Volume 15.33 | Aug 25

Issue 15.33 August 25, 2014
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Marburg Virus Infection in Nonhuman Primates: Therapeutic Treatment by Lipid-Encapsulated siRNA
Investigators assessed the efficacy of lipid nanoparticle delivery of anti-Marburg virus (MARV) nucleoprotein-targeting small interfering RNA (siRNA) at several time points after virus exposure, including after the onset of detectable disease in a uniformly lethal nonhuman primate model of MARV-Angola hemorrhagic fever. [Sci Transl Med] Full Article | Press Release
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PUBLICATIONS (Ranked by impact factor of the journal)
A Selective MicroRNA-Based Strategy Inhibits Restenosis while Preserving Endothelial Function
Scientists developed a microRNA-based approach to inhibit proliferative vascular smooth muscle cells, thus preventing restenosis, while selectively promoting reendothelialization and preserving endothelial cell function. [J Clin Invest] Full Article | Press Release

Safety and Efficacy of Ixmyelocel-T: An Expanded, Autologous Multi-Cellular Therapy in Dilated Cardiomyopathy
Researchers evaluated the safety and efficacy of ixmyelocel-T in two prospective randomized Phase IIa trials administered via mini-thoracotomy or intramyocardial catheter injections in patients with dilated cardiomyopathy stratified by ischemic or non-ischemic status. [Circ Res] Abstract | Press Release

Successful Transplantation of Kidney Allografts in Sensitized Rats after Syngeneic Hematopoietic Stem Cell Transplantation and Fludarabine
The authors tested novel approaches to desensitization using an animal model of allogeneic sensitization with skin grafts from dark agouti (DA) to Lewis rats. At the peak IgG alloantibody response they transplanted DA kidneys into nephrectomized Lewis recipients and all died within 10 days from antibody-mediated rejection. Allogeneic hematopoietic stem cell transplants from DA donors failed to engraft after lethal or sub-lethal irradiation. [Am J Transplant] Abstract

Haploidentical Hematopoietic Stem Cell Transplantation in Adults with Philadelphia-Negative Acute Lymphoblastic Leukemia: No Difference in the High- and Low-Risk Groups
Researchers evaluated the outcomes of haploidentical hematopoietic stem cell transplantation in adult Philadelphia chromosome-negative acute lymphoblastic leukemia in first complete remission patients. [Int J Cancer] Abstract

Transplantation of Mesenchymal Stem Cells into the Renal Medulla Attenuated Salt-Sensitive Hypertension in Dahl S Rat
Investigators detected a stem cell deficiency in the renal medulla in Dahl salt-sensitive (S) rats and then tested the hypothesis that transplantation of mesenchymal stem cells into the renal medulla improves salt-sensitive hypertension in Dahl S rats. [J Mol Med (Berl)] Abstract

Effect of FGF-2 on Collagen Tissue Regeneration by Human Vertebral Bone Marrow Stem Cells
Collagen formation was examined using an in vivo assay model by transplanting human bone marrow stem cells (hBMSCs) into immunocompromised mice and the histologic and immunohistochemical results revealed that significantly more collagen with a well-organized structure was formed by fibroblast growth factor-2 (FGF-2)-treated hBMSCs at eight weeks posttransplantation. [Stem Cells Dev] Abstract

Transplantation of Neural Stem Cells Clonally Derived from Embryonic Stem Cells Promotes Recovery of the Injured Mouse Spinal Cord
Researchers generated clonally derived definitive neural stem cells (dNSCs) from embryonic stem (ES) cells. These cells were transplanted into a mouse thoracic spinal cord injury model. ES-dNSC-transplanted mice were compared to controls using behavioral measures and histopathological analysis of tissue. [Stem Cells Dev] Abstract

MicroRNA-Mediated Multi-Tissue Detargeting of Oncolytic Measles Virus
Researchers report the combination of microRNA target sites for different microRNAs in a single vector to detarget pivotal organs at risk during systemic administration (liver, brain, gastrointestinal tract). [Cancer Gene Ther] Abstract

Homing Effect of Adipose-Derived Stem Cells to the Injured Liver: The Shift of Stromal Cell-Derived Factor 1 Expressions
Whether systemically transplanted human adipose-derived stem cells homed to the injured liver in nude mice under stress with subsequent hepatectomy and ischemia-reperfusion was investigated. [J Hepatobiliary Pancreat Sci] Full Article

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Regenerative Medicine for the Heart: Perspectives on Stem-Cell Therapy
The authors examine the potential sources of cells that could be used for heart therapies, including embryonic stem cells and induced pluripotent stem cells, as well as alternative methods for activating the endogenous regenerative mechanisms of the heart via transdifferentiation and cell reprogramming. [Antioxid Redox Signal] Abstract

Recent Advances of Stem Cell Therapy for Retinitis Pigmentosa
The authors provide an overview of stem cell-based treatment of retinitis pigmentosa and its main problems, to provide evidence for the safety and feasibility for further clinical treatment. [Int J Mol Sci]
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Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

Take the "if" out of "diff": Reduce variability when differentiating to hematopoietic cells
Osteoarthritis Cell Therapy, HiQCell, Shows Continuing Significant Patient Improvements at Two Years Post-Treatment
Regeneus announced further results from the clinical research study that is tracking the safety and effectiveness of its innovative cell therapy, HiQCell, in the treatment of osteoarthritis. [Regeneus] Press Release

Baxter Announces Positive Top-Line Results from Its Phase III Study of BAX 855, Extended Half-Life Recombinant FVIII for Hemophilia A Patients
Baxter International Inc. announced positive results from its Phase III pivotal clinical trial of BAX 855, an investigational, extended half-life recombinant factor VIII (FVIII) treatment for hemophilia A based on ADVATE [Antihemophilic Factor (Recombinant)], which met its primary endpoint in reducing annualized bleed rates in the prophylaxis arm compared to the on-demand arm. [Baxter International Inc.] Press Release

BioProcess International Conference & Exhibition - Boston MA
Iranian Parliament Ousts Reform-Minded Science Minister
A monthslong effort to breathe new life into Iranian universities is at a crossroads after the ouster of the nation’s reformist science minister, Reza Faraji-Dana. [ScienceInsider] Editorial

Flow of Chinese Grad Students to U.S. Slows
For years, U.S. university administrators have worried that China’s massive investment in higher education would eventually mean fewer Chinese students seeking to earn advanced science and engineering degrees at their institutions. A new survey from the Council of Graduate Schools hints that the time may be approaching: For the second straight year, graduate applications from Chinese students are essentially flat. [ScienceInsider] Editorial

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Regeneus and Cryosite Partner for the Development and Manufacture of Human Stem Cell Products
Regeneus and Cryosite announced that they have signed an agreement to collaborate on the development and manufacture of new stem cell products for human applications. [Regeneus] Press Release

Bristol-Myers Squibb and Celgene Enter Clinical Collaboration Agreement to Evaluate Immunotherapy and Chemotherapy Combination Regimen
Bristol-Myers Squibb Company and Celgene Corporation announced the establishment of a clinical trial collaboration to evaluate the safety, tolerability and preliminary efficacy of a combination regimen of Bristol-Myers Squibb’s investigational PD-1 immune checkpoint inhibitor, OPDIVO, and Celgene’s nab® technology-based chemotherapy ABRAXANE®, in a Phase I study. [Bristol-Myers Squibb Company] Press Release

ViaCyte, Inc. Announces Signing of Rights Agreement with Janssen Research & Development LLC.
ViaCyte, Inc. announced that it has entered into a Rights Agreement with Janssen Research & Development LLC. The agreement provides Janssen with a future right to evaluate a transaction related to the VC-01™ combination product that ViaCyte is developing for type 1 diabetes. [ViaCyte, Inc.] Press Release

Gamida Cell Announces Investment and Option Agreement with Major Pharmaceutical Company
Gamida Cell announced that it has signed an investment and option agreement with Novartis Pharma AG. According to the agreement, Novartis will invest $35 million in Gamida Cell and in return will receive 15% equity and an option to fully acquire Gamida Cell. The option is exercisable for a limited period of time following achievement of certain milestones connected to the development of NiCord®. [Gamida Cell] Press Release

Penn Medicine Opens Center for Advanced Retinal and Ocular Therapeutics (CAROT)
The Perelman School of Medicine at the University of Pennsylvania has launched the CAROT to build on its previous success developing novel therapies for the personalized diagnosis and treatment of retinal and ocular disorders. [University of Pennsylvania] Press Release

Alnylam Receives Notice of Allowance from United States Patent and Trademark Office (USPTO) for New Patent Broadly Covering RNAi-Mediating Double-Stranded Molecules Comprising Up to 25 Base Pairs
Alnylam Pharmaceuticals, Inc. announced that the USPTO has issued a Notice of Allowance for claims in the Tuschl et al. patent application 13/725,262. The ‘262 patent application includes newly allowed claims directed to compositions that mediate RNAi, and comprising a double-stranded molecule with up to 25 base pairs and at least one nucleotide analogue. [Alnylam Pharmaceuticals, Inc.] Press Release

CEL-SCI Corporation Moves Ahead with Phase III Immunotherapy Head and Neck Cancer Trial
CEL-SCI Corporation announced its Phase III Head and Neck Cancer clinical trial of its investigational cancer immunotherapy treatment Multikine (Leukocyte Interleukin, Injection) has added Centre Hospitalier Universitaire de Québec’s L’Hotel Dieu de Quebec to its growing number of clinical sites in North America. [CEL-SCI Corporation] Press Release

BARDA Executes Contract Option with Cytori for Continued Development of Thermal Burn Injury Counter Measure
Cytori Therapeutics and Biomedical Advanced Research and Development Authority (BARDA) announced the execution of a contract option to fund the continued investigation and development of Cytori Cell Therapy for use in thermal burn injuries. [Cytori Therapeutics Inc.] Press Release

Cryo-Cell Provides Funding for Cord Blood Clinical Trial Application to Treat ALS
Cryo-Cell International, Inc. announced that it has provided funding to complete an Investigational New Drug Application for a FDA approved Phase I clinical trial for patients that have amyotrophic lateral sclerosis (ALS) disease using umbilical cord blood cells. [Cryo-Cell International, Inc.] Press Release
National Institute on Deafness and Other Communication Disorders; Notice of Closed Meetings (FR Doc. No: 2014-20007)

National Institute of Mental Health; Notice of Meeting (FR Doc. No: 2014-19636)
2014 Meeting Materials, Cellular, Tissue and Gene Therapies Advisory Committee

Proposed Rule: Revocation of General Safety Test Regulations that Are Duplicative of Requirements in Biological License Applications
Food and Drug Administration (United States)

Revocation of General Safety Test Regulations That Are Duplicative of Requirements in Biological License Applications (FR Doc. No: 2014-19888)

Evaluation of Sex-Specific Data in Medical Device Clinical Studies; Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No: 2014-19939)

Reopening of Docket and Request for Comments on the Food and Drug Administration Safety and Innovation Act Action Plan (FR Doc. No: 2014-19881)
NEW Use of iPSC as Screening Tools and Therapeutics
September 11, 2014

NEW New York Stem Cell Foundation (NYSCF) 9th Annual Translational Stem Cell Research Conference
October 22-23, 2014
New York City, New York

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Associate Research Fellow – Cell Therapy Manufacturing Process (Pfizer, Inc.)

Research Associate – Gene Therapy Center (University of Massachusetts)

Postdoctoral Fellow – Induced Pluripotent Stem Cell Based Therapeutics (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – New Combination Immune Therapies for Cancer (Massachusetts General Hospital)

Postdoctoral Position – Scaffolding and Drug Delivery for Regenerative Medicine Applications (Ospedale Niguarda)

Technical Account Manager (GlobalStem)

Research Scientist – iPSC Biology and Genome Editing (Stem Cell Theranostics)

Research Assistant – iPSC Biology and Genome Editing (Stem Cell Theranostics)

Postdoctoral Researcher – Generation of Myocardium In Situ (Van Andel Research Institute)

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.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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