Volume 16.01 | Jan 12

Issue 16.01 January 12, 2015
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Researchers Grow Functional Tissue-Engineered Intestine from Human Cells
A new study has shown that tissue-engineered small intestine grown from human cells replicates key aspects of a functioning human intestine. The tissue-engineered small intestine they developed contains important elements of the mucosal lining and support structures, including the ability to absorb sugars, and even tiny or ultra-structural components like cellular connections. [Press release from the Children’s Hospital Los Angeles discussing online prepublication in American Journal of Physiology: GI & Liver] Press Release | Abstract
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Bone Marrow Minimal Residual Disease Was an Early Response Marker and a Consistent Independent Predictor of Survival after Anti-GD2 Immunotherapy
After achieving first remission, primary refractory disease, or second remission, children with stage four neuroblastoma received anti-GD2 3F8 antibody immunotherapy. Bone marrow minimal residual disease before 3F8 treatment and after cycle two was measured using a four-marker panel by quantitative reverse transcription polymerase chain reaction. [J Clin Oncol] Abstract

Effect of Repetitive Intra-Arterial Infusion of Bone Marrow Mononuclear Cells in Patients with No-Option Limb Ischemia: The Randomized, Double-Blind, Placebo-Controlled Juventas Trial
The Juventas trial is a randomized, double-blind, placebo-controlled clinical trial in 160 patients with severe, non-revascularizable limb ischemia. Scientists determined whether repetitive intra-arterial infusion of bone marrow mononuclear cells in patients with severe, non-revascularizable limb ischemia could prevent major amputation. [Circulation] Abstract

Localized RNAi Therapeutics of Chemoresistant Grade IV Glioma Using Hyaluronan-Grafted Lipid-Based Nanoparticles
Researchers devised a localized strategy to deliver RNA interference (RNAi) directly to the glioblastoma multiforme site using hyaluronan-grafted lipid-based nanoparticles (LNPs). These LNPs were previously shown to be highly effective in delivering small interfering RNAs into various cell types. [ACS Nano] Abstract

Three-Dimensional CaP/Gelatin Lattice Scaffolds with Integrated Osteoinductive Surface Topographies for Bone Tissue Engineering
A novel fabrication process using computer numerically controlled machining and lamination is reported to make 3D calcium phosphate/gelatin composite scaffolds with integrated surface micropatterns that are introduced by embossing prior to machining. Both in vitro and in vivo studies show that micropatterned composite scaffolds with surfaces comprising 40 μm pits and 50 μm grooves were optimal for improved osteogenesis. [Biofabrication] Abstract

Permanent Acceptance of Mouse Cardiac Allografts with CD40 siRNA to Induce Regulatory Myeloid Cells by Use of a Novel Polysaccharide siRNA Delivery System
Scientists developed a novel small interfering RNA (siRNA) delivery system with a poly-dA extension at the 5′-end of the siRNA sense strand that was stably incorporated into 1,3-β-glucan. This was captured and incorporated into dendritic cells through its receptor, Dectin-1, specifically silencing CD40 genes to exert immunoregulatory activity. [Gene Ther] Abstract

TGF-β1-siRNA Delivery with Nanoparticles Inhibits Peritoneal Fibrosis
Investigators attempted to develop an in vivo small interfering RNA (siRNA) delivery system with liposome-based nanoparticles to the peritoneum to inhibit peritoneal fibrosis. [Gene Ther] Abstract

Safety and Feasibility for Pediatric Cardiac Regeneration Using Epicardial Delivery of Autologous Umbilical Cord Blood-Derived Mononuclear Cells Established in a Porcine Model System
Investigators aimed to establish a porcine pipeline to evaluate the feasibility and long-term safety of autologous umbilical cord blood mononuclear cells transplanted into the right ventricle of juvenile porcine hearts. [Stem Cells Transl Med] Abstract

Therapeutic Potential of Adipose-Derived SSEA-3-Positive Muse Cells for Treating Diabetic Skin Ulcers
Skin ulcers were generated in severe combined immunodeficiency (SCID) mice with type 1 diabetes, which showed delayed wound healing compared with nondiabetic SCID mice. Treatment with stage-specific embryonic antigen-3 (SSEA-3)+-enriched fraction (Muse-rich) cells significantly accelerated wound healing compared with treatment with Muse-poor cells. [Stem Cells Transl Med] Abstract

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Stromal Cells and Stem Cells in Clinical Bone Regeneration
The authors highlight the successes and challenges reported in several clinical trials that involved the use of bone-marrow-derived mesenchymal or adipose-tissue-derived stromal cells. [Nat Rev Endocrinol] Abstract

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

Free Cell Stem Cell Poster: Directed Differentiation of ESCs/iPSCs
Dicerna Pharmaceuticals Presents Positive Preclinical Data on Its DsiRNA Therapeutics Targeting MYC and β-Catenin at AACR MYC: From Biology to Therapy Meeting
Dicerna Pharmaceuticals, Inc. announced the presentation of new preclinical data on its Dicer substrate short interfering RNA (DsiRNA) therapeutics targeting MYC and β-catenin oncogenes in models of hepatocellular carcinoma and colorectal cancer. [Press release from Dicerna Pharmaceuticals, Inc. discussing research presented at MYC: From Biology to Therapy, La Jolla] Press Release | Poster

Arrowhead Provides Update on IND for ARC-520 Phase IIb Study
Arrowhead Research Corporation, a biopharmaceutical company developing targeted RNAi therapeutics, announced that the U.S. Food and Drug Administration verbally informed the company in a preliminary call of a partial clinical hold, under which the company is cleared to begin a modified multiple-dose study of ARC-520 in patients with chronic hepatitis B infection. [Arrowhead Research Corporation] Press Release

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Biochemist Questions Peer Review at UK Funding Agency
When the UK Medical Research Council turned down two proposals from biochemist Ian Eperon – despite high scores from peer reviewers — he decided to find out how closely the agency follows the evaluations it invites from experts. After much chasing and a freedom-of-information request, he has received figures that surprised him: in 2013–14, the scores given to grant proposals in his field by outside reviewers had little impact on whether the proposal got funded. [Nature News] Editorial

Largest U.S. Biomedical Research Society Weighs in on NIH’s Budget Woes
Biomedical researchers are once again wringing their hands about a surfeit of scientists chasing too few research dollars and academic jobs. In a report, the Federation of American Societies for Experimental Biology, which represents more than 120,000 scientists, endorses familiar solutions and adds a few new ones. [ScienceInsider] Editorial
Alnylam and Isis Form New Agreement, Extending Decade-Long Partnership for Leadership in RNA Therapeutics
Isis Pharmaceuticals, Inc. and Alnylam Pharmaceuticals, Inc. announced they have formed a new agreement, extending their existing strategic partnership – formed originally in 2004 – to lead the development and commercialization of RNA therapeutics. [Alnylam Pharmaceuticals, Inc.] Press Release

Incyte and Agenus Announce Global Alliance to Develop Novel Immuno-Oncology Antibodies
Incyte Corporation and Agenus Inc. announced a global license, development and commercialization agreement focused on novel immuno-therapeutics using Agenus’ proprietary Retrocyte Display™ antibody discovery platform. [Incyte Corporation] Press Release

Argos Therapeutics and Saint-Gobain Announce Agreement for Production of Disposables Used in Automated Manufacturing of Fully Personalized Immunotherapies
Argos Therapeutics Inc. announced a collaboration with Saint-Gobain’s Performance Plastics division. Saint-Gobain will partner with Argos to design, integrate and scale production of a range of disposables for use in the automated manufacturing of Argos’ lead product candidate, AGS-003, currently being tested in a Phase III clinical trial for the treatment of metastatic renal cell carcinoma. [Argos Therapeutics Inc.] Press Release

Intellia Therapeutics, Novartis Form Partnership to Develop New Cell Therapies Using CRISPR/Cas9 Technology
Intellia Therapeutics announced a five-year research and development collaboration with Novartis to accelerate the ex vivo development of new CRISPR/Cas9-based therapies using chimeric antigen receptor T cells and hematopoietic stem cells. [Intellia Therapeutics] Press Release

MedImmune Enters Licensing Agreement with Omnis Pharmaceuticals for Oncolytic Viruses in Immuno-Oncology
AstraZeneca announced that MedImmune has entered into a licensing agreement with Omnis Pharmaceuticals. This agreement will allow MedImmune to combine key agents from its investigational immunotherapy portfolio with Omnis’ lead investigational oncolytic virus program, a genetically engineered strain of vesicular stomatitis virus. [AstraZeneca] Press Release

Cardio3 BioSciences Enters Immuno-Oncology Space with Acquisition of OnCyte CAR T-Cell Portfolio from Celdara Medical
Cardio3 BioSciences announced the acquisition of OnCyte, the oncology division of privately-held U.S. biotechnology company Celdara Medical, and its portfolio of immuno-oncology product candidates. [Cardio3 BioSciences] Press Release

Aratana Therapeutics Granted Full License for AT-004 by USDA
Aratana Therapeutics, Inc. announced that AT-004, the company’s canine-specific monoclonal antibody targeting CD20 as an aid in the treatment of B-cell lymphoma in dogs, has received a full license from the U.S. Department of Agriculture (USDA). [Aratana Therapeutics, Inc.] Press Release

Argos Therapeutics Provides Update on Clinical Research for Investigational Fully Personalized Immunotherapy for the Treatment of HIV
Argos Therapeutics Inc. provided an update on its clinical research program for AGS-004, the company’s investigational fully personalized immunotherapy for the treatment of HIV. The primary endpoint of the trial was not achieved. [Argos Therapeutics Inc.] Press Release

OncoSec Medical Plans to Initiate Pilot Study in Triple Negative Breast Cancer
OncoSec Medical Inc., a company developing DNA-based intratumoral cancer immunotherapies plans to initiate a pilot study to assess IL-12 ImmunoPulse in patients with triple negative breast cancer. [OncoSec Medical Inc.] Press Release
Center for Scientific Review: Notice of Closed Meetings (FR Doc. No: 2015-00118)
Summary of Safety and Effectiveness Data – INTERCEPT Blood System for Plasma
Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Guidance for Industry on Postmarketing Adverse Event Reporting for Medical Products and Dietary Supplements During an Influenza Pandemic; Availability (FR Doc. No: 2014-30907)
NEW Keystone Symposium – Heart Disease and Regeneration: Insights from Development
March 1-6, 2015
Copper Mountain, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Fellow – Wnt Signaling in Development and Disease (Van Andel Research Institute)

NEW Cell Therapy Manufacturing Scientist (SLS Services Ltd.)

NEW Postdoctoral Fellowship – CNS Inflammation (Rutgers University)

Postdoctoral Fellow – Radiation Therapy and Immunotherapy (Georgia Regents University Cancer Center)

PhD Student – Experimental Leukemia Research (University Children’s Hospital Basel)

Cell Therapy Senior Manager (taylorollinson Ltd.)

Mesenchymal Stem Cell (MSC) Laboratory Technician (California Cryobank)

Clinical Laboratory Scientist – Umbilical Cord Blood Processing (California Cryobank)

Postdoctoral Researcher – Chromatin and Epigenetic Regulation (Van Andel Research Institute)

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Scientist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

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