Volume 15.06 | Feb 17

Issue 15.06 February 17, 2014
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Better RNA Interference, Inspired by Nature
Inspired by tiny particles that carry cholesterol through the body, chemical engineers have designed nanoparticles that can deliver snippets of genetic material that turn off disease-causing genes. The new particles, which encase short strands of RNA within a sphere of fatty molecules and proteins, silence target genes in the liver more efficiently than any previous delivery system, the researchers found in a study of mice. [Press release from the Massachusetts Institute of Technology discussing online prepublication in the Proceedings of the National Academy of Sciences USA] Press Release | Abstract
Free Nature Reviews Immunology Poster: The Immune Response to HIV
PUBLICATIONS (Ranked by impact factor of the journal)
Combination Immunotherapy after ASCT for Multiple Myeloma Using MAGE-A3/Poly-ICLC Immunizations Followed by Adoptive Transfer of Vaccine-Primed and Costimulated Autologous T Cells
Myeloma-directed cellular immune responses after autologous stem cell transplantation (ASCT) may reduce relapse rates. Scientists studied whether coinjecting the TLR-3 agonist and vaccine adjuvant Poly-ICLC with a MAGE-A3 peptide vaccine was safe and would elicit a high frequency of vaccine-directed immune responses when combined with vaccine-primed and costimulated autologous T cells. [Clin Cancer Res] Abstract

Human Adipose Derived Stromal/Stem Cells (hASCs) Protect against STZ-Induced Hyperglycemia; Analysis of hASC-Derived Paracrine Effectors
Scientists showed that systemic administration of hASCs improved glucose tolerance, preserved β cell mass, and increased β cell proliferation in STZ-treated NOD-SCID mice. [Stem Cells] Abstract

Stage-Specific Roles for Cxcr4 Signaling in Murine Hematopoietic Stem/Progenitor Cells in the Process of Bone Marrow Repopulation
Researchers report that modification of C-X-C chemokine receptor type 4 (Cxcr4) signaling in murine hematopoietic stem/progenitor cells does not significantly affect initial homing/lodging events, but leads to alteration in subsequent bone marrow repopulation kinetics, with observations confirmed by both gain- and loss-of-function approaches. [Stem Cells] Abstract

Promotion of Cardiac Differentiation of Brown Adipose Derived Stem Cells by Chitosan Hydrogel for Repair after Myocardial Infarction
In vivo, brown adipose derived stem cells carrying double-fusion reporter gene (firefly luciferase and monomeric red fluorescent protein) were transplanted into infarcted rat hearts with or without chitosan hydrogel. [Biomaterials] Abstract

PlGF-MMP9-Engineered iPS Cells Supported on a PEG-Fibrinogen Hydrogel Scaffold Possess an Enhanced Capacity to Repair Damaged Myocardium
Researchers assessed the reparative potential of induced pluripotent stem (iPS) cells bioengineered to secrete placental growth factor (PlGF) or matrix metalloproteinase 9 (MMP9) and delivered to infarcted myocardium upon a poly(ethylene glycol)-fibrinogen scaffold. [Cell Death Dis] Full Article

Epidermal Stem Cells Cultured on Collagen-Modified Chitin Membrane Induce In Situ Tissue Regeneration of Full-Thickness Skin Defects in Mice
The transplantation of epidermal stem cells- collagen-chitin biomimetic membrane (ESCs-C-CBM) has achieved in situ skin regeneration. In nude mice, compared to controls with collagen-chitin biomimetic membrane only, the ESCs-C-CBM group had significantly more dermatoglyphs on the skin wound 10 weeks after surgery, and the new skin was relatively thick, red and elastic. [PLoS One] Full Article

Human Adipose-Derived Stem Cell Transplantation as a Potential Therapy for Collagen VI-Related Congenital Muscular Dystrophy
Adipose-derived stem cells (ADSCs) were examined for expression of extracellular matrix and related genes using gene expression array analysis. The therapeutic potential of ADSC was assessed after a single intramuscular transplantation in collagen VI-deficient mice. [Stem Cell Res Ther] Full Article

Electrical Integration of Human Embryonic Stem Cell-Derived Cardiomyocytes in a Guinea Pig Chronic Infarct Model
Investigators aimed at determining the ability of human embryonic stem cell-derived cardiomyocytes to integrate and modulate electrical stability following transplantation in a chronic model of cardiac injury. [J Cardiovasc Pharmacol Ther] Abstract

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Interferon-Gamma Modification of Mesenchymal Stem Cells: Implications of Autologous and Allogeneic Mesenchymal Stem Cell Therapy in Allotransplantation
The authors examine the potential benefits of mesenchymal stem cells (MSC) therapy in the context of allotransplantation. They also discuss the use of autologous and allogeneic MSC and the issues associated with their immunogenicity in vivo, with particular focus on the use of enhanced Interferon-gamma preactivated MSC cellular therapy. [Stem Cell Rev] Abstract

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

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SanBio Announces the First Public Presentation of Data from Its On-Going Human Clinical Trial of Cell Therapy for Chronic Stroke Disability
SanBio announced the presentation of safety and efficacy data from its on-going Phase I/IIa clinical trial of SB623 cell therapy in patients with chronic stroke disability. The data presented show that the clinical outcome results showing benefit on all three stroke scales (NIHSS, ESS and Fugl-Meyer) were highly significant at 6, 9 and 12 months post treatment. [Press release from SanBio, Inc. discussing research presented at the International Stroke Conference, San Diego] Press Release

Human Embryonic Stem Cell-Derived RPE Cell Transplantation Yields No ‘Untoward Pathologic Reactions’
A study under way to evaluate safety and tolerability of subretinal transplantation of human embryonic stem cells in patients with dry age-related macular degeneration so far has yielded no “untoward pathologic reactions,” Ninel Z. Gregori, MD said. [Press release from Healio discussing research presented at the Angiogenesis, Exudation, and Degeneration 2014 Meeting, Miami] Press Release

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Myriad Wins First Round in Cancer Gene Testing Battle
After the U.S. Supreme Court last year invalidated human gene patents held by Utah-based Myriad Genetics, the firm’s competitors launched an array of products aimed at cracking Myriad’s stranglehold on the breast cancer gene testing market. Myriad, however, promptly filed a bevy of lawsuits against its challengers, arguing that the high court decision didn’t apply to related patents it held on testing for the BRCA gene mutations linked to cancer risk. Last week, a company called Gene by Gene retreated from its Myriad challenge, agreeing to stop selling test kits in North America. [ScienceInsider] Editorial

AAAS Announces Open-Access Journal
Joining a herd of other scientific societies, AAAS announced that it will launch the organization’s first online, fully open-access journal early next year. The new journal, called Science Advances, will give authors another outlet for papers that they are willing to pay to make immediately free to the public. [ScienceInsider] Editorial

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Cynata Partners with Leading Biologic Product Manufacturer
Cynata Therapeutics Ltd announced that it has signed a manufacturing agreement with Waisman Biomanufacturing under which Waisman will undertake manufacturing process development, scale-up and clinical-grade manufacture of Cynata’s proprietary Cymerus™ off-the-shelf stem cell product. [Cynata Therapeutics Ltd] Press Release

California Stem Cell Partners on CIRM Grant to Develop Transplantable 3D Retinas
California Stem Cell, Inc. announced the initiation of a collaborative study with the University of California, Irvine aimed to create transplantable three-dimensional (3D) retinal tissue. The study, funded by a $4.5 million grant from the California Institute of Regenerative Medicine (CIRM) will investigate the potential of improving a patient’s visual function by transplanting human stem cell-derived 3D retinal tissue into their retina. [California Stem Cell, Inc.] Press Release

DiaVacs Announces a Strategic Collaboration with Profil Institute to Conduct Phase Ib/IIa Randomized Clinical Trial of New Cell-Based Therapy for Type 1 Diabetes
DiaVacs, Inc. and Profil® Institute for Clinical Research, Inc. announced a strategic collaboration to conduct DiaVacs’ Phase Ib/IIa clinical trial. This clinical trial will study the safety and preliminary efficacy of DiaVacs’ novel dendritic cell therapy for treatment of type 1 diabetes in patients who are newly diagnosed. [Profil® Institute for Clinical Research, Inc.] Press Release

Aurigene Discovery Technologies Limited and Pierre Fabre Announce a Licensing Agreement for a New Cancer Therapeutic in Immuno-Oncology: AUNP12, an Immune Checkpoint Modulator Targeting the PD-1 Pathway
Pierre Fabre and Aurigene announced that the two companies have entered into a collaborative license, development and commercialization agreement granting Pierre Fabre global Worldwide rights (excluding India) to a new immune checkpoint modulator, AUNP-12. [Aurigene Discovery Technologies Limited] Press Release

Third Rock Ventures Launches Voyager Therapeutics with $45 Million Series A to Develop Life-Changing Gene Therapies for CNS Disorders
Third Rock Ventures, LLC announced the formation of Voyager Therapeutics, a gene therapy company developing treatments for fatal and debilitating diseases of the central nervous system (CNS), with a $45 million Series A financing. [Business Wire] Press Release

StemCells, Inc. Announces Completion of the First of Two Cohorts in Its Dry Age-Related Macular Degeneration Trial
StemCells, Inc. announced that it has completed enrollment of the first of two planned patient cohorts in the company’s clinical trial of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) for dry age-related macular degeneration. [StemCells, Inc.] Press Release

CardioCel® Cleared for Sale in US
Admedus announced it has received FDA clearance to market CardioCel® in the US. CardioCel® is the group’s lead regenerative tissue product to repair and treat a range of cardiovascular and vascular defects. [Admedus Ltd.] Press Release

Parcell Laboratories Promotes Colin White, Ph.D., to Chief Scientific Officer
Parcell Laboratories, Inc., developer of novel therapeutics based on its patented adult stem cell platform technology, the ELA® cell, has promoted Colin White, Ph.D., to the newly-created position of chief scientific officer. [Marketwire L.P.] Press Release
Government-Owned Inventions; Availability for Licensing (FR Doc No. 2014-03083)

Prospective Grant of Exclusive License: Pre-Clinical Evaluation and Commercial Development of Human Therapeutics for Liver Cancer and Ovarian Cancer Within the Scope of the Licensed Patent Rights (FR Doc No. 2014-03410)
Food and Drug Administration (United States)

Synergizing Efforts in Standards Development for Cellular Therapies and Regenerative Medicine Products; Public Workshop (FR Doc No. 2014-03015)
NEW 2nd Annual Cell Culture Asia Congress
September 25-26, 2014
Singapore, Singapore

NEW 3rd Annual Cell Culture & Bioprocessing Congress 2014
November 17-18, 2014
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Cardiovascular Electrophysiology Research Involving Human ESC or iPSC-Derived Cardiomyocytes (Stanford University)

NEW Research Technologist – Pluripotent Stem Cells (STEMCELL Technologies Inc.)

NEW Research Technologist – Particle Chemistry (STEMCELL Technologies Inc.)

NEW Research Technologist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

Postdoctoral Fellow – Hemostasis Gene Therapy (Children’s Hospital of Philadelphia)

Postdoctoral Fellowships – Tissue Regeneration and Molecular Cell Engineering (MD Anderson Cancer Center)

Project Manager – Cell Therapy (University of California, Irvine)

Postdoctoral Position – Epigenetic Enzymes in Breast Cancer (University of Wisconsin – Madison)

Postdoctoral Position – Tissue Engineering and Regenerative Medicine (RUNMC)

Postdoctoral Fellow – Morphogenetic Signaling Pathways in Development and Disease (University of Rochester Medical Center)

Scientific Affairs Coordinator (American Society of Hematology)

Postdoctoral Research Fellow (Brigham & Women’s Hospital and Harvard Medical School)

Stem Cell Manufacturing Scientist (Paramount Recruitment)

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