Cell Therapy News Volume 20.34 | Oct 7 2019

TOP STORY

Non-Immunogenic Utrophin Gene Therapy for the Treatment of Muscular Dystrophy Animal Models

Researchers showed that utrophin was a highly functional, non-immunogenic substitute for dystrophin, preventing the most deleterious histological and physiological aspects of muscular dystrophy in small and large animal models. [Nat Med] Abstract

(Ranked by impact factor of the journal)

Safety Profile of Autologous Macrophage Therapy for Liver Cirrhosis

Scientists conducted a first-in-human, Phase I dose-escalation trial of autologous macrophage therapy in nine adults with cirrhosis and a Model for End-Stage Liver Disease score of 10-16. [Nat Med] Abstract

Restoration of Visual Function by Transplantation of Optogenetically Engineered Photoreceptors

For an retinal pigment epithelium-independent treatment approach, the authors introduced a hyperpolarizing microbial opsin into photoreceptor precursors from newborn mice, and transplanted them into blind mice lacking the photoreceptor layer. [Nat Commun] Full Article

Development of a Forward-Oriented Therapeutic Lentiviral Vector for Hemoglobin Disorders

Investigators report a clinically relevant forward-oriented β-globin-expressing vector, which has sixfold higher vector titers and four to tenfold higher transduction efficiency for long-term hematopoietic repopulating cells in humanized mice and rhesus macaques. [Nat Commun] Full Article | Press Release

Regulatory T Cells Promote Corneal Endothelial Cell Survival following Transplantation via Interleukin-10

Using two well-established models of corneal transplantation, investigators showed that regulatory T cells (Tregs) derived from low-risk graft recipients had a superior capacity in protecting corneal endothelial cells against effector T cell-mediated and IFN-γ and TNF-α-induced cell death compared to Tregs derived from high-risk hosts. [Am J Transplant] Abstract

Electrical Stimulation of Pediatric Cardiac-Derived C-Kit⁺ Progenitor Cells Improves Retention and Cardiac Function in Right Ventricular Heart Failure

Scientists investigated the ability of electrical stimulation to enhance the retention and therapeutic function of pediatric cardiac-derived c-kit⁺ progenitor cells in an animal model of right ventricular heart failure. [Stem Cells] Abstract

Human Amniotic Mesenchymal Stem Cells Improve the Follicular Microenvironment to Recover Ovarian Function in Premature Ovarian Failure Mice

The oestrus cycle was recovered after human amniotic mesenchymal stem cell transplantation at 7 and 14-days. Oestrogen levels increased, while follicle-stimulating hormone levels decreased. [Stem Cell Res Ther] Full Article

The Oncolytic Adenovirus VCN-01 Promotes Anti-Tumor Effect in Primitive Neuroectodermal Tumor Models

In vivo, VCN-01 increased significantly the median survival of mice and led to long-term survivors in two orthotopic models of primitive neuroectodermal tumors. [Sci Rep] Full Article

In Vivo Bone Regeneration Induced by a Scaffold of Chitosan/Dicarboxylic Acid Seeded with Human Periodontal Ligament Cells

The authors evaluated a chitosan/dicarboxylic acid scaffold with and without seeded primary human periodontal ligament cells in its capacity to regenerate bone in calvarial defects of mice. [Int J Mol Sci] Abstract

Infant Cardiosphere-Derived Cells Exhibit Non-Durable Heart Protection in Dilated Cardiomyopathy Rats

After intramyocardial administration of infant cardiosphere-derived cells (CDCs) in the heart of dilated cardiomyopathy (DCM) rats, researchers found that infant CDCs remained in the heart of DCM rats for at least seven days, improved DCM-induced cardiac function impairment and protected the myocardium by elevating the left ventricular ejection fraction and fraction shortening. [Cytotechnology] Abstract

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Engineered Materials for In Vivo Delivery of Genome-Editing Machinery

The authors summarize delivery approaches and provide perspectives on the challenges and possible solutions, aiming to stimulate further development of engineered materials for in vivo delivery of genome-editing machinery. [Nat Rev Mater] Abstract

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Tmunity Announces Exclusive License and Research Collaboration with Children’s Hospital of Philadelphia to Advance GPC2 CAR-T Cell Therapy for Neuroblastoma

Tmunity Therapeutics, Inc. announced it has entered into an exclusive license and research collaboration agreement with Children’s Hospital of Philadelphia to advance a glypican 2 (GPC2) chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of neuroblastoma, a rare cancer that affects the developing nervous system. [Tmunity Therapeutics, Inc.] Press Release

ElevateBio Launches HighPassBio to Advance Novel Targeted T Cell Immunotherapies with Technology from Fred Hutchinson Cancer Research

ElevateBio announced it has established and launched HighPassBio, a company dedicated to advancing novel targeted T cell immunotherapies [ElevateBio] Press Release

Cynata Advances Toward Phase II Clinical Trial of Cymerus^™ MSCs in Patients with Osteoarthritis

Cynata Therapeutics Limited provided an update on start-up activities of a Phase II clinical trial to evaluate Cynata’s Cymerus^™ mesenchymal stem cells (MSCs) as a treatment for osteoarthritis, following the signing of an agreement with the University of Sydney. [Cynata Therapeutics Limited (GlobeNewswire, Inc.)] Press Release

Ziopharm Oncology Announces FDA Clearance of IND for Rapid Personalized Manufacture of CD19-Specific CAR-T

Ziopharm Oncology, Inc. announced that the FDA has cleared an investigational new drug application (IND) for a Phase I clinical trial to evaluate CD19-specific CAR-T, produced using a process termed rapid personalized manufacture, as an investigational treatment for patients with relapsed CD19⁺ leukemias and lymphomas. [Ziopharm Oncology, Inc.] Press Release

Cabaletta Bio Receives IND Clearance from FDA to Initiate First Clinical Trial of DSG3-CAART in Patients with Mucosal Pemphigus Vulgaris

Cabaletta Bio, Inc. announced that it has received clearance of its Investigational New Drug (IND) application from the FDA to initiate a first-in-human clinical trial of desmoglein 3 chimeric autoantibody receptor T cells (DSG3-CAART) in patients with mucosal pemphigus vulgaris to assess the safety and tolerability of DSG3-CAART in these patients. [Cabaletta Bio, Inc.] Press Release

BrainStorm Announces Notice of US Patent Allowance for NurOwn^® Cellular Therapeutic Technology Platform

BrainStorm Cell Therapeutics Inc. announced that the United States Patent and Trademark Office has issued a Notice of Allowance for BrainStorm’s new US Patent Application, number: 15/113,105, titled: ‘Method of Qualifying Cells’. [BrainStorm Cell Therapeutics Inc.] Press Release

Biologists Who Decoded How Cells Sense Oxygen Win Medicine Nobel

A trio of researchers has won the 2019 Nobel Prize in Physiology or Medicine for describing how cells sense and respond to changing oxygen levels by switching genes on and off — a discovery that has been key in understanding human diseases such as cancer and anemia. [Nature News] Editorial

Split Decisions: How Brexit Has Taken a Toll on Five Researchers

On 23 June 2016, 34 million U.K. citizens took to the polls to vote on a simple, fateful question: Should the United Kingdom remain a part of the European Union, or should it leave? Nearly 52%—a majority of 1.3 million—wanted out, and conservative politicians vowed to carry out the people’s wishes. [ScienceInsider] Editorial

There’s No Taboo, No Bad Ideas’: Anyone Can Help Shape European Union’s New €94 Billion Research Program

These encouraging words were spoken here last week at the European Research and Innovation Days, where policy bigwigs working with the European Commission invited the audience to help shape Europe’s next seven-year research funding program, Horizon Europe, set to begin in 2021. Their message: Anyone can help “cocreate” the program’s outlines. [ScienceInsider] Editorial

NIH

Prospective Grant of an Exclusive Patent License: Compositions, Devices and Processes for Production and Delivery of Cell Grafts of Manufactured Retinal Pigment Epithelium Cell(s) Alone, or in Combination with Photoreceptor Cells, and on a Biodegradable Support Scaffold Transplanted Subretinally for Intra-Ocular Ophthalmic Treatment of Conditions of Degeneration, Dysfunction or Terminal Injury of Retinal Pigment Epithelium and/or Photoreceptors in Humans (FR Doc. No:2019-21520) Notice

Prospective Grant of an Exclusive Patent License: Development and Commercialization of Cell Therapies for Cancer (FR Doc. No:2019-21519) Notice

NEW Gene Regulation: From Mechanisms to Disease
January 26-30, 2020
Keystone, Colorado, United States

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NEW Tenure-Track Faculty Position – Cancer Biology and Therapeutics (Geisel School of Medicine)

Research Technologist (STEMCELL Technologies Inc.)

Postdoctoral Fellow – Cancer Research (BC Cancer)

Assistant Professor – Regenerative Medicine (Washington University School of Medicine)

Research Assistant – Cancer Research (BC Cancer Agency)

Postdoctoral Fellow – Cancer Research (Baylor College of Medicine)

Postdoctoral Fellow – Molecular Genetic Basis of Behavioral Variation (University of California, San Francisco)

Postdoctoral Fellow – Cancer Therapy (Purdue University)

Postdoctoral Fellowship – Cancer Immunotherapy (McGill University)

Postdoctoral Position – Gene Therapy of Muscular Dystrophy (Ohio State University)

Research Scientist – Cellular Electrophysiology (Duke University)

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

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

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