Volume 16.26 | Jul 6

Issue 16.26 July 6, 2015
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Repeated Nebulization of Non-Viral CFTR Gene Therapy in Patients with Cystic Fibrosis: A Randomized, Double-Blind, Placebo-Controlled, Phase IIb Trial
Lung delivery of plasmid DNA encoding the CFTR gene complexed with a cationic liposome is a potential treatment option for patients with cystic fibrosis. Investigators assessed the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis. [Lancet Resp Med] Full Article | Press Release
Get Fresh Human Peripheral Whole Blood and Leuko Paks, Human Primary Cells
PUBLICATIONS (Ranked by impact factor of the journal)
Allogeneic Bone Marrow-Derived Mesenchymal Stromal Cells Promote Healing of Refractory Perianal Fistulas in Patients with Crohn’s Disease
Researchers evaluated the effects of local administration of bone marrow-derived mesenchymal stromal cells to these patients from healthy donors in a double-blind, placebo-controlled study. [Gastroenterology] Abstract

Interleukin-17A-Induced Human Mesenchymal Stem Cells Are Superior Modulators of Immunological Function
Interferon-γ-preactivated mesenchymal stem cells (MSC) are highly immunosuppressive but immunogenic in vivo due to their inherent expression of major histocompatibility molecules. The authors present an improved approach where they modified human bone marrow-derived MSC with interleukin-17A to enhance T cell immunosuppression but not their immunogenicity. [Stem Cells] Abstract | Press Release

Tightly Regulated ‘All in One’ Lentiviral Vectors for Protection of Human Hematopoietic Cells from Anticancer Chemotherapy
Investigators describe a doxycycline-inducible “all-in-one” lentiviral vector design employing the pTET-T11 minimal-promoter and a reverse transactivator protein driven by the phosphoglycerate kinase promoter allowing for tight regulation of transgene expression. [Gene Ther] Abstract

Prediction of Adeno-Associated Virus Neutralizing Antibody Activity for Clinical Application
To develop a standard assay for detecting neutralizing antibodies (Nab) inhibition activity, researchers systematically studied current adeno-associated virus Nab assays in vitro and in vivo. [Gene Ther] Abstract

Three Dimensional Multi-Cellular Muscle-Like Tissue Engineering in Perfusion-Based Bioreactors
The authors investigated the influence of different serum supplementations on the skeletal myoblast ability to proliferate and differentiate during three-dimensional perfusion-based culture. They tested percentages of serum promoting monolayer skeletal myoblast- proliferation and differentiation and suitable for stromal cell culture with a view to identify the most suitable condition for the subsequent co-culture. [Biotechnol Bioeng] Abstract

3D-Printed Bioresorbable Scaffold for Periodontal Repair
Researchers provide the first reported human case of treatment of a large periodontal osseous defect with a 3D-printed bioresorbable patient-specific polymer scaffold and signaling growth factor. [J Dent Res]
Full Article | Press Release

Neutrophil Gelatinase Associated Lipocalin Is an Early and Accurate Biomarker of Graft Function and Tissue Regeneration in Kidney Transplantation from Extended Criteria Donors
NGAL is a 25KDa protein proposed as biomarker of acute kidney injury. Investigators evaluated plasma levels of NGAL in 50 kidney transplantation patients from extended criteria donors in the first four days after surgery or after Tacrolimus introduction. [PLoS One] Full Article

Improved Structure and Function in Autosomal Recessive Polycystic Rat Kidneys with Renal Tubular Cell Therapy
Using PCK female rats, an orthologous model of autosomal recessive polycystic kidney disease harboring mutant Pkhd1, researchers tested the hypothesis that intravenous renal cell transplantation with normal Sprague Dawley male kidney cells would improve the polycystic kidney disease phenotype. [PLoS One] Full Article

Preclinical Validation of Multilevel Intraparenchymal Stem Cell Therapy in the Porcine Spinal Cord
Although multiple clinical trials are currently testing different stem cell therapies as treatment alternatives for many neurodegenerative diseases and spinal cord injury, the optimal injection parameters have not yet been defined. Scientists tested the spinal cord’s tolerance of increasing volumes and numbers of stem cell injections in the pig. [Neurosurgery] Abstract

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Recent Therapeutic Approaches for Spinal Cord Injury
The authors highlight clinical trials and recent encouraging strategies for spinal cord repair comprising both cell therapy and nanomedicine. [Biotechnol Bioeng] Abstract

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

Only One of These Antibodies Will Work for Your Cell Analysis. We Take the Guesswork Out of Finding the Right
Navidea Reports Clinical Data Demonstrating Manocept™ Agent Localizes in Multiple KS Lesions
Navidea Biopharmaceuticals, Inc. and its subsidiary, Macrophage Therapeutics, Inc. announced that imaging results from the Manocept™ clinical trial in Kaposi’s Sarcoma (KS) and other preclinical studies were presented. [Press release from Navidea Biopharmaceuticals, Inc. discussing research presented at the at the 18th International Workshop on Kaposi’s Sarcoma Herpesvirus (KSHV) and Related Agents, Hollywood] Press Release

Alnylam Reports New Positive Clinical Data for ALN-AT3, a Subcutaneously Administered, Investigational RNAi Therapeutic Targeting Antithrombin (AT) for the Treatment of Hemophilia and Rare Bleeding Disorders
Alnylam Pharmaceuticals, Inc. announced that new positive data from its ongoing Phase I clinical trial with ALN-AT3 – an investigational RNAi therapeutic for the treatment of hemophilia and rare bleeding disorders. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress, Toronto] Press Release

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US Vaccine Researcher Sentenced to Prison for Fraud
Dong-Pyou Han, a former biomedical scientist at Iowa State University in Ames, was sentenced to 57 months for fabricating and falsifying data in HIV vaccine trials. Han has also been fined $7.2 million and will be subject to three years of supervised release after he leaves prison. [Nature News] Editorial

Greek Scientists Lose Access to Digital Journals
The Internet portal that provides scientists in many Greek universities and research institutes with access to electronic journals from 27 publishers suspended many of its services because the government has not provided the funds to keep it going. [Nature News] Editorial
AMAG Pharmaceuticals to Acquire Cord Blood Registry
AMAG Pharmaceuticals, Inc. announced that it has entered into a definitive agreement to acquire Cord Blood Registry®, the world’s largest stem cell collection and storage company serving pregnant women and their families, for $700 million. [Cord Blood Registry®] Press Release

Amarna Therapeutics Teaming Up with Austrian CBmed in the Development of Immunotherapies
Amarna Therapeutics announced that it has entered into a cooperation with Austrian Center for Biomarker Research in Medicine (CBmed), for the development of immunotherapies against acute lymphoblastic leukemia, obesity, diabetes mellitus type 2 and atherosclerotic cardiovascular disease. [Amarna Therapeutics] Press Release

Biogen and AGTC Enter Collaboration to Develop Gene Therapies in Ophthalmology
Biogen and AGTC announced a broad collaboration and license agreement to develop gene-based therapies for multiple ophthalmic diseases. The collaboration will focus on the development of a portfolio of AGTC’s therapeutic programs, including both a clinical stage candidate and a pre-clinical candidate for orphan diseases of the retina that can lead to blindness in children and adults. [Biogen] Press Release

STEMCELL Technologies Launches Innovative Products to Support CAR T Cell Research
STEMCELL Technologies Inc. announced the launch of ImmunoCult™ Human T Cell Activator reagents and ImmunoCult™-XF T Cell Expansion Medium to support T cell researchers in their goal to develop CAR T cell immunotherapeutics. The products provide significant benefits compared to existing technologies. [STEMCELL Technologies Inc.] Press Release

CTCA at Western Launches 3rd New Clinical Trial Combining Immunotherapy with Chemotherapy
Cancer Treatment Centers of America® (CTCA) at Western Regional Medical Center, has begun a new Phase Ib/II clinical trial using a new immunotherapy treatment for patients with advanced kidney, non-small cell lung cancer, pancreatic cancer and colorectal carcinoma. [Cancer Treatment Centers of America® (EurekAlert!)] Press Release

TxCell Announces FDA Acceptance of IND for Ovasave
TxCell SA announced that the United States Food and Drug Administration (FDA) has accepted TxCell’s Investigational New Drug (IND) application for the company’s lead product, Ovasave®, currently in a Phase IIb clinical trial for the treatment of patients with refractory Crohn’s disease. [TxCell SA] Press Release

Exelixis Provides Update on Genentech’s Pending New Drug Application for Cobimetinib, an Exelixis-Discovered Compound
Exelixis’ partner Genentech informed Exelixis that, in order to accommodate its review of a supplemental data submission, the U.S. Food and Drug Administration has extended the Prescription Drug User Fee Act action date for its review of Genentech’s New Drug Application for cobimetinib by the standard extension period of three months. [Exelixis, Inc.] Press Release

R-Japan Obtained Autologous Stem Cell Manufacturing License from the Ministry of Health, Labor and Welfare
R-Japan Co., Ltd. obtained the license of cell processing facility under the Act on the Safety of Regenerative Medicine from the Ministry of Health, Labor and Welfare Kinki Bureau of Health and Welfare. [R-Japan Co., Ltd. (PR Newswire Association LLC)] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-16063)
Guidance Agenda: Guidance Documents CBER is Planning to Publish During Calendar Year 2015
Food and Drug Administration (United States)

Revocation of General Safety Test Regulations That Are Duplicative of Requirements in Biologics License Applications (FR Doc. No: 2015-16366)

Removal of Review and Reclassification Procedures for Biological Products Licensed Prior to July 1, 1972 (FR Doc. No: 2015-16367)
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.
NEW Postdoctoral Research Associate – Breast Stem Cell Biology (University of Miami Miller School of Medicine)

NEW Aseptic Manufacturing Associate (Lonza Biologics, Inc.)

NEW Postdoctoral Position – Translational Immunology (University of Utah School of Medicine)

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

Postdoctoral Position – Splicing in Hematopoiesis (Northwestern University)

Primary Cell Transduction Process Scientist (GlaxoSmithKline)

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

Postdoctoral Fellow – Stem Cell Biology (INSERM)

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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