Cell Therapy News Volume 17.09 | Mar 7 2016

Silencing of Anti-Chondrogenic MicroRNA-221 in Human Mesenchymal Stem Cells Promotes Cartilage Repair In Vivo
In vivo, the potential of microRNA-221 (miR-221) silenced human mesenchymal stromal cells was investigated by first encapsulating the cells in alginate and then by filling a cartilage defect in an osteochondral biopsy. After implanting the biopsy subcutaneously in nude mice, they found that silencing of miR-221 strongly enhanced in vivo cartilage repair compared to the control conditions. [Stem Cells] Abstract

CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo
The authors used a clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR-associated 9 genome-editing system to target the human proprotein convertase subtilisin/kexin type 9 PCSK9 gene in chimeric liver-humanized mice bearing human hepatocytes. [Arterioscler Thromb Vasc Biol] Abstract

Autologous Transplantation of Intestine-Isolated Glia Cells Improves Neuropathology and Restores Cognitive Deficits in β Amyloid-Induced Neurodegeneration
Scientists showed that the autologous transplantation of enteric glial cells into the brain of β-amyloid-injected rats arrested the development of the disease after their engraftment. [Sci Rep] Full Article

Intralymphatic mRNA Vaccine Induces CD8 T-Cell Responses that Inhibit the Growth of Mucosally Located Tumors
The authors demonstrated that E7-TriMix mRNA vaccine-induced CD8⁺ T lymphocytes migrate into the tumor nest and control tumor growth, although they do not express mucosa-associated markers such as CD103 or CD49a. [Sci Rep] Full Article

BMP7 Enhances the Effect of BMSCs on Extracellular Matrix Remodeling in a Rabbit Model of Intervertebral Disc Degeneration
Researchers investigated the efficacy and feasibility of stem cell therapy using bone morphogenetic protein (BMP)-7-transduced bone marrow mesenchymal stem cells (BMSCs) to promote extracellular matrix remodeling of degenerated intervertebral discs. [FEBS J] Abstract

Opposing Effects of Sca-1⁺ Cell-Based Systemic FGF-2 Gene Transfer Strategy on Lumbar versus Caudal Vertebrae in the Mouse
Investigators compared the osteogenic effects of stem cell-based FGF2 therapy on red marrow-filled lumbar vertebrae with those on yellow marrow-filled caudal vertebrae of young adult W⁴¹/W⁴¹ mice. [Gene Ther] Abstract

CD19xCD3 DART Protein Mediates Human B-Cell Depletion In Vivo in Humanized BLT Mice
Administration of the CD19xCD3 DART protein resulted in a dramatic sustained depletion of human CD19⁺ B cells from the peripheral blood, as well as a dramatic systemic reduction of human CD19⁺ B-cell levels in all tissues analyzed. [Mol Ther Oncolytics] Full Article

Stem Cell Therapy: A Promising Biological Strategy for Tendon–Bone Healing after Anterior Cruciate Ligament Reconstruction
From commonly investigated bone marrow-derived mesenchymal stem cells to emerging anterior cruciate ligament-derived CD34⁺ stem cells, multiple stem cell types have been proven to be effective in accelerating tendon–bone healing. The authors describe the current understanding of tendon–bone healing and summarize the current status of related stem cell therapy. [Cell Prolif] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research fields.

Top-Line Data from All Patients on the Primary Endpoint of Kiadis Pharma’s Phase II Trial with ATIR101™ Selected for Oral Presentation
Kiadis Pharma N.V. announced that key data from the company’s ongoing single-dose Phase II clinical trial with its lead product ATIR101™ has been selected for an oral presentation to discuss the top-line data. [Press release from the Kiadis Pharma N.V. discussing research to be presented at the 42^nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Valencia] Press Release

bluebird bio to Present Clinical Data on Lenti-D in CALD in Plenary Session
bluebird bio, Inc. announced that interim data from the ongoing Phase II/III Starbeam Study for the treatment of cerebral adrenoleukodystrophy (CALD) will be presented in an oral presentation. [Press release from the bluebird bio, Inc. discussing research to be presented at the American Academy of Neurology (AAN) 2016 Annual Meeting, Vancouver] Press Release

Advaxis Study in Head and Neck Cancer Selected for Late-Breaking Poster
Advaxis, Inc. announced that data from a Phase II study of its lead Lm immunotherapy candidate in HPV-associated head and neck cancer, axalimogene filolisbac (AXAL), has been selected as a late-breaker poster presentation. [Press release from Advaxis, Inc. discussing research to be presented at the 2016 American Association for Cancer Research (AACR) Annual Meeting, New Orleans] Press Release

Omalizumab Decreases Colds in Inner-City Children with Asthma, NIH Study Reports
Treatment with omalizumab significantly decreases the number of colds in inner-city children with allergic asthma, researchers reported at a press conference. [Press release from National Institute of Allergy and Infectious Diseases discussing research presented at the American Academy of Allergy, Asthma and Immunology (AAAAI) 2016 Annual Meeting, Los Angeles] Press Release

Republicans Promise ‘Sustained’ NIH Funding Hikes without Budget Gimmicks
The two legislators with arguably the most clout over the budget of the National Institutes of Health (NIH) promised to give NIH “at least” the $1 billion increase that President Barack Obama has requested. [ScienceInsider] Editorial

Indian Scientists Decry Spending Plan
Science spending in India is slated to rise 11% in the 2016–17 fiscal year to $1.19 billion, according to the budget proposal presented by the government of Prime Minister Narendra Modi. [ScienceInsider] Editorial

Italian Scientists Protest ’Serious Neglect’ of Research
Fed up with their country’s politicians, Italian scientists have launched a national debate about the dire state of their country’s research system. They want the government to reverse years of budget cuts and prevent increasing numbers of researchers moving overseas for work. [ScienceInsider] Editorial

How the US CRISPR Patent Probe Will Play Out
There is no shortage of optimism about the scientific potential of CRISPR–Cas9, a technique that can precisely alter the genomes of everything from wheat to elephants. But there is a great deal of confusion over who will benefit financially from its use. On 10 March, the US Patent and Trademark Office will begin an investigation into who deserves the patent on using CRISPR–Cas9 to edit genes. [Nature News] Editorial

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Baxalta Commences Phase I Clinical Trial of BAX 826, the Company’s Second Extended Half-Life Factor VIII Treatment for Hemophilia A
Baxalta Incorporated announced that it has dosed the first patient in its Phase I, first-in-human clinical trial of BAX 826, a recombinant Factor VIII treatment for hemophilia A that uses proprietary polysialic acid technology to extend its circulating half-life. [Baxalta Incorporated] Press Release

miRagen Therapeutics Initiates First Clinical Trial for MRG-106 in Lymphoma Patients
miRagen Therapeutics, Inc. announced that it has initiated a Phase I clinical study of its anti-cancer product candidate MRG-106, a synthetic microRNA antagonist of microRNA-155. [miRagen Therapeutics, Inc.] Press Release

Celyad Receives the First US Patent Covering a Method of Producing Allogeneic TCR-Deficient CAR T-Cells
Celyad announced the issuance of United States Patent No. 9,273,283 relating to a method of producing allogeneic primary human T cells that are engineered to be T-Cell Receptor (TCR)-deficient and express a chimeric antigen receptor (CAR). [Celyad] Press Release

Merck, Pfizer and Verastem Announce Combination Trial of Avelumab and VS-6063 in Ovarian Cancer
Merck, Pfizer and Verastem announced that they have entered into an agreement to evaluate avelumab, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, in combination with Verastem’s VS-6063, an investigational focal adhesion kinase inhibitor, in patients with advanced ovarian cancer. [Verastem, Inc.] Press Release

SELLAS Life Sciences Announces FDA Orphan Drug Designation for WT1 Cancer Vaccine in Patients with Malignant Pleural Mesothelioma
SELLAS Life Sciences Group announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for the company’s WT1 cancer vaccine for the treatment of patients with malignant pleural mesothelioma. [SELLAS Inc.] Press Release

Wills Eye Hospital Treats 1st U.S. Patient in New Gene Therapy RESCUE Trial
Wills Eye Hospital announced it has treated the first RESCUE trial patient in the United States enrolled in an FDA-approved gene therapy vision research study. [Wills Eye Hospital (PR Newswire Association LLC)] Press Release

Cancer MoonShot 2020 Community Oncology Milestone: National Immunotherapy Coalition (NIC) Partners with New England Cancer Specialists to Advance Cancer Care in New England
Cancer MoonShot 2020 announced its clinical affiliation with New England Cancer Specialists (NECS), aimed to greatly expand patients’ access to advanced cancer care, innovative clinical trials, and survivorship resources in the community. [Cancer MoonShot 2020] Press Release

FDA Accepts Supplemental Biologics License Application (sBLA) for KEYTRUDA^® (Pembrolizumab) in Advanced Non-Small Cell Lung Cancer
Merck announced that the U.S. Food and Drug Administration (FDA) has accepted for review a new sBLA for KEYTRUDA^®, the company’s anti-PD-1 therapy, to include data from KEYNOTE-010. [Merck Sharp & Dohme Corp.] Press Release

Center for Scientific Review; Notice of Closed Meeting (FR Doc. No: 2016-04446)

National Institute of Allergy And Infectious Diseases; Notice of Closed Meeting (FR Doc. No: 2016-04361)

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.

Scientist – Human Pluripotent Stem Cell Neural Differentiation (STEMCELL Technologies Inc.)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Fellowship – Stem Cells, Oligodendrogenesis and Remyelination (UC Davis)

PhD Positions – Cancer, Microbiology and Stem Cell Engineering (Virginia Commonwealth University)

PhD Positions – Tendon Repair and Regeneration (NUI Galway)

PhD Position – Leukemogenesis and Treatment Resistance (Institute for Tumor Biology and Experimental Therapy)

Postdoctoral Positions – Gene Regulation and Translational Cancer Research (University of Texas Health Science Center)

Postdoctoral Position – Hematology/Oncology (Penn State College of Medicine)

Postdoctoral Fellow/Research Associate – Cancer Therapy (Memorial Sloan Kettering Cancer Center)

Translational Research Scientist – Hematology/Oncology (University of Chicago)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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