Cell Therapy News Volume 18.18 | May 29 2017

TOP STORY

U of T Research Helps Pave the Way for an Off-the-Shelf Supply of Cells for Immunotherapy

Researchers developed new technologies that clear some of the barriers to having a limitless source of cells to target cancer and other diseases. These breakthroughs could lead to new cell therapies for boosting patients’ immune systems against disease, and for cancer immunotherapy, in which immune cells can be engineered to attack tumors. [Press release from the University of Toronto discussing online prepublication in Nature Communications] Press Release | Full Article

(Ranked by impact factor of the journal)

Chimeric PD-1:28 Receptor Upgrades Low-Avidity T Cells and Restores Effector Function of Tumor-Infiltrating Lymphocytes for Adoptive Cell Therapy

In mouse tumor models of adoptive T cell therapy, PD-1:28 engineering enabled low-avidity T cells to proliferate stronger and prevented PD-L1 upregulation and Th2 polarization in the tumor milieu. [Cancer Res] Full Article

Depletion of Tumor-Associated Macrophages with a CSF-1R Kinase Inhibitor Enhances Antitumor Immunity and Survival Induced by DC Immunotherapy

The authors showed that M-CSFR-inhibition using the CSF-1R kinase inhibitor PLX3397 effectively reduced numbers of tumor-associated macrophages, circulating nonclassical monocytes, as well as amount of neo-angiogenesis and ascites in mesothelioma mouse models, but did not improve survival. [Cancer Immunol Res] Abstract

Transplantation of Human Villous Trophoblasts Preserves Cardiac Function in Mice with Acute Myocardial Infarction

Delivery of human villous trophoblasts (HVTs) to ischemic area of heart preserved cardiac function and reduced fibrosis in a mouse model of acute myocardial infarction (AMI). Histological analysis revealed that transplantation of HVTs promoted angiogenesis in AMI mouse hearts. [J Cell Mol Med] Full Article

In Vivo Endocrine Secretion of Prostacyclin following Expression of a Cox-1/PGIS Fusion Protein in the Salivary Glands of Rats via Non-Viral Gene Therapy

Scientists utilized ultrasound-assisted gene transfer, a non-viral gene transfer strategy that achieves robust gene transfer to the salivary gland. They found that cyclooxygenase-1 (Cox-1)-prostacyclin synthase (PGIS) expression in livers of mice using an adenoviral vector dramatically increased circulating prostacyclin relative to untreated rats or rats treated with PGIS alone. [Hum Gene Ther] Abstract

7,8,3′-Trihydroxyflavone Protects H/R-Induced Apoptosis and Induces In Vivo Growth of Human Embryonic Stem Cell-Derived Cardiomyocytes

Investigators examined whether treating human embryonic stem cell-derived cardiomyocytes (hES-CMs) with 7,8,3′-Trihydroxyflavone may improve hES-CM developments both in vitro and in vivo. [J Cell Biochem] Abstract

Antigen-Presenting Cell-Targeted Lentiviral Vectors Do Not Support the Development of Productive T-Cell Effector Responses: Implications for In Vivo Targeted Vaccine Delivery

Researchers report that broad tropism lentiviral vectors (LVs) are more immunogenic than antigen-presenting cell-targeted LVs because they transduce stromal cells, which has a role in activating antigen-specific T cells. [Gene Ther] Abstract

Novel Murine Xenograft Model for the Evaluation of Stem Cell Therapy for Profound Dysphagia

Muscle-derived stem cells (MDSCs) hold potential to restore dynamic function, and their application in the damaged tongue is appealing. The authors examined the safety and efficacy of human MDSC implantation into a novel mouse tongue model. [Laryngoscope] Abstract

Improvement of Local Cell Delivery Using Helix Transendocardial Delivery Catheter in a Porcine Heart

The helix transendocardial delivery system has been developed to enable percutaneous transendocardial biotherapeutic delivery. Scientists evaluated cell retention using this unique system compared with direct transepicardial injection and intracoronary infusion in an animal model. [Int Heart J] Abstract | Full Article | Press Release

Developing Gene and Cell Therapies for Rare Diseases: An Opportunity for Synergy between Academia and Industry

Cooperation between academia and industry is an opportunity to de-risk innovative approaches and ensure a faster and more economical development of therapies for diseases with high unmet medical needs and low-profit expectations. [Gene Ther] Abstract

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

bluebird bio to Present Updated Clinical Results from Novel Anti-BCMA CAR T Cell Therapy bb2121

bluebird bio, Inc. announced that updated interim data from its study of bb2121, the company’s anti-BCMA CAR T cell therapy will be presented. [Press release from bluebird bio, Inc. discussing research to be presented at American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

Cancer Experts from Sarah Cannon Research Institute Present Latest in Cancer Research

Sarah Cannon announced that it will present cancer research insights through more than 90 presentations. [Press release from Sarah Cannon Corporate discussing research presented at American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

Sangamo Therapeutics Presents Recent Developments from Research and Clinical Programs

Sangamo Therapeutics, Inc. highlighted data from research and clinical-stage programs presented. [Press release from Sangamo Therapeutics, Inc. discussing research presented at the 20^th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, D.C.] Press Release

Progenitor Life Sciences, Developer of Technologies for ‘Off-the-Shelf’ Cellular Immunotherapies, Announces Its Proprietary Stealth™ Immune-Compatibility Platform

Progenitor announces Stealth™ platform enabling sourcing of cells from a wide range of healthy donors, without donor-recipient HLA-matching. Stealth™-engineered cells also reduce immune-mediated adverse events caused by unintended activation of the recipient’s immune system following infusion of engineered cell therapies. [Progenitor Life Sciences (Business Wire, Inc.)] Press Release

ViaCyte Receives IND Allowance from FDA and Clearance from Health Canada to Commence International Clinical Trial of PEC-Direct Cell Therapy for High-Risk Type 1 Diabetes

ViaCyte, Inc. announced that the U.S. Food and Drug Administration (FDA) has allowed the company’s Investigational New Drug Application (IND) for the PEC-Direct™ product candidate, a novel islet cell replacement therapy in development as a potential cure for patients with type 1 diabetes who are at high risk for acute life-threatening complications. [ViaCyte, Inc.] Press Release

Lysogene Selects Brammer Bio to Produce GM1 Gangliosidosis Gene Therapy Product

Lysogene announced that it has entered into a strategic manufacturing agreement with Brammer Bio. Brammer Bio will produce LYS-GM101, an AAVrh10-based gene therapy, for clinical testing of the therapeutic candidate in patients with GM1 Gangliosidosis, a rare neuronopathic lysosomal storage disorder. [Lysogene] Press Release

Kite Receives U.S. Food and Drug Administration Priority Review for Axicabtagene Ciloleucel

Kite Pharma, Inc., announced that the U.S. Food and Drug Administration has accepted for priority review the Biologics License Application for axicabtagene ciloleucel. [Kite Pharma, Inc.] Press Release

PCI Biotech and RXi Pharmaceuticals Extend Research Collaboration to the Field of Immuno-Oncology

PCI Biotech and RXi Pharmaceuticals announced that they are extending their preclinical research collaboration initiated April 7, 2015. [PCI Biotech] Press Release

Longeveron to Receive Grant from the Maryland Stem Cell Research Fund

Longeveron announced receiving a $750,000 grant from the Maryland Stem Cell Research Fund to continue groundbreaking stem cell research. Longeveron will partner with the University of Maryland and Johns Hopkins University to conduct a clinical trial for hypoplastic left heart syndrome, a rare and often fatal condition in infants caused by an underdeveloped heart. [Longeveron] Press Release

Sangamo Receives Fast Track Designation from the FDA for SB-525 Investigational Hemophilia A Gene Therapy

Sangamo Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SB-525, the company’s clinical stage cDNA gene therapy candidate for hemophilia A, which is being developed as part of an exclusive, global collaboration and license agreement with Pfizer Inc. [Sangamo Therapeutics, Inc.] Press Release

bluebird bio Announces Collaboration with Duke University’s Robert J. Margolis, MD, Center for Health Policy on Value-Based Payment Framework for Gene Therapy

bluebird bio, Inc. announced that it will participate in a consortium led by Duke University’s Robert J. Margolis, MD, Center for Health Policy to develop a broadly-supported path for value-based payment reform models for gene therapies and other innovative treatments. [bluebird bio, Inc.] Press Release

Strategic Appointment of Dr. Joe Vitale and Expanded US Footprint as TrakCel Aims to Triple Client Base

TrakCel has made a further strategic appointment to support its growth in America. The company has appointed Dr. Joe Vitale as Director of Process Engineering. [TrakCel] Press Release

NIH Scales Back Plan to Curb Support for Big Labs after Hearing Concerns

Faced with a barrage of criticism, the National Institutes of Health (NIH) has scaled back a plan to cap its support for individual labs in order to free up funds for more scientists. The changes did not appease scientists who gave NIH a tongue-lashing at a meeting of NIH’s Council of Councils. [ScienceInsider] Editorial

House Science Panel Joins Trump in Questioning Research Overhead Payments

A hearing on how the U.S. government defrays the cost of doing federally funded research on college campuses might put most people to sleep. But when budgets are tight, the billions of dollars being spent each year on so-called overhead become an irresistible target for lawmakers. [ScienceInsider] Editorial

How NSF Cut 11% from Its Budget

Donald Trump planned to include an 11% cut to National Science Foundation’s (NSF)’s $7.4 billion budget in his first full spending request to Congress. The news—which was publicly unveiled as part of the president’s $4.1 trillion budget for 2018—sent Jim Olds, who leads the biology directorate at the NSF, and the heads of NSF’s other six research and education programs scrambling to erase big chunks of their portfolios without sacrificing NSF’s ability to fund the best new ideas. [ScienceInsider] Editorial

FDA

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting (FR Doc. No:2017-10976) Notice

NIH

Prospective Grant of Exclusive Patent License: Chimeric L1/L2 Protein and Virus-Like Particles Based Human Papillomavirus Vaccines (FR Doc. No:2017-10153) Notice

NEW Gordon Research Conferences: Collagen
July 16-21, 2017
New London, United States

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

NEW Professor – Stem Cell Biology (University College London)

NEW Postdoctoral Researcher – Cellular Therapy for Degenerative Disc Disease (NUI Galway)

Specialist – Cell Procurement (Celgene Corporation)

Head – CAR-T Operations and Technology (Celgene Corporation)

Director – Quality Control (KBI Biopharma)

Scientist – Analytical Development (KBI Biopharma)

PhD Fellow – Experimental Genetic Cardiology (Oslo University Hospital)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center)

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.