Cell Therapy News Volume 21.17 | May 11 2020

TOP STORY

Systematically Optimized BCMA/CS1 Bispecific CAR-T Cells Robustly Control Heterogeneous Multiple Myeloma

Investigators report the rational design and optimization of bispecific CAR-T cells with robust activity against heterogeneous multiple myeloma that is resistant to conventional CAR-T cell therapy targeting B-cell maturation antigen. [Nat Commun] Full Article

(Ranked by impact factor of the journal)

CRISPR-Based Gene Editing Enables FOXP3 Gene Repair in IPEX Patient Cells

Scientists describe a CRISPR-based gene correction permitting regulated expression of forkhead box protein 3 (FOXP3) protein. They demonstrated that gene editing preserved hematopoietic stem and progenitor cell differentiation potential, and that edited regulatory and effector T cells maintained their in vitro phenotype and function. [Sci Adv] Full Article

CD28.OX40 Co-Stimulatory Combination Is Associated with Long In Vivo Persistence and High Activity of CAR.CD30 T-Cells

CAR.CD30 T-cells demonstrated anti-lymphoma activity in two in vivo xenograft immune-deficient mouse models of metastatic Hodgkin lymphoma and non-Hodgkin Lymphoma. [Haematologica] Abstract

Protection against SIV in Rhesus Macaques Using Albumin and CD4 Based Vector Mediated Gene Transfer

Antibody-like molecules were evaluated with potent SIV neutralizing properties that were delivered by a recombinant adeno-associated virus vector in the SIV infected rhesus macaque model. When injected intramuscularly into the host, the vector directed in vivo production of the transgenes with antibody-like binding properties that led to serum neutralizing activity against SIV. [Mol Ther Methods Clin Dev] Abstract | Full Article | Graphical Abstract

Cerebellar Astrocyte Transduction as Gene Therapy for Megalencephalic Leukoencephalopathy

The authors showed a preclinical gene therapy approach for megalencephalic leukoencephalopathy using the Mlc1 knock-out mouse. Myelin vacuolation was extremely reduced in treated mice at all ages and correlated with the amount of expressed MLC1 in Bergmann glia, indicating not only the preventive potential of this strategy but also its therapeutic capacity. [Neurotherapeutics] Abstract

Corneal Stromal Stem Cells Restore Transparency after N₂ Injury in Mice

Injection of stromal stem cells in nitrogen (N₂)-injured cornea resulted in improved corneal transparency associated with corneal stromal stem cell migration and growth in the recipient stroma, absence of inflammatory response, recipient corneal epithelial cell growth, decreased collagen type III stromal content, restored stromal ultrastructure, decreased stromal haze, decreased corneal rigidity, and improved vision. [Stem Cells Transl Med] Full Article

High Porous Electrospun Poly(ε-Caprolactone)/Gelatin/MgO Scaffolds Preseeded with Endometrial Stem Cells Promote Tissue Regeneration in Full-Thickness Skin Wounds: An In Vivo Study

Scientists confirmed that the wounds covered with oly(ε-caprolactone)-gelatin fibrous scaffolds containing 2 wt% MgO and preseeded with human endometrial stem cells had nearly 79% wound closure ability while sterile gauze showed 11% of wound size reduction. [J Biomed Mater Res B Appl Biomater] Abstract

Combination Immunotherapy of Oncolytic Virus Nanovesicles and PD-1 Blockade Effectively Enhances Therapeutic Effects and Boosts Antitumor Immune Response

Researchers investigated in vivo antitumor activity, systemic toxicity and mechanism of antitumor immune responses of Lipo-Cap-Ad5⁺anti-PD-1 blockade, in a murine B16F10 tumor xenograft model. [J Drug Target] Abstract

Oncolytic Newcastle Disease Virus Delivered by Mesenchymal Stem Cells-Engineered System Enhances the Therapeutic Effects Altering Tumor Microenvironment

Tumor therapy experiments indicated that oncolytic Newcastle disease virus delivered by mesenchymal stem cell-engineered system significantly reduced tumor growth, which was associated with the enhancement of E7-specific lymphocyte proliferation, CD8⁺ T cell cytolysis responses, and splenic IFN-γ, IL-4 and IL-12 responses compared with control groups. [Virol J] Full Article

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Towards Stem Cell Therapies for Skeletal Muscle Repair

The syncytial nature of skeletal muscle uniquely permits the engraftment of stem/progenitor cells to contribute to new myonuclei and restore the expression of genes mutated in myopathies. Historically however, the implementation of this approach has been significantly limited by the inability to expand undifferentiated muscle stem cells in culture whilst maintaining transplantation potential. [npj Regen Med] Full Article

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Genprex Enters into Exclusive Worldwide Patent and Technology License Agreement for Combination of its TUSC2 Gene Therapy with Immunotherapies

Genprex, Inc. announced that it has entered into a Patent and Technology License Agreement with The University of Texas MD Anderson Cancer Center in which MD Anderson granted to Genprex an exclusive worldwide license to a portfolio of 16 patent applications and related technology for the treatment of cancer using Genprex’s lead drug candidate and TUSC2 gene therapy, known as “Oncoprex” or “GEN-001,” in combination with immunotherapies. [Genprex, Inc.] Press Release

Dyno Therapeutics Announces Ocular Collaboration with Novartis to Develop Improved Gene Therapies with AAV Vectors Based on AI Technology

Dyno Therapeutics announced a collaboration with Novartis to develop improved AAV vectors for research, development, and commercialization of gene therapies for ocular diseases. [Dyno Therapeutics] Press Release

Lineage Cell Therapeutics to Apply Allogeneic Dendritic Cell Therapy Program to COVID-19 Vaccine Development

Lineage Cell Therapeutics, Inc. announced that it has applied for grant funding from the California Institute for Regenerative Medicine (CIRM) to support the use of VAC, Lineage’s allogeneic dendritic cell therapy, toward the development of a potential vaccine against SARS-CoV-2, the virus which causes COVID-19. [Lineage Cell Therapeutics, Inc. (Business Wire, Inc.)] Press Release

Triumvira Announces Grant of T Cell Antigen Coupler (TAC) Technology Patent by US Patent and Trademark Office

Triumvira Immunologics announced the issuance of key claims in US Patent No. 10640562 entitled “T cell-antigen coupler with various construct optimizations.” [Triumvira Immunologics (Business Wire, Inc.)] Press Release

Secretive Jasons to Offer Advice on How to Reopen Academic Labs Shut by Pandemic

A group of prominent academic scientists that has been advising the US government on security matters since the Cold War is conducting a quick-turnaround, pro bono study of a new threat to national security-the impact of COVID-19 on academic research. And this time it’s personal. [ScienceInsider] Editorial

How Swamped Preprint Servers Are Blocking Bad Coronavirus Research

Repositories are rapidly disseminating crucial pandemic science — and they’re screening more closely to guard against poor-quality work. [Nature News] Editorial

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

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

Lab Director – Cancer Immunotherapy (Vrije Universiteit Brussel)

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

Scientist – Cancer Immunotherapy (Vrije Universiteit Brussel)

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

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

Postdoctoral Research Associate – Induced Pluripotent Stem Cell Therapies (The University of Arizona College of Medicine)

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

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

Scientist – Cancer Immunotherapy (Lund University)

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