Cell Therapy News Volume 21.13 | Apr 13 2020

TOP STORY

KTE-X19 CAR T-Cell Therapy in Relapsed or Refractory Mantle-Cell Lymphoma

In a multicenter, Phase II trial, scientists evaluated KTE-X19 in patients with relapsed or refractory mantle-cell lymphoma. Patients underwent leukapheresis and optional bridging therapy, followed by conditioning chemotherapy and a single infusion of KTE-X19 at a dose of 2×10⁶ CAR T cells per kilogram of body weight. [N Engl J Med] Abstract

(Ranked by impact factor of the journal)

Glia-to-Neuron Conversion by CRISPR-CasRx Alleviates Symptoms of Neurological Disease in Mice

Researchers report that downregulation of a single RNA-binding protein, polypyrimidine tract-binding protein 1, using in vivo viral delivery of a recently developed RNA-targeting CRISPR system CasRx, resulted in the conversion of Müller glia into retinal ganglion cells (RGCs) with a high efficiency, leading to the alleviation of disease symptoms associated with RGC loss. [Cell] Abstract | Graphical Abstract

An Off-the-Shelf Artificial Cardiac Patch Improves Cardiac Repair after Myocardial Infarction in Rats and Pigs

In a rat model of acute myocardial infarction, transplantation of the acellular artificial cardiac patch supported cardiac recovery by reducing scarring, promoting angiomyogenesis, and boosting cardiac function. [Sci Transl Med] Abstract

Regeneration of Pulpo-Dentinal-Like Complex by a Group of Unique Multipotent CD24a⁺ Stem Cells

Multipotent dental pulp regenerative stem cell exhibited enhanced osteogenic/odontogenic differentiation capabilities and could form regenerative dentin and neurovascular-like structures that mimicked native teeth in vivo. [Sci Adv] Full Article

Autologous Transplantation of Spermatogonial Stem Cells Restores Fertility in Congenitally Infertile Mice

Investigators analyzed the role of Cldn11, a major component of the blood-testis barrier, in spermatogenesis using spermatogonial transplantation. [Proc Natl Acad Sci USA] Abstract

CD39 Produced from Human Gingiva-Derived MSC Regulates the Balance of Osteoclasts and Osteoblasts through Wnt/β-Catenin Pathway in Osteoporosis

Using an ovariectomized mouse model, the authors demonstrated that adoptive transfer of gingiva-derived mesenchymal stem cells regulated the balance of osteoclasts and osteoblasts, eventually contributing to dynamic bone formation. [Mol Ther] Abstract | Full Article | Graphical Abstract

Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I (MPS I)

AAV8G9-alpha-L-iduronidase injection in the MPS I canine eye with early corneal disease prevented the development of advanced corneal changes while restoring clarity. [Mol Ther] Abstract | Graphical Abstract

Intra-CSF AAV9 and AAVrh10 Administration in Non-Human Primates: Promising Routes and Vectors for What Neurological Diseases?

Scientists demonstrated that AAV9 lumbar intrathecal delivery using a catheter achieved consistent transgene expression in the motor neurons of the spinal cord and in the neurons/glial cells of several brain regions, whereas AAV9 intracerebroventricular delivery led to a consistent transgene expression in the brain. [Mol Ther Methods Clin Dev] Abstract | Full Article

Autologous Hematopoietic Cell Transplantation for the Treatment of Relapsed/Refractory Pediatric, Adolescent, and Young Adult Hodgkin Lymphoma: A Single Institutional Experience

Investigators describe a single institutional experience utilizing autologous hematopoietic cell transplantation in 74 patients treated from 1988–2015. Their results demonstrated significantly improved outcomes over time. [Bone Marrow Transplant] Abstract

Injectable Therapeutic Organoids Using Sacrificial Hydrogels

The authors introduced a method of using hydrogels as sacrificial scaffolds, which allowed cells to form self-organized clusters followed by gentle release, resulting in highly reproducible multicellular structures on a large scale. They demonstrated this strategy for endothelial cells and mesenchymal stem cells to self-organize into blood-vessel units, which were injected into mice, and rapidly formed perfusing vasculature. [iScience] Abstract | Full Article | Graphical Abstract

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Mechanisms of Resistance to NK Cell Immunotherapy

A first contributing factor that modulates the efficacy of the natural killer (NK) cell therapy is the genetic profile of the individual, which regulates all aspects of NK cell biology. Additionally, the resistance of cancer cells to apoptosis and the immunoediting of cancer cells, a process that decreases their immunogenicity and promotes immunosuppression, are major determinants of the resistance to NK cell therapy. [Cancers] Full Article

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Fate Therapeutics Announces Worldwide Collaboration with Janssen for Novel iPSC-Derived Cell-Based Cancer Immunotherapies

Fate Therapeutics, Inc. announced a global collaboration and option agreement with Janssen Biotech, Inc. (Janssen). Under the multi-year collaboration agreement, Janssen will contribute proprietary antigen binding domains for up to four tumor-associated antigen targets. The company will apply its induced pluripotent stem cell (iPSC) product platform to research and preclinically develop new iPSC-derived CAR NK and CAR T-cell product candidates. [Fate Therapeutics, Inc.] Press Release

CYTOVIA Therapeutics and MACROMOLTEK to Develop Dual-Acting Natural Killer Immunotherapy against SARS CoV2 (COVID-19)

Cytovia Therapeutics announced that it is expanding its programs to help urgently address the current SAR CoV2 (COVID-19) crisis. [Cytovia Therapeutics (GlobeNewswire, Inc.)] Press Release

Hope Biosciences Receives Second FDA Clearance for a Phase II Clinical Trial for COVID-19

Hope Biosciences announced that FDA has approved a Phase II clinical trial evaluating the safety and efficacy of Hope Biosciences’ allogeneic, adipose-derived mesenchymal stem cells to provide immune support against COVID-19. [Hope Biosciences] Press Release

Precision BioSciences Announces Dosing of First Patient in Phase I/IIa Clinical Trial of PBCAR20A for Relapsed/Refractory NHL, CLL, and SLL

Precision BioSciences, Inc. announced the initiation of patient dosing in a Phase I/IIa clinical trial of its second off-the-shelf CAR T cell therapy candidate, PBCAR20A. [Precision BioSciences, Inc.] Press Release

FDA Authorizes Athersys to Initiate a Pivotal Clinical Trial Evaluating MultiStem^® Cell Therapy in Patients with COVID-19 Induced Acute Respiratory Distress Syndrome

Athersys, Inc. announced that the FDA has authorized the company to initiate a Phase II/III pivotal study to assess the safety and efficacy of MultiStem^® therapy in subjects with moderate to severe acute respiratory distress syndrome induced by the novel coronavirus disease. [Athersys, Inc.] Press Release

FibroGenesis Files Expanded Patent Coverage for Its Fibroblast Cell Therapy to Treat Coronavirus (COVID-19) ARDS

FibroGenesis announced the filing of United States Provisional Patent Number 63/002,134 titled, “Peptides and Adjuvants for Augmentation of Fibroblast Therapy for Coronavirus.” [FibroGenesis] Press Release

President of Europe’s Premier Science Funder Resigns amid Criticism That He Neglected Post

The president of Europe’s most prestigious funding agency has resigned suddenly after only three months in the post – prompting fiery accusations from the organization’s governing body that he largely neglected his responsibilities. [Nature News] Editorial

Early-Career Scientists at Critical Career Junctures Brace for Impact of COVID-19

For some scientists, the shutdowns have made it more challenging for them to complete their work. But for others, they come at a critical juncture in the scientist’s career, threatening to derail or delay a major event along their professional path—a small slice of the human suffering and inconvenience that COVID-19 has caused. [ScienceInsider] Editorial

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NEW Postdoctoral Associate – Diabetes (University of Miami Health System)

Associate/Senior Editor – Nature Biotechnology (Nature Research)

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)

Scientist/Senior Scientist – Cell Therapy (Zelluna Immunotherapy AS)

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)

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

Sr Associate Scientist – Autoimmunity and Tumor Immunotherapy (Amgen)

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