Cell Therapy News 19.21 June 18, 2018 | |
| |
TOP STORYInvestigators 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 | |
| |
PUBLICATIONS(Ranked by impact factor of the journal)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 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 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-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 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 | |
| |
REVIEWSProgramming 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. | |
| |
SCIENCE NEWSbluebird 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 23rd Congress of the European Hematology Association (EHA), Stockholm] Press Release | |
| |
INDUSTRY NEWSSarepta 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 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 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, 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 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 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 | |
| |
POLICY NEWSU.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
| |
REGULATORYFDAAgency 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
| |
EVENTSNEW 2018 Tissue Engineering and Regenerative Medicine International Society (TERMIS) World Congress Visit our events page to see a complete list of events in the community.
| |
JOB OPPORTUNITIESNEW PhD Student – Cellular Immunotherapy (Glycostem Therapeutics) Research Associate/PhD Position – iPS Cell-Derived Neurons to Model Parkinson’s Disease (TU Dresden) 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) 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. | |
|