Cell Therapy News Volume 20.27 | Aug 12 2019

TOP STORY

Epicardial Cells Derived from Human Embryonic Stem Cells Augment Cardiomyocyte-Driven Heart Regeneration

The authors tested the ability of human embryonic stem cell (hESC)-derived epicardium to augment the structure and function of engineered heart tissue in vitro and to improve efficacy of hESC-cardiomyocyte grafts in infarcted athymic rat hearts. [Nat Biotechnol] Abstract

(Ranked by impact factor of the journal)

Therapeutically Relevant Engraftment of a CRISPR-Cas9-Edited HSC-Enriched Population with HbF Reactivation in Nonhuman Primates

Scientists evaluated the therapeutic potential of hematopoietic stem and progenitor cells edited with the CRISPR-Cas9 nuclease platform to recapitulate naturally occurring mutations identified in individuals who expressed increased amounts of fetal hemoglobin (HbF), a condition known as hereditary persistence of HbF. [Sci Transl Med] Abstract

Multispecific Anti-HIV DuoCAR-T Cells Display Broad In Vitro Antiviral Activity and Potent In Vivo Elimination of HIV-Infected Cells in a Humanized Mouse Model

Multispecific anti-HIV duoCAR-T cells demonstrated long-term control of HIV infection in vivo and prevented the loss of CD4⁺ T cells during HIV infection using a humanized NSG mouse model of intrasplenic HIV infection. [Sci Transl Med] Abstract

Phase I Study of Lentiviral-Transduced Chimeric Antigen Receptor Modified T Cells Recognizing Mesothelin in Advanced Solid Cancers

This Phase I study investigated the safety and activity of lentiviral-transduced CAR-modified autologous T cells redirected against mesothelin in patients with malignant pleural mesothelioma, ovarian carcinoma, and pancreatic ductal adenocarcinoma. [Mol Ther] Abstract

AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-Human Primates Compared to Mice

The authors compared AAV-PHP.B transduction efficiency in wildtype C57BL/6J mice using four clinically-applicable delivery strategies including two intravascular and two intra-CSF routes. [Mol Ther] Abstract

Long-Term Safety, Immunologic Response, and Imaging Outcomes following Neural Stem Cell Transplantation for Pelizaeus-Merzbacher Disease

Four boys with Pelizaeus-Merzbacher disease, an X-linked leukodystrophy, underwent transplantation with human allogeneic central nervous system stem cells. Subsequently, all subjects were followed for an additional four years in this separate follow-up study to evaluate safety, neurologic function, magnetic resonance imaging data, and immunologic response. [Stem Cell Reports] Full Article

Optimized CRISPR/Cas9-Mediated In Vivo Genome Engineering Applicable to Monitoring Dynamics of Endogenous Proteins in the Mouse Neural Tissues

By combining the CRISPR/Cas9 genome editing technology with an electroporation technique, investigators succeeded in creating knock-in alleles, from which GFP-tagged endogenous proteins are produced, in neurons and glial cells in vivo in the developing mouse retina and brain. [Sci Rep] Full Article

In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

A humanized mouse model of β-thalassemia was used, in which heterozygous animals were anemic with splenomegaly and extramedullary hematopoiesis. Intrahepatic in utero injections of a β globin-expressing lentiviral vector (GLOBE), were performed in fetuses at E13.5 of gestation. [Sci Rep] Full Article

Cancer Immunotherapy Using the Fusion Gene of Sendai Virus

Researchers attempted to deliver fusion (F) gene into tumor tissue in mice by electroporation and demonstrated that F gene therapy retarded tumor growth, increased CD4⁺ and CD8⁺ T-cell infiltration into tumors and induced tumor-specific IFN-γ T-cell response. [Cancer Gene Ther] Abstract

Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News.

Enhancing CAR T-Cell Therapy through Cellular Imaging and Radiotherapy

Investigators discuss the short-term strategies to improve CAR T-cell therapy responses, particularly for solid tumors, by combining CAR T-cell therapy with radiotherapy through the use of careful monitoring and non-invasive imaging. [Lancet Oncol] Abstract

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

Bayer Acquires BlueRock Therapeutics to Build Leading Position in Cell Therapy

Bayer AG and BlueRock Therapeutics announced an agreement under which Bayer will fully acquire BlueRock Therapeutics, a privately held US-headquartered biotechnology company focused on developing engineered cell therapies in the fields of neurology, cardiology and immunology, using a proprietary induced pluripotent stem cell platform. [Bayer AG] Press Release

Selecta Biosciences Combines ImmTOR™ Platform with AskBio’s Industry Leading Gene Therapy Platform in Strategic Partnership

Selecta Biosciences, Inc. and Asklepios BioPharmaceutical, Inc. announced a strategic partnership to jointly develop, manufacture and commercialize targeted therapeutics for life-changing, next-generation adeno-associated virus gene therapies in areas of high medical need. [Asklepios BioPharmaceutical, Inc.] Press Release

Eureka Therapeutics Announces Initiation of Phase I/II Clinical Trial of ET140202 ARTEMIS™ T-Cell Therapy in Liver Cancer

Eureka Therapeutics, Inc. announced initiation of Phase I/II clinical trial of ET140202 ARTEMIS™ T-Cell therapy in liver cancer at City of Hope. [Eureka Therapeutics, Inc.] Press Release

Precigen Announces First Patient Dosed in Phase I Study of PRGN-3005 UltraCAR-T™ in Patients with Advanced, Recurrent Platinum Resistant Ovarian, Fallopian Tube or Primary Peritoneal Cancer

Precigen, Inc. announced that the first patient has been dosed with Precigen’s PRGN-3005, a first-in-class investigational therapy using Precigen’s UltraCAR-T™ therapeutic platform. [Precigen, Inc. (PR Newswire Association LLC.)] Press Release

Evox Therapeutics Granted Key Exosome Patents Covering RNA Therapeutics and Targeted Drug Delivery

Evox Therapeutics Ltd announced that the company has been granted three key patents by the United States Patent and Trademark Office and the European Patent Office. These three new grants are from one of several foundational patent families held by Evox and further reinforce the Company’s leading position within exosome-mediated RNA therapeutics and targeted exosome drug delivery. [Evox Therapeutics Ltd (PR Newswire Association LLC.)] Press Release

ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP)

ProQR Therapeutics N.V. announced that the FDA has cleared the Investigational New Drug (IND) application for QR-1123. ProQR plans to start enrolling patients in a Phase I/II trial for QR-1123 in 2019. [ProQR Therapeutics N.V.] Press Release

City of Hope Scientist Receives $9.28 Million for CAR T Cell Immunotherapy Clinical Trial for Patients with HER2-Positive Breast Cancer That has Spread to the Brain

Saul Priceman, Ph.D., City of Hope assistant professor in the Department of Hematology & Hematopoietic Cell Transplantation, and his research team have received a $9.28 million award from the California Institute for Regenerative Medicine to support a CAR T cell Phase I clinical trial for the treatment of women with HER2-positive breast cancer that has spread to the brain. [City of Hope] Press Release

Senators Blast Novartis over Data Manipulation before Approval of Zolgensma

A group of senators, including presidential hopefuls Sens. Elizabeth Warren and Bernie Sanders, blasted Novartis for failing to promptly disclose that falsified data were used to win approval for the company’s $2.1 million gene therapy, Zolgensma. [STAT News] Editorial

With Its CRISPR Revolution, China Becomes a World Leader in Genome Editing

In 2012, the year researchers transformed a bacterial immune system into the fast and versatile tool for genome engineering, scientific publications mentioning CRISPR totaled 127. Since then there have been more than 14,000. Although the United States has had the most CRISPR publications—and continues to have the most cited papers—China is now a close second and is pouring money into CRISPR’s uses. [ScienceInsider] Editorial

Survey of Stem Cell Clinics Reveals Cause for Concern

Two coauthors of a new study discuss their findings about nearly 170 facilities in the southwestern US selling unproven therapies—and what should be done about them. [The Scientist] Editorial

The World Health Organization Says No More Gene-Edited Babies

Eight months after a rogue Chinese scientist revealed he had secretly created the world’s first gene-edited children, the World Health Organization is asking countries to put a stop to any experiments that would lead to the births of more gene-edited humans. On Friday, the WHO’s director-general put out a statement urging “that regulatory authorities in all countries should not allow any further work in this area until its implications have been properly considered.” [Wired] Editorial

FDA

Agency Information Collection Activities; Proposed Collection; Comment Request; Biological Products: Reporting of Biological Product Deviations and Human Cells, Tissues, and Cellular and Tissue-Based Deviations in Manufacturing (FR Doc. No:2019-16243) Notice

NEW American Society of Human Genetics Annual Meeting 2019
October 10-14, 2019
Houston, Texas, United States

Visit our events page to see a complete list of events in the community.

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

NEW Postdoctoral Scholar – Gene and Cell Therapy (Stanford University)

Scientific Communications Coordinator (STEMCELL Technologies Inc.)

Postdoctoral Scientist – HSV Vectors as a Therapy for Nervous System Tumors (Nationwide Children’s Hospital)

Postdoctoral Position – Translational Gene Therapy (Fred Hutchinson Cancer Research Center)

Senior Scientist – Immuno-Oncology of Hematologic Malignancies (University of Kansas School of Medicine)

Research Scientist – Cellular Electrophysiology (Duke University)

Postdoctoral Scientist – Computational Biologist (Cedars-Sinai Medical Center)

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

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

Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University)

Assistant/Associate or Professor – Cancer Immunotherapy/Tumor Immunology (University of California, Irvine)

Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.

Have we missed an important article or publication in Cell Therapy News? Click here to submit!

Comments or suggestions? Submit your feedback here.