Volume 15.23 | Jun 16

Issue 15.23 June 16, 2014
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A Vaccination to Prevent Heart Attacks?
Scientists developed a “genome editing” approach for permanently reducing cholesterol levels in mice with a single injection, potentially reducing heart attack risk by up to 90 percent. The work focused on altering the function of a gene in the liver called PCSK9. [Press release from the Harvard Stem Cell Institute discussing online prepublication in Circulation Research] Press Release | Abstract
ISSCR 2014: Our City. Our Science. Visit us at Booth #817.
PUBLICATIONS (Ranked by impact factor of the journal)
Sirolimus-Based Graft-versus-Host Disease Prophylaxis Promotes the In Vivo Expansion of Regulatory T Cells and Permits Peripheral Blood Stem Cell Transplantation from Haploidentical Donors
Investigators explored in 121 patients, mostly with advanced stage diseases, a sirolimus-based, calcineurin-inhibitor-free prophylaxis of graft-versus-host disease to allow the infusion of unmanipulated peripheral blood stem cell grafts from partially HLA-matched family donors. [Leukemia] Abstract

HLA-Haploidentical Transplantation with Regulatory and Conventional T Cell Adoptive Immunotherapy Prevents Acute Leukemia Relapse
Researchers investigated whether T regulatory cell-conventional T cell adoptive immunotherapy prevents post-transplant leukemia relapse. 43 adults with high-risk acute leukemia were conditioned with a TBI-based regimen. [Blood] Abstract

Rapamycin Relieves Lentiviral Vector Transduction Resistance in Human and Mouse Hematopoietic Stem Cells
Investigators showed that rapamycin, an allosteric inhibitor of the mammalian target of rapamycin complexes, facilitates highly efficient lentiviral transduction of mouse and human hematopoietic stem cells and dramatically enhances marking frequency in long-term engrafting cells in mice. [Blood] Abstract

Mononuclear Cells from Dedifferentiation of Mouse Myotubes Display Remarkable Regenerative Capability
Scientists showed that ectopic expression of Msx1 reprograms post-mitotic, multinucleated, primary mouse myotubes to become proliferating mononuclear cells. These dedifferentiated cells reactivate genes expressed by embryonic muscle progenitor cells and generate only muscle tissue in vivo both in an ectopic location and inside existing muscle. [Stem Cells] Abstract

Cardiac RNAi Therapy Using RAGE siRNA/Deoxycholic Acid-Modified Polyethylenimine Complexes for Myocardial Infarction
To avoid deleterious effects of overwhelming inflammation, researchers blocked the expression of receptor for advanced glycation end-products (RAGE), a key mediator of the local and systemic inflammatory responses, via RNAi mechanism. [Biomaterials] Abstract

Naturally Enveloped AAV Vectors for Shielding Neutralizing Antibodies and Robust Gene Delivery In Vivo
The authors demonstrate that in mice, adeno-associated virus (AAV) vectors associated with extracellular vesicles (EVs) can evade human anti-AAV neutralizing antibodies. They observed different antibody evasion and gene transfer abilities with populations of EVs isolated by different centrifugal forces. [Biomaterials] Abstract

Application of Cell Sheet Technology to Bone Marrow Stromal Cell Transplantation for Rat Brain Infarct
Investigators assessed whether a novel cell-sheet technology non-invasively serves therapeutic benefits to ischemic stroke. The monolayered cell sheet was engineered by culturing rat bone marrow stromal cells on a temperature-responsive dish. The cell sheet was analyzed histologically and then transplanted onto the ipsilateral neocortex of rats subjected to permanent middle cerebral artery occlusion at seven days after the insult. [J Tissue Eng Regen Med] Abstract

Bone Tissue Engineering with a Collagen-Hydroxyapatite Scaffold and Culture Expanded Bone Marrow Stromal Cells
The authors developed a collagen-hydroxyapatite scaffold that supports consistent osteogenesis by donor-derived osteoprogenitors, and is more easily degraded than a pure ceramic scaffold. They examined the biological performance in vivo in a critical-size mouse calvarial defect. [J Biomed Mater Res B Appl Biomater] Abstract

Hypoxia-Targeted Triple Suicide Gene Therapy Radiosensitizes Human Colorectal Cancer Cells
Scientists investigated whether hypoxia-driven triple suicide gene TK/CD/UPRT expression enhances cytotoxicity to ganciclovir and 5-fluorocytosine, and sensitizes human colorectal cancer to radiation in vitro and in vivo. [Oncol Rep] Abstract

Human ESC-Derived MSCs Outperform Bone Marrow MSCs in the Treatment of an EAE Model of Multiple Sclerosis
Researchers showed that mesenchymal stem cells derived from human embryonic stem cells (hES-MSCs) significantly reduce clinical symptoms and prevent neuronal demyelination in a mouse experimental autoimmune encephalitis (EAE) model of MS, and that the EAE disease-modifying effect of hES-MSCs is significantly greater than that of human bone-marrow-derived MSCs. [Stem Cells Rep] Full Article | Press Release

Long-Term Health of Dopaminergic Neuron Transplants in Parkinson’s Disease Patients
To determine the long-term health and function of transplanted dopamine neurons in Parkinson’s disease patients, the expression of dopamine transporters and mitochondrial morphology were examined in human fetal midbrain cellular transplants. [Cell Rep] Full Article | Graphical Abstract | Press Release

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Plasticity of Epithelial Stem Cells in Tissue Regeneration
The authors discuss how different populations of naturally lineage-restricted stem cells and committed progenitors can display remarkable plasticity and reversibility and reacquire long-term self-renewing capacities and multilineage differentiation potential during physiological and regenerative conditions. They also discuss the implications of cellular plasticity for regenerative medicine and for cancer. [Science] Abstract

Cell Based Strategies to Manage Leukemia Relapse: Efficacy and Feasibility of Immunotherapy Approaches
Unmanipulated or minimally manipulated donor T cells may also be effective in a minority of patients but the main limit remains the induction of severe graft versus host disease. This clinical complication has brought about a huge research effort which led to the development of leukemia specific T cell therapy aiming at the direct recognition of leukemia specific rather than minor histocompatibility antigens. [Leukemia] Abstract

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

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Cardium to Announce Interim Results to Generx Angiogenic Gene Therapy Phase III Clinical Study
Taxus Cardium Pharmaceuticals Group Inc. announced that Cardium Therapeutics will announce interim results on the Generx® angiogenic gene therapy product candidate international ASPIRE Phase III. [Press release from Taxus Cardium Pharmaceuticals Group Inc. discussing research to be presented at the 2014 BIO International Convention, San Diego] Press Release

Five Scientific Communications Further Support DBV’s EPIT™ Disease Modifying Effect
DBV Technologies presented robust preclinical data. The data further confirms epicutaneous immunotherapy’s ability (EPIT™) to modulate over time immune responses due to a powerful T cell induction effect in allergic patients. [Press release from DBV Technologies, Inc. discussing research presented at the 2014 European Academy of Allergy and Clinical Immunology (EAACI) Annual Meeting, Copenhagen] Press Release

bluebird bio Reports Rapid Transfusion Independence in Beta-Thalassemia Major Patients Treated with Its LentiGlobin Product Candidate
bluebird bio, Inc., a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, released initial positive clinical data from its HGB-205 clinical study of its LentiGlobin BB305 product candidate in beta-thalassemia major subjects. [Press release from bluebird bio, Inc. discussing research presented research at the 19th Annual Congress of the European Hematology Association (EHA), Milan] Press Release

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E.U. Medicines Agency Relaxes Data-Sharing Rules
Academics breathed a cautious sigh of relief when the European Medicines Agency announced that it would soften controversial draft rules to open clinical trial data to public scrutiny. In particular, the agency says it will let researchers “download, save and print the trial data for academic and non-commercial research purposes.” [ScienceInsider] Editorial

Japanese Stem Cell Debacle Could Bring Down Center
Shutting down the research center at the heart of an unfolding scientific scandal may be necessary to prevent a recurrence of research misconduct, according to a report released at a press conference. A committee reviewing conduct at the RIKEN Center for Developmental Biology found lax oversight and a failure on the part of senior authors of two papers in Nature outlining a surprisingly simple way of reprogramming mature cells into stem cells. [ScienceInsider] Editorial

U.S. Senate Bill Would Give NIH 2% Raise in 2015
A U.S. Senate spending panel approved a draft bill that would raise the National Institutes of Health’s (NIH’s) budget by $605.7 million, to $30.5 billion, in the 2015 fiscal year. [ScienceInsider] Editorial
Kadmon Corporation Announces NIH Agreement to Evaluate Gene Therapy Program
Kadmon Corporation, LLC, announced that it has established a Cooperative Research and Development Agreement with the National Institute of Dental and Craniofacial Research, an institute of the National Institutes of Health, to develop an Adeno-associated virus vector containing the human aquaporin-1 gene for the treatment of xerostomia resulting from the use of ionizing radiation in the treatment of head and neck cancer. [Kadmon Corporation, LLC] Press Release

CCRM and Stem Cells Australia Agree to Collaborate in the Global Translation of Stem Cell Research
The Centre for Commercialization of Regenerative Medicine (CCRM) and the Stem Cells Australia consortium have entered into a memorandum of understanding to work together to increase the translation of stem cell research in the two countries. [Stem Cells Australia] Press Release

Celsion Corporation to Acquire EGEN, Inc.
Celsion Corporation and EGEN, Inc. announced the signing of a definitive asset purchase agreement in which Celsion will acquire substantially all of the assets of EGEN, including its Phase Ib DNA-based immunotherapy product candidate EGEN-001 and its therapeutic platform technologies, TheraPlas™ for delivery of DNA and mRNA, TheraSilence™ for delivery of RNA, and RAST™ for Cell Enabled Expression and Secretion of RNA. [Celsion Corporation] Press Release

Aratana Therapeutics Expands Pain Franchise with Novel Regenerative Cell Therapy for Treating Osteoarthritis
Aratana Therapeutics, Inc. announced it has entered into an exclusive license agreement with Vet-Stem, Inc., for its novel, allogeneic stem cell therapy technology. [Aratana Therapeutics, Inc.] Press Release

Grant Supports Innovative Immunotherapy Research at Baylor
Baylor College of Medicine, Texas Children’s Cancer Center and Center for Cell and Gene Therapy researcher Dr. Stephen Gottschalk has received a $250,000 Innovation Award from Alex’s Lemonade Stand Foundation to support his work to find innovative new treatments for childhood cancer. Gottschalk’s Innovation Award will support his research on cancer treatments using immunotherapy. [Baylor College of Medicine] Press Release

Response Genetics Launches New Test for Cancer Immunotherapy Drug Development
Response Genetics, Inc. announced the availability of new testing capabilities to advance cancer immunotherapy clinical development. The Immuno-Oncology assay, which runs on HTG Molecular’s Edge System, is designed to measure the RNA expression of 26 commonly investigated immunotherapy related genes enabling screening for response to immunoregulatory pathways. [GlobeNewswire, Inc.] Press Release

Alnylam Announces United States Patent and Trademark Office (USPTO) Issues Patent Covering RNAi Therapeutics for the Treatment of Hepatitis B Virus (HBV) Infection
Alnylam Pharmaceuticals, Inc. announced that the USPTO has issued a new patent (U.S. patent no. 8,618,277) in the company’s McSwiggen patent estate. The McSwiggen patent estate broadly describes chemical modifications of RNAi therapeutics needed to achieve “drug-like” properties in siRNA, the molecules that mediate RNAi. [Alnylam Pharmaceuticals, Inc.] Press Release

TxCell Receives Certificate of GMP Compliance for Cell Therapy Manufacturing Facility in France
TxCell SA announced that it has been granted Certificate of Good Manufacturing Practice (GMP) compliance for its cell therapy manufacturing facility in Besançon, France. [TxCell SA] Press Release

Quest PharmaTech Completes Patient Enrollment in Phase IIb Study of Oregovomab for the Immunotherapy of Ovarian Cancer
Quest PharmaTech Inc. reports that patient enrollment has been completed in the company’s Phase IIb clinical trial of its lead immunotherapy product candidate oregovomab for the treatment of ovarian cancer. [Quest PharmaTech Inc.] Press Release

Inovio Pharmaceuticals Initiates Immune Therapy Trial for Head & Neck Cancer Caused by HPV
Inovio Pharmaceuticals, Inc. announced it has initiated a Phase I/IIa clinical trial to evaluate safety, immunogenicity and clinical responses of its immunotherapy product, INO-3112, in treating human papillomavirus (HPV)- associated head and neck cancer. [Inovio Pharmaceuticals, Inc.] Press Release
National Institute of General Medical Sciences; Notice of Closed Meeting (FR Doc. No: 2014-13681)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2014-13673)
CBER Is Moving
Food and Drug Administration (United States)

Draft Guidance for Industry on Abbreviated New Drug Application Submissions; Content and Format of Abbreviated New Drug Applications; Availability (FR Doc. No: 2014-13641)

Agency Information Collection Activities; Proposed Collection; Comment Request; Good Laboratory Practice Regulations for Nonclinical Studies (FR Doc. No: 2014-13787)

Draft Guidance for Industry on Distributing Scientific and Medical Publications on Risk Information for Approved Prescription Drugs and Biological Products-Recommended Practices; Availability (FR Doc. No: 2014-13569)

Postmarketing Safety Reports for Human Drug and Biological Products; Electronic Submission Requirements (FR Doc. No: 2014-13480)

European Medicines Agency (European Union)

European Medicines Agency Agrees Policy on Publication of Clinical Trial Data with More User-Friendly Amendments
NEW South Central Association of Blood Banks & Association of Donor Recruitment Professionals Joint Educational Symposium
July 18, 2014
Fort Worth, United States

NEW Get Connected 4: Cellular Microenvironment
September 10-12, 2014
Manchester, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Scientist – Immune Focused In Vitro Analytics (Celentyx Ltd.)

NEW Assistant or Associate Professor – Stem Cell Transplantation Program (Dana-Farber Cancer Institute)

PhD Studentship – Endogenous Retroviruses as Immunotherapy Targets (University of Plymouth)

Postdoctoral Fellow – Prostate Cancer Research (Henry Ford Hospital)

Cell Biologists – T Cell-Based Therapies (Adaptimmune)

Postdoctoral/Clinical Pharmacist – Hematopoiesis and Laboratory for Cell Therapy (Sanquin)

Postdoctoral Position – Neurotoxicity and Epigenetics (Thomas Jefferson University)

Research/Review Scientist – Cell Therapy and Regenerative Medicine (Center for Biologics Evaluation and Research)

Postdoctoral Position – Cell Death and Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Position – Role of Calreticulin in TGF-Beta Signaling in the Kidney Proximal Tubule under Diabetic Conditions (University of Alabama)

Postdoctoral Fellow – Transgene Immunity in AAV Vector-Based Antiviral and/or Gene Therapeutics (University of Massachusetts)

Postdoctoral Fellow – Neurophysiology of Addiction (Medical University of South Carolina)

Principal Investigator – Molecular Mechanisms of Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Associate – Hematopoietic Differentiation (Northwestern University Feinberg School of Medicine)

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

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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

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

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