Cell Therapy News Volume 20.00 | Jan 7 2019

TOP STORY

A Pilot Trial of the Combination of Transgenic NY-ESO-1-Reactive Adoptive Cellular Therapy with Dendritic Cell Vaccination with or without Ipilimumab

Scientists studied the safety, feasibility, and antitumor efficacy of transgenic adoptive cell therapy with dendritic cell (DC) vaccination, with and without CTLA-4 blockade with ipilimumab. Freshly prepared autologous NY-ESO-1-specific T-cell receptor transgenic lymphocytes were adoptively transferred together with NY-ESO-1 peptide-pulsed DC vaccination in HLA-A2.1-positive subjects alone or with ipilimumab in patients with advanced sarcoma or melanoma. [Clin Cancer Res] Abstract

(Ranked by impact factor of the journal)

Human Induced Pluripotent Stem Cell-Derived MGE Cell Grafting after Status Epilepticus Attenuates Chronic Epilepsy and Comorbidities via Synaptic Integration

Investigators demonstrated that grafting of human induced pluripotent stem cell-derived medial ganglionic eminence (MGE)-like interneuron precursors into the hippocampus after status epilepticus greatly restrained spontaneous recurrent seizures and alleviated cognitive, memory, and mood dysfunction in the chronic phase of temporal lobe epilepsy. [Proc Natl Acad Sci USA] Full Article | Press Release

A Phase I, Open-Label Study of LCAR-B38M, a Chimeric Antigen Receptor T Cell Therapy Directed against B Cell Maturation Antigen, in Patients with Relapsed or Refractory Multiple Myeloma

The authors evaluated the safety and efficacy of LCAR-B38M, a dual epitope-binding CAR T cell therapy directed against two distinct B cell maturation antigen epitopes, in patients with relapsed/refractory multiple myeloma. [J Hematol Oncol] Full Article

TALEN-Mediated Gene-Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

Researchers developed TALENs targeting the homologous HBG promoters to de-repress fetal hemoglobin. Transfection of human CD34⁺ cells with TALEN mRNA resulted in indel generation in HBG1 and HBG2 including the 13bp hereditary persistence of fetal hemoglobin deletion. [Mol Ther Methods Clin Dev] Abstract | Graphical Abstract | Full Article

Overexpression of ERBB4 Rejuvenates Aged Mesenchymal Stem Cell and Enhances Angiogenesis via PI3K/AKT and MAPK/ERK Pathway

Scientists investigated whether Erb-B2 receptor tyrosine kinase 4 (ERBB4) rejuvenates aged mesenchymal stem cells (MSCs) and how ERBB4 enhances therapeutic efficacy of aged MSCs in treating myocardial infarction. [FASEB J] Abstract

The Impact of GPIbα on Platelet‐Targeted FVIII Gene Therapy in Hemophilia A Mice with Pre‐Existing Anti‐FVIII Immunity

Platelet‐FVIII expression was introduced by 2bF8 lentivirus transduction of hematopoietic stem cells from GPIbα^null mice or rhF8‐primed FVIII^null (F8^null) mice followed by transplantation into lethally irradiated rhF8‐primed F8^null recipients. [J Thromb Haemost] Abstract

Umbilical Cord‐Derived Mesenchymal Stromal Cells (MSCs) for Knee Osteoarthritis: Repeated MSC Dosing Is Superior to a Single MSC Dose and to Hyaluronic Acid in a Controlled Randomized Phase I/II Trial

Investigators assessed the safety and efficacy of the intra‐articular injection of single or repeated umbilical cord‐derived MSCs in knee osteoarthritis. [Stem Cells Transl Med] Full Article

Study of the Adoptive Immunotherapy on Rheumatoid Arthritis with Thymus-Derived Invariant Natural Killer T Cells

The therapeutic effect of adoptive infusion of specific thymus-derived invariant natural killer T cells in a mouse model of rheumatoid arthritis was observed, and the mechanism of cellular immunotherapy was preliminarily explored. [Int Immunopharmacol] Abstract | Graphical Abstract

Functional Gene Knockout of NRF2 Increases Chemosensitivity of Human Lung Cancer A549 Cells In Vitro and in a Xenograft Mouse Model

Scientists utilized CRISPR/Cas9 to disable the nuclear factor erythroid 2-related factor (NRF2) gene in lung cancer cells by disrupting the NRF2 nuclear export signal domain. [Mol Ther Oncolytics] Full Article | Editorial

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Nucleic Acids and Analogs for Bone Regeneration

The authors introduce delivery systems for nucleic acids and nucleic acid analogs and their applications in bone repair and regeneration. At the same time, the application of conventional scaffold materials for the delivery of nucleic acids and nucleic acid analogs is also discussed. [Bone Res] Full Article

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

CGT Catapult and pHion Therapeutics Announce Licencing Agreement for the RALA Technology

The Cell and Gene Therapy (CGT) Catapult and pHion Therapeutics announced an exclusive licence agreement for pHion’s proprietary RALA technology for ex vivo applications. The agreement builds upon the September 2017 collaboration between the organizations and aims to build alternative platforms for gene delivery and lower the costs of goods for cell therapy manufacturing. [The Cell and Gene Therapy Catapult] Press Release

Orgenesis Announces Collaboration with MangoGen Pharma for Advanced Gene Delivery Platform

Orgenesis Inc. announced a collaboration with MangoGen Pharma Inc. initially focused on the pre-clinical development of insulin producing cells using MangoGen’s advanced gene delivery platform. [Orgenesis Inc.] Press Release

Sarepta Therapeutics Enters into Long-Term Strategic Relationship with Aldevron for GMP-Grade Plasmid in Support of Gene Therapy Development and Commercial Manufacturing Strategy

Sarepta Therapeutics, Inc. and Aldevron announced that they have entered into a long-term strategic relationship for the supply of plasmid DNA to fulfill Sarepta’s needs for its gene therapy clinical trials and commercial supply. [Sarepta Therapeutics, Inc.] Press Release

Avalon GloboCare Establishes Joint Venture with Arbele Limited to Co-Develop Next-Generation Multi-Targeted CAR-T Cellular Immunotherapy

Avalon GloboCare Corp. announced that the company and its wholly owned subsidiary, Avactis Biosciences, have entered into a joint venture and exclusive license agreement with Arbele Limited to co-develop next generation, transposon-based Chimeric Antigen Receptor-T and CAR-Natural Killer cellular therapies. [Avalon GloboCare Corp.] Press Release

Berkeley Lights Announces Collaboration with University of Pennsylvania to Advance CAR-T Therapeutics

Berkeley Lights, Inc. announced that it has entered into a collaboration to explore multiple lines of inquiry aimed at advancing cellular therapies, including CAR-T therapeutics, with Megan Suhoski Davis, Director of the Product Development Laboratory within the Center for Cellular Immunotherapies in the University of Pennsylvania’s Perelman School of Medicine. [Berkeley Lights, Inc. (PR Newswire Association LLC.)] Press Release

Adaptive Biotechnologies to Enter Into Worldwide Collaboration and License Agreement with Genentech to Develop Personalized Cellular Therapies for the Treatment of Cancer

Adaptive Biotechnologies announced that it will enter into a worldwide collaboration and license agreement with Genentech to develop, manufacture and commercialize novel neoantigen directed T-cell therapies for the treatment of a broad range of cancers. [Adaptive Biotechnologies] Press Release

Takeda Announces Multiple Cell Therapy Collaborations to Advance the Company’s Novel Immuno-Oncology Portfolio

Takeda Pharmaceutical Company Limited announced new research collaborations in immuno-oncology. Through these collaborations, Takeda seeks to accelerate the discovery of next-generation cancer immunotherapies, including novel cell therapy approaches that may provide important opportunities for addressing the needs of patients with hard-to-treat cancers. [Takeda Pharmaceutical Company Limited] Press Release

Vertex and Arbor Biotechnologies Establish Collaboration to Discover Novel Proteins to Advance Discovery of Gene-Editing Therapies

Vertex Pharmaceuticals Incorporated and Arbor Biotechnologies announced that the two companies have entered into a strategic research collaboration focused on the discovery of novel proteins including DNA endonucleases to advance the development of new gene-editing therapies for cystic fibrosis and four other diseases to be selected later. [Vertex Pharmaceuticals Incorporated] Press Release

Novartis Announces Offer to Acquire CellforCure to Expand Manufacturing Capacity for Innovative Cell and Gene Therapies

Novartis announced an offer to acquire CellforCure from LFB. CellforCure is one of the first and largest contract development and manufacturing organizations producing cell and gene therapies in Europe. [Novartis AG] Press Release

Hemostemix Announces Two Additional Sites Enrolled in Phase II Clinical Trial

Hemostemix Inc. announced that it has received all approvals from two new medical centers to be clinical trial sites for the company’s Phase II clinical trial for critical limb ischemia. [Hemostemix Inc.] Press Release

Ultragenyx Announces Positive Topline Results from First Cohort of Phase I/II Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

Ultragenyx Pharmaceutical Inc. announced positive topline safety and efficacy data from the first, lowest dose cohort of the ongoing Phase I/II study of DTX401, an adeno-associated virus based gene therapy for the treatment of glycogen storage disease type Ia. [Ultragenyx Pharmaceutical Inc.] Press Release

Amicus Therapeutics Announces Phase I/II Study of Gene Therapy for CLN3 Batten Disease

Amicus Therapeutics announced the initiation of a Phase I/II clinical study to evaluate the safety and efficacy of a single intrathecal administration of adeno-associated virus serotype 9-CLN3 gene therapy in children with CLN3 Batten disease. [Amicus Therapeutics, Inc.] Press Release

StemCyte Receives Phase II Investigational New Drug (IND) Clearance from the U.S. Food and Drug Administration (FDA)

StemCyte announced that the FDA approved its Phase II IND application for allogeneic human leukocyte antigen-matched umbilical cord blood mononuclear stem cells for the treatment of spinal cord injury. [StemCyte (PR Newswire Association LLC.)] Press Release

Precigen Announces Clearance of IND to Initiate Phase I/Ib Study for First-in-Class PRGN-3006 UltraCAR-T™ Therapy in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML) and Higher Risk Myelodysplastic Syndrome (MDS)

Precigen, Inc. announced that the FDA has cleared the Investigational New Drug (IND) application for PRGN-3006, a first-in-class investigational therapy using Precigen’s UltraCAR-T™ platform. [Precigen, Inc. (PR Newswire Association LLC.)] Press Release

Cynata Completes Clinical Study Report for Phase I Trial of CYP-001 in GvHD

Cynata Therapeutics Limited announced that the clinical study report describing full details of the results from the Phase I clinical trial of CYP-001 for the treatment of steroid-resistant acute graft versus host disease (GvHD) has been completed. [Cynata Therapeutics Limited (GlobeNewswire, Inc.)] Press Release

MiNA Therapeutics Announces Enrolment of Patients in Expansion of Phase Ib Trial Evaluating MTL-CEBPA in Combination with Sorafenib

MiNA Therapeutics announced enrolment of the first patients treated with MTL-CEBPA in combination with Sorafenib in OUTREACH, the multi-centre Phase Ib clinical trial in patients with advanced liver cancer. [MiNA Therapeutics Limited] Press Release

Mustang Bio Receives Orphan Drug Designation for MB-102 (CD123 CAR T) for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm

Mustang Bio, Inc. announced that the FDA has granted Orphan Drug Designation to MB-102 for the treatment of blastic plasmacytoid dendritic cell neoplasm, a rare and incurable blood cancer with a median survival of less than 18 months and no standard of care. [Mustang Bio, Inc.] Press Release

Myonexus Therapeutics Receives Orphan Drug Designation for MYO-102, an Investigational Gene Therapy for Alpha-Sarcoglycanopathy (LGMD2D)

Myonexus Therapeutics announced that the FDA has granted Orphan Drug Designation for MYO-102, a novel gene therapy candidate for alpha-sarcoglycanopathy, also known as LGMD2D. [Myonexus Therapeutics] Press Release

CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease

CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced that the FDA has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease. [CRISPR Therapeutics] Press Release

BrainStorm Expands Manufacturing of NurOwn^® for Phase II Progressive MS Trial

BrainStorm Cell Therapeutics Inc. announced that it has expanded its autologous cell manufacturing capacity to produce NurOwn^® to support clinical trials in additional indications, including a Phase II progressive Multiple Sclerosis (MS) study, to begin in first quarter of 2019. [BrainStorm Cell Therapeutics Inc.] Press Release

FUJIFILM Cellular Dynamics to Establish New Production Facility for iPS Cells for Cell Therapy

FUJIFILM Cellular Dynamics, Inc. will establish a new cGMP-compliant production facility with an investment of about 21 million US dollars in order to enhance its production of iPS cells for cell therapy. [FUJIFILM Corporation] Press Release

Surge in Cell and Gene Therapy Trials Highlight the UK’s Leading Position as Developmental Hub in This Innovative Industry

New research published by the Cell and Gene Therapy Catapult indicated that the UK pipeline of advanced therapy medicinal products continues to go from strength to strength. Currently, there are 85 clinical trials and 875 preclinical research projects in cell and gene therapies ongoing, representing a 37% and 20% increase year-on-year respectively. [The Cell and Gene Therapy Catapult] Press Release

FDA Cracks Down on Purveyors of Stem Cell Treatments

The agency cites safety concerns with customers of Genetech who reported getting E. coli infections and sends letters to other stem cell companies reminding them to follow the rules. [The Scientist] Editorial

Donald Trump Finally Has a White House Science Adviser

Nearly two years after he took office, US President Donald Trump has a White House science adviser in place. The Senate confirmed meteorologist Kelvin Droegemeier for the job in a voice vote on 2 January. [Nature News] Editorial

Scientists Despair as US Government Shutdown Drags On

As the shutdown hits the two-week mark with no end in sight, its effects on science have begun to compound, leaving many government researchers weary, worried and demoralized. [Nature News] Editorial

UK Universities Warn ‘No Deal’ Brexit Will Hit Crucial Funding Streams

UK universities have warned the country’s government that leaving the European Union without a deal is “one of the biggest threats” the institutions have ever faced. [Nature News] Editorial

Most Americans Support Gene-Editing Embryos to Prevent Diseases, Poll Shows

Most Americans say it would be OK to use gene-editing technology to create babies protected against a variety of diseases — but a new poll shows they’d draw the line at changing DNA so children are born smarter, faster or taller. [STAT News] Editorial

NIH

Prospective Grant of an Exclusive Patent License: “Multifunctional RNA Nanoparticles and Methods of Uses” and “RNA/DNA Hybrid Nanoparticles Modified With Single Stranded RNA Toeholds and Uses Thereof” (FR Doc. No:2018-27671) Notice

NEW Novel Biomarkers, TMB and Immuno-Oncology Summit
January 31-February 1, 2019
London, United Kingdom

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

NEW Postdoctoral Position – Pluripotent Stem Cells and Rare Diseases (I-STEM)

NEW Postdoctoral Fellow – Genome and Metabolic Engineering (Columbia University Medical Center)

NEW Postdoctoral Research Associate – Gene Therapy (University of Massachusetts)

NEW Research Fellow – Stem Cell Biology (Brigham & Women’s Hospital – Harvard Medical School)

Postdoctoral Position – Technology-Driven Research in Biomedicine (CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences)

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

Faculty Position – Cancer Bioengineering (Ecole Polytechnique Fédérale de Lausanne)

Postdoctoral Position – Regenerative Medicine, Stem Cells and Bioengineering (Igenomix Foundation)

Postdoctoral Associate – Gene Therapy (University of Massachusetts)

Research Scientist – Cellular Manufacturing (California Institute for Biomedical Research)

Assistant Project Scientist – Hematology and Oncology (University of California, Davis)

Faculty Positions – Hematology and Oncology (University of California, Davis)

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