Adoptive Cell Immunotherapy (Wake Forest School of Medicine)

Scientist – Cell Therapy (Athersys, Inc.)

Postdoctoral Fellow – CRISPR Research (Boston Children’s Hospital)

Postdoctoral Position – RNA Editing and Immunotherapy (University of Chicago)

Principal Scientist – Human iPSC Gene Engineering (Empyrean Therapeutics Ltd)

Lab Director – Cancer Immunotherapy (Vrije Universiteit Brussels)

Postdoctoral Fellow – Cancer Drug Discovery (Uniformed Services University of Health Sciences)

Scientist – Cancer Immunotherapy (Vrije Universiteit Brussels)

Senior Research Scientist – Orthopedic Surgery/Regenerative Medicine (NYU Grossman School of Medicine)

Team Leaders – Stem Cell Biology (RIKEN Center for Biosystems Dynamics Research)

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