Cell Therapy News Volume 19.21 | Jun 18 2018

TOP STORY

Antitumor Activity Associated with Prolonged Persistence of Adoptively Transferred NY-ESO-1c259T Cells in Synovial Sarcoma

Investigators evaluated safety and activity of autologous T cells expressing NY-ESO-1c259, an affinity-enhanced T cell receptor recognizing an HLA-A2-restricted NY-ESO-1/LAGE-1a-derived peptide, in patients with metastatic synovial sarcoma (NY-ESO-1c259T cells). [Cancer Discov] Abstract | Press Release | Full Article

(Ranked by impact factor of the journal)

Association between Autologous Stem Cell Transplant and Survival among Californians with Multiple Myeloma

The authors utilized population-based data to identify 13 494 newly diagnosed patients younger than age 80 years between 1998 and 2012. Patient characteristics of autologous hematopoietic stem cell transplant (aHSCT) and non-aHSCT groups were balanced using inverse probability weighting of a propensity score predicting aHSCT use. [J Natl Cancer Inst] Abstract

Immune-Evasive Gene Switch Enables Regulated Delivery of Chondroitinase after Spinal Cord Injury

Scientists applied a novel immune-evasive dual vector system, in which the chondroitinase gene is under a doxycycline inducible regulatory switch, utilizing a chimeric transactivator designed to evade T cell recognition. [Brain] Full Article | Press Release

RNAi-Based GluN3A Silencing Prevents and Reverses Disease Phenotypes Induced by Mutant Huntingtin

Researchers tested the therapeutic potential of silencing GluN3A expression in YAC128 mice, a well-established Huntington’s disease model. Recombinant adeno-associated viruses encoding a short-hairpin RNA against GluN3A were generated, and the ability of different serotypes to transduce medium-sized spiny neurons was compared. [Mol Ther] Abstract

Autologous CD19-Targeted CAR T Cells in Patients with Residual CLL following Initial Purine Analog-Based Therapy

Scientists report the use of CD19-targeted CAR T cells incorporating the intracellular signaling domain of CD28 as a consolidative therapy in eight patients with residual chronic lymphocytic leukemia (CLL) following first-line chemoimmunotherapy with pentostatin, cyclophosphamide, and rituximab. [Mol Ther] Abstract | Graphical Abstract

Co-Stimulation of LPAR₁ and S1PR_1/3 Increases the Transplantation Efficacy of Human Mesenchymal Stem Cells in Drug-Induced and Alcoholic Liver Diseases

The authors delineated the mechanistic involvement of lysophosphatidic acid receptors (LPARs) and sphingosine-1-phosphate receptors (S1PRs) in the regulation of anti-stress and transplantation efficacy of stem cells. [Stem Cell Res Ther] Full Article

Epithelial Disruption: A New Paradigm Enabling Human Airway Stem Cell Transplantation

Four μL of 2% polidocanol (PDOC) in phosphate-buffered saline was administered to the nasal airway of mice to disrupt the epithelium. At 2 or 24 hours after PDOC treatment, two types of reporter gene-expressing cells were transplanted into the animals: luciferase-transduced human airway basal cells or luciferase-transduced human amnion epithelial cells. [Stem Cell Res Ther] Full Article

Metformin Preconditioning of Human Induced Pluripotent Stem Cell-Derived Neural Stem Cells Promotes Their Engraftment and Improves Post-Stroke Regeneration and Recovery

Metformin-preconditioned hiPSC-derived neural stem cells showed increased engraftment one-week post-transplant in a rat endothelin-1 focal ischemic stroke model. [Stem Cells Dev] Abstract

Porous Lithium-Doped Hydroxyapatite Scaffold Seeded with Hypoxia-Preconditioned Bone-Marrow Mesenchymal Stem Cells for Bone-Tissue Regeneration

Lithium-doped hydroxyapatite/bone-marrow mesenchymal stem cells enhanced new bone formation, reducing the GSK-3β and increasing the β-catenin, but the angiogenic effect was not modified significantly. [Biomed Mater] Abstract

Programming CAR-T Cells to Kill Cancer

The authors survey major advances in the engineering of next-generation CAR-T therapies for hematologic cancers and solid cancers, with particular emphasis on strategies for the control of CAR specificity and activity and on approaches for improving CAR-T-cell persistence and overcoming immunosuppression. [Nat Biomed Eng] Full Article

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

bluebird bio Announces New Interim Data from Phase I (HGB-206) Study of LentiGlobin™ Gene Therapy in Patients with Severe Sickle Cell Disease

bluebird bio, Inc. announced new interim data from the ongoing HGB-206 Phase I multicenter clinical study of LentiGlobin investigational gene therapy in patients with severe sickle cell disease will be presented in an oral presentation. [Press release from bluebird bio, Inc. discussing research presented at the 23^rd Congress of the European Hematology Association (EHA), Stockholm] Press Release

Sarepta Therapeutics Enters into Long-Term Strategic Manufacturing Partnership with Brammer Bio to Support Gene Therapy Development and Commercial Supply

Sarepta Therapeutics, Inc. announced that it has entered into a long-term strategic manufacturing partnership with Brammer Bio, which will provide Sarepta access to clinical and commercial manufacturing capacity for its micro-dystrophin Duchenne muscular dystrophy gene therapy program and a manufacturing platform for future gene therapy programs, such as Limb girdle muscular dystrophy. [Sarepta Therapeutics, Inc.] Press Release

Herantis Pharma Announces Initiation of Phase II Study for Lymfactin^® Gene Therapy in Secondary Lymphedema

Herantis Pharma Plc announced the initiation of the Phase II clinical study adenoviral gene therapy for the treatment of secondary lymphedema (LE), which will evaluate the efficacy of Lymfactin^® gene therapy in the treatment of LE. [Herantis Pharma Plc] Press Release

REGENXBIO Receives FDA Fast Track Designation for RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I

REGENXBIO Inc. announced that the FDA has granted Fast Track designation for RGX-111. RGX-111 is a novel, one-time investigational treatment for mucopolysaccharidosis type I, that is designed to deliver the human iduronidase gene directly to the central nervous system using the NAV AAV9 vector. [REGENXBIO Inc.] Press Release

Ziopharm Oncology Reports on Status of Investigational New Drug Application for Phase I Trial to Evaluate CD19-Targeted CAR T Therapy

Ziopharm Oncology, Inc. announced the FDA placed on clinical hold a Phase I trial to evaluate CD19-specific CAR-T therapies manufactured under point-of-care and requested additional information in support of the investigational new drug application for the trial. [Ziopharm Oncology, Inc.] Press Release

Chiesi and Holostem Announce FDA Orphan Drug Designation for GPLSCD01 for Treatment of Limbal Stem Cell Deficiency (LSCD)

Chiesi USA and Holostem Terapie Avanzate S.r.l., announced that the FDA has granted orphan drug designation to an investigational product, GPLSCD01, ex vivo expanded autologous human corneal epithelial cells containing stem cells, for the treatment of LSCD. [Chiesi USA (GlobeNewswire, Inc.)] Press Release

Solid Biosciences Announces FDA Removes Clinical Hold on SGT-001

Solid Biosciences Inc. announced that the FDA has lifted the clinical hold on IGNITE DMD, the company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). [Solid Biosciences Inc.] Press Release

Nightstar Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for NSR-REP1 in Choroideremia

Nightstar Therapeutics plc announced that the FDA has granted RMAT designation to NSR-REP1, the company’s lead product candidate currently in Phase III development for the treatment of choroideremia, a rare, degenerative, genetic retinal disorder that leads to blindness. [Nightstar Therapeutics plc] Press Release

U.S. Legislators Back Larger Facilities Budget for NSF

The National Science Foundation (NSF) in Alexandria, Virginia, is in line for a budget increase of 4% to 5% next year. That assessment is based on bills approved recently by the spending committees in both chambers of Congress. Lawmakers have also signaled support for growing the account that NSF uses to build major new scientific facilities. [ScienceInsider] Editorial

Why the Medical Research Grant System Could Be Costing Us Great Ideas

The medical research grant system in the United States, run through the National Institutes of Health, is intended to fund work that spurs innovation and fosters research careers. In many ways, it may be failing. [The New York Times] Editorial

House Bill Gives NIH a 3% Boost in 2019, to $38.3 Billion

A draft bill released by a House of Representatives spending panel would give the National Institutes of Health (NIH) in Bethesda, Maryland, a $1.25 billion raise in 2019, to $38.3 billion. That is 3% more than this year’s level and $4.1 billion more than President Donald Trump’s administration had requested. [ScienceInsider] Editorial

FDA

Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry: Formal Meetings between the Food and Drug Administration and Biosimilar Biological Product Sponsors or Applicants (FR Doc. No:2018-12968) Notice

Human Immunodeficiency Virus-1 Infection: Developing Systemic Drug Products for Pre-Exposure Prophylaxis; Draft Guidance for Industry; Availability (FR Doc. No:2018-12761) Notice

NEW 2018 Tissue Engineering and Regenerative Medicine International Society (TERMIS) World Congress
September 4-7, 2018
Kyoto, Japan

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

NEW PhD Student – Cellular Immunotherapy (Glycostem Therapeutics)

Research Associate/PhD Position – iPS Cell-Derived Neurons to Model Parkinson’s Disease (TU Dresden)

Postdoctoral Fellow – Cell and Gene Therapies for Cardiac Arrhythmias and Heart Failure (Duke University)

Postdoctoral Position – Human Pluripotent Stem Cell-Based Cell Therapy in Type 1 Diabetes (University of Copenhagen)

Research Officer – Immunology (The Walter and Eliza Hall Institute of Medical Research)

Postdoctoral Fellow – Gene Editing and Cellular Reprogramming (Houston Methodist Research Institute)

Faculty Position – Adoptive T Cell Therapy (Dana-Farber Cancer Institute)

Group Manager – AAV Vector Expression Systems (Genethon)

Postdoctoral Fellow – Gene Therapy in Cardiovascular Disease (Emory University)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

Scientist/Senior Scientist – Bioinformatics and Data Sciences (Obsidian Therapeutics)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

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