Cell Therapy News Volume 18.00 | Jan 9 2017

TOP STORY

Donor CD19 CAR T Cells Exert Potent Graft-versus-Lymphoma Activity with Diminished Graft-versus-Host Activity

To understand the mechanism whereby allogeneic CD19 chimeric antigen receptor (CAR) T cells may mediate anti-lymphoma activity without causing a significant increase in the incidence of graft-versus-host disease, scientists studied donor-derived CD19 CAR T cells in allo-hematopoietic stem cell transplantation and lymphoma models in mice. [Nat Med] Abstract

(Ranked by impact factor of the journal)

Optimized T-Cell Receptor-Mimic Chimeric Antigen Receptor T Cells Directed toward the Intracellular Wilms Tumor 1 Antigen

Researchers report a T-cell receptor-mimic chimeric antigen receptor, termed WT1-28z, that is reactive to a peptide portion of the intracellular onco-protein Wilms Tumor 1 (WT1), as it is expressed on the surface of the tumor cell in the context of HLA-A*02:01. T cells modified to express WT1-28z specifically targeted and lysed HLA-A*02:01⁺ WT1⁺ tumors and enhanced survival of mice engrafted with HLA-A*02:01⁺, WT1⁺ leukemia or ovarian tumors. [Leukemia] Abstract

TCR-Based Therapy for Multiple Myeloma and Other B-Cell Malignancies Targeting Intracellular Transcription Factor BOB1

T-cell receptor (TCR)-transduced T-cells efficiently lysed primary B-cell leukemia, mantel cell lymphoma and multiple myeloma in vitro. Investigators observed recognition and lysis of healthy BOB1-expressing B-cells. In addition, strong BOB1-specific proliferation was demonstrated for TCR-modified T-cells upon antigen encounter. Furthermore, clear in vivo antitumor reactivity was observed of BOB1-specific TCR-engineered T-cells in a xenograft mouse model of established multiple myeloma. [Blood] Abstract

Therapeutic Microparticles Functionalized with Biomimetic Cardiac Stem Cell Membranes and Secretome

The authors fabricated a synthetic cell-mimicking microparticle (CMMP) that recapitulates stem cell functions in tissue repair. CMMPs carried similar secreted proteins and membranes as genuine cardiac stem cells do. In a mouse model of myocardial infarction, injection of CMMPs lead to the preservation of viable myocardium and augmentation of cardiac functions similar to cardiac stem cell therapy. [Nat Commun] Full Article

piggyBac Mediates Efficient In Vivo CRISPR Library Screening for Tumorigenesis in Mice

With their piggyBac (PB)-CRISPR libraries, scientists conducted an in vivo genome-wide screen in mice and identified genes mediating liver tumorigenesis, including known and unknown tumor suppressor genes. Their results demonstrated that PB can be a simple and nonviral choice for efficient in vivo delivery of CRISPR libraries. [Proc Natl Acad Sci USA] Abstract

Near Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-HNAGLU Gene Delivery

Using global metabolic profiling by mass spectrometry targeting 361 metabolites, scientists detected significant decreases in 225 and increases in 6 metabolites, in serum samples from 7mo-old mucopolysaccharidosis (MPS) IIIB mice, compared to WT mice. The metabolic disturbances involve virtually all major pathways of amino acid, peptide, carbohydrate, lipid, nucleotide, energy, vitamin and co-factors and xenobiotics metabolism. [Mol Ther] Abstract

Radioiodinated Capsids Facilitate In Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors

Researchers presented two methods for radiolabeling adeno-associated virus, one of the most commonly used viral vectors for gene therapy trials, and demonstrated their potential usefulness in the development of surrogate markers for vector delivery during the first week after administration. [Sci Rep] Full Article

Human Heart Valve-Derived Scaffold Improves Cardiac Repair in a Murine Model of Myocardial Infarction

Investigators hypothesized that human heart valve-derived scaffold, as a clinically relevant novel biomaterial, may provide the proper microenvironment of native myocardial extracellular matrix for cardiac repair. [Sci Rep] Full Article

Cotransplantation of Bone Marrow-Derived Mesenchymal Stem Cells in Haploidentical Hematopoietic Stem Cell Transplantation in Patients with Severe Aplastic Anemia: An Interim Summary for a Multicenter Phase II Trial Results

In a multicenter Phase II trial, researchers developed an approach with co-transplantation of mesenchymal stem cells in patients undergoing haplo-hematopoietic stem cell transplantation. Forty-four patients with severe aplastic anemia were included. The conditioning regimen included busulfan, cyclophosphamide and thymoglobulin. [Bone Marrow Transplant] Abstract

Reduced-Intensity and Non-Myeloablative Allogeneic Stem Cell Transplantation from Alternative HLA-Mismatched Donors for Hodgkin Lymphoma: A Study by the French Society of Bone Marrow Transplantation and Cellular Therapy

The authors compared the outcome of patients with Hodgkin lymphoma undergoing a RIC or NMA allo-SCT from haploidentical, mismatched unrelated donor or cord blood. Ninety-eight patients were included. Median follow-up was 31 months for the whole cohort. [Bone Marrow Transplant] Abstract

Neurons Generated from Carcinoma Stem Cells Support Cancer Progression

Investigators showed that a fraction of cancer stem cells (CSCs) derived from patients with gastric carcinoma and colorectal carcinoma are capable of producing neurons that are involved in tumor neurogenesis and tumor growth. Cancer stem cell monoclone derived from a single cancer stem cell was able to generate neurons including sympathetic and parasympathetic neurons to take part in the nervous system in cancer tissues. [Signal Transduct Target Ther] Full Article

T Memory Stem Cells in Health and Disease

The authors describe emerging findings demonstrating that T memory stem cells, owing to their extreme longevity and robust potential for immune reconstitution, are central players in many physiological and pathological human processes. [Nat Med] Abstract

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

TissueGene to Highlight Invossa^™, the World’s First Cell-Mediated Gene Therapy for Degenerative Osteoarthritis

TissueGene, Inc. announced that CEO Woosok Lee will present. Mr. Lee will introduce TissueGene’s Invossa^™, the world’s first cell and gene therapy for degenerative arthritis. Invossa^™ is a first-in-class osteoarthritis drug designed to conveniently and effectively treat osteoarthritis of the knee through a single intra-articular injection. [Press release from TissueGene, Inc. (PR Newswire Association LLC.) discussing research to be presented at the 35^th Annual JP Morgan Healthcare, San Francisco] Press Release

MaxCyte, Inc. to Present Pre-Clinical Data for Targeted Gene-Correction in a Rare Disease

MaxCyte^® Inc. announced data to be presented summarizing in vitro and long-term preclinical toxicity and engraftment studies following targeted gene correction aimed at reversing mutations to wild-type sequence at clinically relevant levels in CD34⁺ hematopoietic stem cells obtained from individuals with X-CGD. [Press release from MaxCyte^® Inc. discussing research to be presented at the Keystone Symposia on Precision Genome Engineering, Breckenridge] Press Release

Johnson & Johnson Innovation, New York State and New York Genome Center Collaborate to Launch JLABS in New York City

Johnson & Johnson Innovation LLC, New York State and the New York Genome Center announced a collaboration to launch a new JLABS in New York City. Called JLABS @ NYC, the 30,000-square foot facility will be located at the New York Genome Center in SoHo and will open in 2018. The project is receiving $17 million in New York State funding. [Johnson & Johnson Innovation LLC] Press Release

Promethera Biosciences and LifeLiver Sign Strategic Alliance to Develop Liver Disease Therapeutics in South Korea

Promethera Biosciences SA and LifeLiver announced a strategic alliance to jointly develop Promethera’s suite of cell-based liver disease therapies in South Korea. Promethera and LifeLiver will collaborate on the clinical development of Promethera’s and LifeLiver’s product applications. Initially, LifeLiver will combine Promethera’s human mature hepatocytes liver cell suspension, Heparesc^®, with its proprietary bioartificial liver system for the ex vivo treatment of patients with severe liver diseases such as Acute Liver Failure. [Promethera Biosciences SA] Press Release

Renova Therapeutics Selects Worldwide Clinical Trials as Its Clinical Research Organization for Phase III Trial of RT-100 AC6 Gene Transfer

Renova^™ Therapeutics announced that it has selected Worldwide Clinical Trials as its Clinical Research Organization for a Phase III study of RT-100 AC6 gene transfer for the treatment of patients with reduced ejection fraction heart failure. [Renova^™ Therapeutics] Press Release

4D Molecular Therapeutics Enrolls First Patient in Natural History Study in Lead Clinical Program to Develop Gene Therapy Treatment for Choroideremia

4D Molecular Therapeutics announced the enrollment of the first participant in its Choroideremia Natural History Study. This study is an important step in developing a groundbreaking gene therapy product optimized for intravitreal administration to treat Choroideremia patients. [4D Molecular Therapeutics] Press Release

Asterias Announces Successful Commencement of Operations at New GMP Manufacturing Facility for AST-OPC1

Asterias Biotherapeutics, Inc. announced that the company has completed the validation and start-up of its internal Good Manufacturing Practices (GMP) manufacturing facility at its headquarters in Fremont, CA. GMP is a set of rigorous quality and safety requirements for manufacturing of pharmaceutical and biotechnology products for clinical trials and commercial sale. [Asterias Biotherapeutics, Inc.] Press Release

Allele to Develop iPSC-Derived Cell Therapies with Focus on Diabetes and Spinal Cord Injury

Allele Biotechnology & Pharmaceuticals announced that it has officially entered into the development of induced pluripotent stem cell (iPSC)-derived cell therapies after the development plan received strong support by the Scientific Advisory Board and was approved by the Board of Directors in December of 2016. [Allele Biotechnology & Pharmaceuticals] Press Release

RaNA Acquires Shire’s Industry Leading Messenger RNA Platform

RaNA Therapeutics announced the acquisition of the MRT platform, an mRNA therapy platform, developed by subsidiaries of Shire plc. In addition, Shire’s former MRT employees, the group focused on the development of this technology since 2008, have joined RaNA to continue to advance the platform with lead programs in cystic fibrosis and urea cycle disorders. [RaNA Therapeutics] Press Release

FASEB Launches Survey on Shared Research Resources

FASEB launched a survey of researchers’ experiences with shared research resources. [Federation of American Societies for Experimental Biology] Editorial

US Scientists Fear New Restrictions on Fetal-Tissue Research

House Republicans conclude that tissue from aborted fetuses is of limited value for research and seek to reduce funding. [Nature News] Editorial

FDA

Authorization of Emergency Use of an In Vitro Diagnostic Device for Detection of Zika Virus; Availability (FR Doc. No:2017-00084) Notice

Obstetrical and Gynecological Devices; Reclassification of Surgical Instrumentation for Use with Urogynecologic Surgical Mesh (FR Doc. No:2016-31862) Notice

Labeling of Red Blood Cell Units With Historical Antigen Typing Results; Draft Guidance for Industry; Availability (FR Doc. No:2016-31771) Notice

NEW ICSCRM 2017: 19^th International Conference on Stem Cells and Regenerative Medicine
February 16-17, 2017
London, United Kingdom

NEW Interventional Orthopedics Foundation 2017 Annual Conference
February 17-18, 2017
Broomfield, United States

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

NEW Senior Scientist – Engineered T Cells Used in Adoptive Cell Therapies (Immatics)

NEW Research Assistant – Decisions Regarding Cell Fate Regulation (University of Melbourne)

NEW Senior Scientist – Cell Isolation, Culture, and Genetic Engineering of Human B Cells for Expression of Therapeutic Gene Products (Immusoft Corporation)

NEW Tenure Track Faculty – Stem Cells and Regenerative Medicine (University of Notre Dame)

Staff Research Associate III – Translational Research to Clinical Cell Therapies (University of California Davis Health System)

Senior Research Scientist – Otolaryngology (Vanderbilt University Medical Center)

Laboratory Technician – Cellular Production Associate II-III (Fred Hutchinson Cancer Research Center)

Staff Research Associate III – Clinical Gene, Cellular, and Other Pharmaceutical Product Manufacturing (University of California Davis Health System)

Independent Research Group Leaders – Stem Cell Science (University of Cambridge)

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

Pioneer/Division Head – Human Organoids (Helmholtz Zentrum München)

Assistant Professor – Molecular Therapeutics of Cancer (Dartmouth College)

Research Fellow – Fat Tissue-Derived Stem Cells for Treating Metabolic Diseases (SBIC, A*STAR)

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

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