Volume 16.38 | Oct 19

Issue 16.38 October 19, 2015
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Scientists Test New Gene Therapy for Vision Loss from a Mitochondrial Disease
Researchers developed a novel mouse model for the vision disorder Leber hereditary optic neuropathy and found that they can use gene therapy to improve visual function in the mice. [Press release from National Eye Institute discussing online publication in Proceedings of the National Academy of Sciences] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Successful Arrest of Photoreceptor and Vision Loss Expands the Therapeutic Window of Retinal Gene Therapy to Later Stages of Disease
Investigators evaluated consequences of gene therapy delivered at intermediate stages of disease in a canine model of X-linked retinitis pigmentosa caused by a mutation in the retinitis pigmentosa GTPase regulator gene. [Proc Natl Acad Sci USA] Abstract | Press Release | Video

Nanoparticle-Enhanced Generation of Gene-Transfected Mesenchymal Stem Cells for In Vivo Cardiac Repair
Scientists designed and synthesized molecularly organic-inorganic hybrid hollow mesoporous organosilica nanoparticles based on nano-synthetic chemistry, which are featured with concurrent large pore size over 20 nm, small particulate size, hollow cavity and high dispersity for gene transfection in bone marrow-derived mesenchymal stem cells and subsequent in vivo cardiac repair. [Biomaterials] Abstract

Neural Stem Cells Rescue Cognitive and Motor Dysfunction in a Transgenic Model of Dementia with Lewy Bodies through a BDNF-Dependent Mechanism
Scientists showed that striatal transplantation of neural stem cells (NSCs) into aged α-syn transgenic mice significantly improves performance in multiple cognitive and motor domains. This recovery is associated with NSC expression of brain-derived neurotrophic factor (BDNF), which restores depleted levels and modulates dopaminergic and glutamatergic systems. [Stem Cell Reports] Full Article | Press Release

Nicorandil Enhances the Efficacy of Mesenchymal Stem Cell Therapy in Isoproterenol-Induced Heart Failure in Rats
Researchers investigated whether nicorandil could offer an additional benefit over bone marrow-derived mesenchymal stem cell therapy in isoproterenol-induced myocardial damage and its progression to heart failure in rats. [Biochem Pharmacol] Abstract

Comparative Analysis of Human Mesenchymal Stem Cells Derived from Bone Marrow, Placenta and Adipose Tissue as Sources of Cell Therapy
Investigators characterized mesenchymal stem cells from different sources, including human bone marrow, placenta, and adipose tissue, in terms of the phenotype, surface antigen expression, differentiation ability, proteome reference map and blood flow recovery in a hindlimb ischemic disease model. [J Cell Biochem] Abstract

Generation of Tissue-Engineered Small Intestine Using Embryonic Stem Cell-Derived Human Intestinal Organoids
Scientists investigated the ability of human embryonic stem cells or human intestinal organoids to populate acellular porcine intestinal matrices and artificial polyglycolic/poly L lactic acid scaffolds, and examined the ability of matrix/scaffolds to thrive when transplanted in vivo. [Biol Open] Full Article

Lentivirus-Mediated RNAi Silencing of VEGF Inhibits Angiogenesis and Growth of Renal Cell Carcinoma in a Nude Mouse Xenograft Model
Investigators aimed to construct and screen short hairpin RNA (shRNA) targeting vascular endothelial growth factor (VEGF), and investigate potential values of VEGF-shRNA on angiogenesis and growth in renal cell carcinoma in a xenograft tumor model. [DNA Cell Biol] Abstract

Adenoviral Vector Carrying Glial Cell-Derived Neurotrophic Factor for Direct Gene Therapy in Comparison with Human Umbilical Cord Blood Cell-Mediated Therapy of Spinal Cord Injury in Rat
Researchers examined the efficacy of glial cell-derived neurotrophic factor (GDNF) delivered using an adenoviral vector (AdV-GDNF) and human umbilical cord blood mononuclear cells (UCB-MCs)+AdV-GDNF therapy by conducting behavioral tests on the animals and morphometric studies on the spinal cord, performing immunofluorescence analyses on glial cells, investigating the survival and migration potential of UCB-MCs, and evaluating the expression of the recombinant GDNF gene. [Spinal Cord] Abstract | Press Release

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Gene Therapy Returns to Center Stage
Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an excellent safety record. They provide evidence for the long-sought promise of gene therapy to deliver ‘cures’ for some otherwise terminal or severely disabling conditions. [Nature] Full Article

Stem Cell Microenvironment on a Chip: Current Technologies for Tissue Engineering and Stem Cell Biology
The authors reviewed the various types of microplatforms on which stem cell microenvironments are mimicked. They assigned the various microplatforms to four categories based on their practical uses to assist stem cell biologists in using them for research. [Stem Cells Transl Med] Abstract

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Alnylam Advances New Innovations in RNAi Therapeutics
Alnylam Pharmaceuticals, Inc. announced advancement of innovations in RNAi therapeutics with presentation of scientific data on two new platform technologies. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at 11th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), Leiden] Press Release

SELLAS Life Sciences Reports Promising Top-Line Phase II Data for WT1 Vaccine in Mesothelioma and Acute Myeloid Leukemia Patients
SELLAS Life Sciences Group announced top-line data from the company’s Phase II clinical study of its WT1 cancer vaccine in patients with malignant pleural mesothelioma. [Press release from SELLAS Life Sciences Group (PR Newswire Association, LLC) discussing research to be presented at the 8th International WT1 Conference, Kyoto] Press Release

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UNESCO Panel of Experts Calls for Ban on “Editing” of Human DNA to Avoid Unethical Tampering with Hereditary Traits
At the close of a meeting at UNESCO in Paris, independent experts of the Organization’s International Bioethics Committee published a report “Updating its Reflection on the Human Genome and Human Rights.” In it, the experts argue that “gene therapy could be a watershed in the history of medicine and genome editing is unquestionably one of the most promising undertakings of science for the sake of all humankind.” [UNESCO] Editorial

Cancer Research UK Announces Grand Challenges Competition
A £20 million grant awaits a team of researchers who submit the best proposal for tackling one of seven grand challenges in cancer. The new competition, announced by Cancer Research UK, the giant U.K. research charity, is meant to spur collaborations aimed at exploring untested but promising ideas. [ScienceInsider] Editorial

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Cell Therapy Catapult Signs Major Development Agreement with Asterias Biotherapeutics Inc.
The Cell Therapy Catapult announced the signing of an agreement with Asterias Biotherapeutics Inc. The agreement engages the Cell Therapy Catapult to advance development of a large scale manufacturing processes for AST-VAC2, Asterias’ allogeneic dendritic cell immunotherapy. [Cell Therapy Catapult] Press Release

Generex Announces Memorandum of Understanding in Respect of a Phase II Clinical Trial for the Antigen Express Prostate Cancer Vaccine
Generex Biotechnology Corporation announced that its wholly-owned immunotherapeutics subsidiary, Antigen Express, Inc., has entered into a memorandum of understanding with CompanDX Ltd. to conduct a Phase II clinical trial of the Antigen Express proprietary prostate cancer vaccine. [Generex Biotechnology Corporation] Press Release

Immatics and UTHealth to Collaborate on Cellular Manufacturing for Phase I Clinical Trials
Immatics US, Inc. and The University of Texas Health Science Center at Houston (UTHealth) will collaborate on cellular manufacturing for adoptive cellular therapy clinical trials. [The University of Texas Health Science Center at Houston] Press Release

Incyte and Merck Expand Clinical Collaboration to Include Phase III Study Investigating the Combination of Epacadostat with Keytruda® (Pembrolizumab) as First-Line Treatment for Advanced Melanoma
Incyte Corporation and Merck announced the expansion of the companies’ ongoing clinical collaboration to include a Phase III study evaluating the combination of epacadostat, Incyte’s investigational selective IDO1 inhibitor, with Keytruda® (pembrolizumab), Merck’s anti-PD-1 therapy, as first-line treatment for patients with advanced or metastatic melanoma. [Merck & Co., Inc.] Press Release

HemaCare Committed to Cell Therapy Research through Strategic Agreements
HemaCare Corporation has expanded its growing cell therapy client portfolio through several supply agreements with Cellectis, Dendreon, and Sotio. Under the agreements, HemaCare provides process development support and supplies high-quality cGMP and research-grade biological material for use in cell therapy research and development of commercially available products, including Provenge®. [HemaCare Corporation (Business Wire)] Press Release

German and US Partners Join Forces in Stem Cell Research to Accelerate Development of New Therapies
Researchers at The Scripps Research Institute, and two German institutes, the Center for Integrated Psychiatry Kiel and the Fraunhofer Institute for Molecular Biology and Applied Ecology IME, have announced a partnership to advance the quality control of human stem cells. [The Scripps Research Institute] Press Release

Medifocus Inc. Announces Exclusive Agreement with Major Academic Medical Center to License Technology to Develop Heat-Activated and Tumor-Targeted Immunotherapy and Gene Therapy
Medifocus, Inc. announced that the company has formally entered into an exclusive license agreement with Duke University regarding heat-activated and tumor-targeted immunotherapy and gene therapy. [Medifocus, Inc. (Marketwired L.P.)] Press Release

RXi Pharmaceuticals Bolsters Protection for Its Novel Self-Delivering RNAi Platform with Notice of Allowance from USPTO
RXi Pharmaceuticals Corporation announced that it has been granted a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for both the composition and methods of use of RXi’s self-delivering platform technology. [RXi Pharmaceuticals Corporation] Press Release

OncoQR’s Breast Cancer Vaccine Successfully Completes Pre-Clinical Proof-of-Concept Study
OncoQR ML announced that its breast cancer vaccine candidate OQR200 has successfully completed a proof-of-concept study in non-human primates. The vaccine elicited the expression of very high levels of polyclonal antibodies targeting multiple epitopes on Her2/neu. [OncoQR ML (B3C Group GmbH)] Press Release

Celsion Announces Impressive Preclinical Data for Its GEN-1 IL-12 Immunotherapy in Combination with Avastin® and Doxil® for Ovarian Cancer
Results from the comprehensive studies confirmed remarkable initial GEN-1 + Avastin® findings and show convincingly that GEN-1 when combined with Avastin® and Doxil®, standard of care for platinum resistant patients, demonstrated a greater than 98% reduction in tumor burden when compared to the untreated control group. [Celsion Corporation] Press Release

Human Longevity, Inc. Launches the Health Nucleus, a Comprehensive and Personalized Health Platform for Individuals
Human Longevity, Inc. announced the launch of the company’s first Health Nucleus™, a genomic powered clinical research project that has the potential to transform healthcare. [Human Longevity, Inc.] Press Release

Cell Therapy Ltd. to Submit Heartcel™ for Conditional Marketing Authorization Post Meeting with the European Medicines Agency
Cell Therapy Ltd. announced that, following a pre-submission meeting with the European Medicines Agency, CTL will begin the application process for a conditional marketing authorization for Heartcel™, the first allogeneic stem cell therapy to regenerate the human heart, in Europe. [Cell Therapy Ltd.] Press Release
Proposed Action Under the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (FR Doc. No: 2015-26388)
CBER Expanded Access Submission Receipt Reports
Food and Drug Administration (United States)

Recommendations for Microbial Vectors Used for Gene Therapy; Draft Guidance for Industry; Availability (FR Doc. No: 2015-26108)

Integrated Summary of Effectiveness; Guidance for Industry; Availability (FR Doc. No: 2015-25630)
NEW Stem Cells: Development & Management
November 30-December 2, 2015
Phitsanulok, Thailand

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NEW Postdoctoral Research Fellow/Research Scientist – Biotechnology (Qu Biologics Inc.)

NEW Project Manager – Manufacturing (Qu Biologics Inc.)

Faculty Positions – Stem Cell Biology (University of Notre Dame)

Research Technician – TCR Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Technician – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant/Associate/Full Professor – Biomedical Engineering (Worcester Polytechnic Institute)

Assistant Professor – Stem Cell Biology (University of South Carolina)

Postdoctoral Researcher – Epigenetic Modifiers (Van Andel Research Institute)

Postdoctoral Fellow – Immunotherapy/T Cell Biology (California Institute for Biomedical Research)

Postdoctoral Position – Spinal Cord Injury Research (Indiana University-Purdue University)

Postdoctoral Fellow – HMGA Chromatin Remodeling Proteins in Cancer and Stem Cell Biology (Johns Hopkins University School of Medicine)

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