Volume 16.24 | Jun 22

Issue 16.24 June 22, 2015
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Publications | Reviews | Science | Policy | Business | NIH | CBER | Regulatory | Events | Jobs
 
TOP STORY
Gene Therapy Prevents Parkinson’s Disease in Animal Model
Gene therapy to reduce production of a brain protein successfully prevented development of Parkinson’s disease in an animal study. The findings could lead to new understanding of how genetic and environmental factors converge to cause the disease, and the development of effective treatments to prevent disease progression. [Press release from the University of Pittsburgh discussing online publication in Journal of Clinical Investigation] Press Release | Full Article
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PUBLICATIONS (Ranked by impact factor of the journal)
Aligned-Braided Nanofibrillar Scaffold with Endothelial Cells Enhances Arteriogenesis
To test the efficacy of endothelial cell (EC)-seeded aligned nanofibrillar scaffolds in improving neovascularization in vivo, the ischemic limbs of mice were treated with EC-seeded aligned nanofibrillar scaffold; EC-seeded nonpatterned scaffold; ECs in saline; aligned nanofibrillar scaffold alone; or no treatment. [ACS Nano] Abstract

Development and Rescue of Human Familial Hypercholesterolemia in a Xenograft Mouse Model
The authors developed a xenograft mouse model using hepatocytes from a patient with familial hypercholesterolemia caused by loss-of-function mutations in the low-density lipoprotein receptor (LDLR). They replaced the missing LDLR in familial hypercholesterolemia liver chimeric mice using an adeno-associated virus 9-based gene therapy and restore normal lipoprotein profiles after administration of a single dose. [Nat Commun] Full Article

Neural Stem Cell Transplantation at Critical Period Improves Learning and Memory through Restoring Synaptic Impairment in Alzheimer’s Disease Mouse Model
Scientists demonstrated that transplantation of neural stem cells into 12-month-old Tg2576 brains markedly improved both cognitive impairments and neuropathological features by reducing β-amyloid processing and upregulating clearance of β-amyloid, secretion of anti-inflammatory cytokines, endogenous neurogenesis, as well as synapse formation. [Cell Death Dis] Full Article

Regulation of Gamma-Secretase Activating Protein by the 5-Lipoxygenase: In Vitro and In Vivo Evidence
Investigators provide in vitro experimental evidence that 5-Lipoxygenase (5LO) is as an endogenous regulator for γ-secretase activating protein formation, but not for other known γ-secretase modulators, by directly and specifically activating caspase-3. These results were confirmed in vivo by using transgenic mouse models of Alzheimer’s disease in which 5LO level and activity were modulated genetically or pharmacologically. [Sci Rep] Full Article

Preliminary Evaluation of a Bunyavirus Vector for Cancer Immunotherapy
Researchers demonstrated that nonspreading Rift Valley fever virus (NSR)-infected human dendritic cells can activate CD8+ T-cells in vitro, and that prophylactic and therapeutic vaccination of mice with NSR encoding a tumor associated CD8 peptide can control outgrowth of lymphoma cells in vivo. [J Virol] Abstract

Melatonin Protects ADSCs from ROS and Enhances Their Therapeutic Potency in a Rat Model of Myocardial Infarction
Scientists showed evidence that ex vivo pre-treatment with melatonin, an endogenous hormone with newly found anti-oxidative activity, could improve survival and function of adipose tissue derived mesenchymal stem cells (ADSCs) in vitro as well as in vivo. [J Cell Mol Med] Full Article

Autoantibody-Targeted Treatments for Acute Exacerbations of Idiopathic Pulmonary Fibrosis
Investigators conducted a pilot trial to test the hypothesis that autoantibody-targeted therapies may benefit acute exacerbation of idiopathic pulmonary fibrosis patients. [PLoS One] Full Article | Press Release

Expansion and Characterization of Neonatal Cardiac Pericytes Provides a Novel Cellular Option for Tissue Engineering in Congenital Heart Disease
Scientists investigated the antigenic profile, expansion/differentiation capacity, paracrine activity, and pro-angiogenic potential of cardiac pericytes and to assessed their engrafting capacity in clinically certified prosthetic grafts. [J Am Heart Assoc] Abstract

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REVIEWS
Cell Therapy in Muscular Dystrophies: Many Promises in Mice and Dogs, Few Facts in Patients
The authors explain the properties that are required for a cell to be used in cell therapy (CT) of muscular dystrophy. They briefly review most of the cells that were proposed for this CT, and to what extent these properties were met not only in laboratory animals but also in clinical trials. [Expert Opin Biol Ther] Abstract

Cell Therapy for Immunosuppression after Kidney Transplantation
The authors give an overview over cell therapeutic approaches to immunosuppression in clinical kidney transplantation. A focus is on myeloid suppressor cell therapy by mitomycin C-induced cells. [Langenbecks Arch Surg] Abstract

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

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SCIENCE NEWS
Spark Therapeutics to Present with Pfizer Inc. Preclinical Data on Their Lead SPK-FIX Product Candidate for Hemophilia B
Spark Therapeutics announced that it, along with collaboration partner, Pfizer Inc., will present the results of preclinical testing that supports the safety and efficacy of the lead compound in their SPK-FIX hemophilia B program. [Press release from Spark Therapeutics (Globe Newswire) discussing research to be presented at the International Society on Thrombosis and Haemostasis (ISTH), Toronto] Press Release

Avalanche Biotechnologies, Inc. Announces Positive Top-Line Phase IIa Results for AVA-101 in Wet Age-Related Macular Degeneration
Avalanche Biotechnologies, Inc. announced that its Phase IIa clinical study for AVA-101 met its 12-month primary endpoint, based on ophthalmic and systemic safety, demonstrating that AVA-101 was well tolerated with a favorable safety profile in subjects with wet age-related macular degeneration. [Avalanche Biotechnologies, Inc.] Press Release

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POLICY
France Finally Fills Top Science Policy Post
France’s socialist government has finally found someone to run its science and higher education policy. President François Hollande appointed Thierry Mandon as the new state secretary for higher education and research, a post that became vacant when Geneviève Fioraso stepped down for health reasons. [ScienceInsider] Editorial

House Bill Would Give NIH $1.1 Billion More in 2016
The National Institutes of Health (NIH) would receive a $1.1 billion boost in 2016 under a draft measure released by a House of Representatives spending panel. That 3.6% increase, to $31.2 billion, is $100 million more than the president’s request. [ScienceInsider] Editorial
 
BUSINESS
BellBrook Labs Teams Up with UW-Madison to Fight Neurodegenerative Diseases Using Stem Cells and Microfluidics
BellBrook Labs, in collaboration with the University of Wisconsin-Madison, was recently awarded federal funding to develop a microscale system to accelerate discovery of more effective drugs for neurodegenerative diseases such as multiple sclerosis and Parkinson’s disease. [BellBrook Labs] Press Release

TGen and Mayo Clinic Help Launch National Clinical Trials to Combat Advanced Skin Cancer
The Translational Genomics Research Institute (TGen) and Mayo Clinic are helping launch a national clinical trial that will apply the latest in precision medicine to treat advanced melanoma skin cancer. [Translational Genomics Research Institute] Press Release

Adimab Establishes New Collaboration with Kite Pharma for the Discovery of Antibodies for T-Cell Therapies
Adimab, LLC announced a new collaboration with Kite Pharma, Inc. Under the terms of the agreement, Adimab will use its proprietary platform to generate IgGs against multiple targets selected by Kite. [Adimab, LLC] Press Release

Kite Pharma and bluebird bio Announce Strategic Collaboration to Advance Second Generation TCR Cell Therapy Products to Treat HPV-Associated Cancers
Kite Pharma, Inc. and bluebird bio, Inc. announced that they have entered into a collaboration agreement to co-develop and co-commercialize second generation T cell receptor (TCR) product candidates directed against the human papillomavirus type 16 E6 (HPV-16 E6) oncoprotein incorporating gene editing and lentiviral technologies. [Kite Pharma, Inc.] Press Release

Benitec Collaborates with ReNeuron on Cellular Therapies with Combined ddRNAi and Exosome Program
Benitec Biopharma announced the launch of a new exploratory cellular therapy program including exosome-based delivery utilizing the company’s proprietary ddRNAi technology. Entry into these areas have been facilitated by the commencement of a collaboration with stem cell therapeutics company, ReNeuron. [Benitec Biopharma Limited] Press Release

Tekmira Provides Update on TKM-Ebola-Guinea
Tekmira Pharmaceuticals Corporation announced that the Phase II clinical trial of TKM-Ebola-Guinea has reached a predefined statistical endpoint and enrollment has been closed. [Tekmira Pharmaceuticals Corporation ] Press Release

Genticel Receives FDA Clearance of IND Application for U.S. Phase I Clinical Trial of GTL001 (ProCervix), Company’s First-in-Class HPV Therapeutic Vaccine Candidate
Genticel announced that the U.S. Food and Drug Administration (FDA) has cleared Genticel’s Investigational New Drug (IND) application to conduct in the U.S. a Phase I clinical study of GTL001 in patients infected with human papillomavirus (HPV) 16 and/or 18, the two HPV types responsible for 70% of cervical cancer cases. [Genticel] Press Release

GSK to Create Independent Research Institute with Goal of Radically Changing and Improving Medicines Development
GSK announced that it is making a substantial investment to launch the Altius Institute for Biomedical Sciences, an independent, non-profit research institute. GSK expects to capitalize on rapid progress in understanding gene control to select and validate better drug targets, and to accelerate many key aspects of developing new medicines. [GlaxoSmithKline plc.] Press Release

Lonza to Expand Viral Therapy Business with Planned Construction of New Facility in Houston, TX
Lonza announced the planned expansion of its industry-leading viral gene therapy business. Lonza plans to construct a new 100,000 square foot facility for viral and immunotherapy development and manufacturing, complete with a fully segregated fill/finish suite. [Lonza Group Ltd.] Press Release
 
NIH
National Center for Advancing Translational Sciences; Notice of Closed Meeting (FR Doc. No: 2015-15021)

Human Immunodeficiency Virus (HIV) Organ Policy Equity (HOPE) Act Safeguards and Research Criteria for Transplantation of Organs Infected with HIV (FR Doc. No: 2015-15034)
 
CBER
2015 Biological Device Application Approvals
 
REGULATORY
Food and Drug Administration (United States)

Factors to Consider when Making Benefit-Risk Determinations for Medical Device Investigational Device Exemptions; Draft Guidance for Investigational Device Exemption Sponsors, Sponsor-Investigators, and Food and Drug Administration Staff; Availability (FR Doc. No: 2015-14982)

Content and Format of Abbreviated 510(k)s for Early Growth Response 1 Gene Fluorescence In-Situ Hybridization Test System for Specimen Characterization Devices; Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No: 2015-14881)
 
EVENTS
NEW 3rd International Annual Conference of the German Stem Cell Network (GSCN)
September 9-11, 2015
Frankfurt, Germany

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Primary Cell Transduction Process Scientist (GlaxoSmithKline)

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

Postdoctoral Fellow – Antibody Engineering/Immuno-Oncology (California Institute for Biomedical Research)

Postdoctoral Fellow – Stem Cell Biology (INSERM)

MICA Stem Cell Scientist (Medicines and Healthcare Products Regulatory Agency)

Stem Cell Scientist (Medicines and Healthcare Products Regulatory Agency)

Cell Therapy GMP Manager (Paramount Recruitment)

Postdoctoral Fellow – Neural Stem Cells (Children’s Hospital of Philadelphia)

Lead Device and Delivery Scientist (Cell Therapy Catapult)

Postdoctoral Fellow – Immune Reconstitution in Hematopoietic Stem Cell Transplantation (Stanford University)

Postdoctoral Fellow – Immune Tolerance (Stanford University)

Postdoctoral Fellow Positions – iPSC Research and Banking (University of Magna Graecia)

Process Facilitator (Opexa Therapeutics)


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