Cell Therapy News Volume 14.46 | Nov 25 2013

Platelet-Targeted Gene Therapy with Human Factor VIII Establishes Hemostasis in Dogs with Hemophilia A
As activated blood platelets mediate the primary response to vascular injury, researchers hypothesized that storage of coagulation Factor VIII within platelets may provide a locally inducible treatment to maintain hemostasis for hemophilia A. [Nat Commun] Full Article

Inhibition of Rac1 Activity by Controlled Release of NSC23766 from Chitosan Microspheres Effectively Ameliorates Osteoarthritis Development In Vivo
Scientists investigated the role and mechanistic pathway of Rac1 involvement in pathological changes of osteoarthritis (OA) chondrocytes in vitro and OA development in vivo, as well as developed a strategy of modulating Rac1 activity for OA treatment. [Ann Rheum Dis] Abstract

Stem Cell Derived Endochondral Cartilage Stimulates Bone Healing by Tissue Transformation
The authors used a translational murine model of a segmental tibia defect to test the clinical utility of bone regeneration from a cartilage graft. They further tested the mechanism by which cartilage promotes bone regeneration using in vivo lineage tracing and in vitro culture experiments. [J Bone Miner Res] Abstract

Evaluation of Engraftment and Immunological Tolerance after Reduced Intensity Conditioning in a Rhesus Hematopoietic Stem Cell Gene Therapy Model
Researchers evaluated reduced intensity conditioning transplantation with 4Gy total body irradiation in two rhesus macaques that received equal parts of CD34⁺ cells transduced with green fluorescent protein-expressing lentiviral vector and empty vector not expressing transgenes. [Gene Ther] Abstract

Suppression of the Immune System as a Critical Step for Bone Formation from Allogeneic Osteoprogenitors Implanted in Rats
Using immunohistological techniques in a rat ectopic implantation model, scientists demonstrated that allogeneic osteoprogenitors mount a T cell- and B cell-mediated immune response resulting in an absence of in vivo bone formation. [J Cell Mol Med] Full Article

Intracavernous Delivery of Clonal Mesenchymal Stem Cells Restores Erectile Function in a Mouse Model of Cavernous Nerve Injury
Scientists investigated the effectiveness of mouse clonal bone marrow-derived stem cells obtained from a single colony by using subfractionation culturing method for erectile function in a mouse model of cavernous nerve injury. [J Sex Med] Abstract

Progranulin, a Major Secreted Protein of Mouse Adipose-Derived Stem Cells, Inhibits Light-Induced Retinal Degeneration
Investigators examined the protective effects of adipose-derived stem cells (ASCs) and ASC-conditioned medium (ASC-CM) against retinal damage and identified the neuroprotective factors in ASC-CM. [Stem Cells Transl Med] Abstract | Press Release

Towards Reaching the Target: Clinical Application of Mesenchymal Stem Cell (MSC) for Diabetic Foot Ulcers (DFUs)
The authors review the potential efficacy and systematic use of MSCs for the treatment of non-healing DFUs, current advances, MSC delivery systems and possible options to enhance the therapeutic potential of stem cell for wound healing. [Rejuvenation Res] Abstract

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

Bone Marrow Mononuclear Stem Cells Show No New Gains in Heart Function Says TIME Study
New data showed that the use of bone marrow mononuclear stem cells did not improve heart function significantly more at one year than at six months. While there was measurable decrease in the size of scar tissue at six and 12 months, stem cells administered as a part of the TIME (Transplantation In Myocardial Infarction Evaluation) study did not improve overall heart functionality. [Press release from the Cardiovascular Cell Therapy Research Network discussing research presented at the 2013 Scientific Sessions of the American Heart Association, Dallas] Press Release

Communist Party Takes Aim at Elite Scientists
Long considered the capstone of a scientific career in China, election to the Chinese Academy of Sciences (CAS) confers so much prestige on those anointed that organizations often try to recruit CAS members or reward their own with a guarantee of lifetime employment. But China’s Communist Party has ordered a reform of China’s academy membership system that may be aimed at curtailing privileges for academicians, or yuanshi. [ScienceInsider] Editorial

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Life Stem Genetics Announces Collaborative Agreement with American CryoStem
Life Stem Genetics, Inc. announced a strategic collaborative agreement with American CryoStem Corporation (CRYO). CRYO is a leading developer, marketer and global licensor of patented adipose tissue-based cellular technologies for the Regenerative and Personalized Medicine industries. [Business Wire] Press Release

Actinium Pharmaceuticals Announces Plans for Iomab™-B Phase III Pivotal Trial Following Meeting with FDA
Actinium Pharmaceuticals, Inc. a biopharmaceutical company developing targeted payload immunotherapeutics for the treatment of advanced cancers, provided a corporate update on its two most advanced clinical programs. Kaushik J. Dave Ph.D., MBA, President and Chief Executive Officer discussed recent progress and outline development plans for the company’s clinical stage products: Iomab™-B and Actimab™-A. [Actinium Pharmaceuticals, Inc.] Press Release

Alnylam Earns $7 Million Milestone Payment from Genzyme for Phase II Success with Patisiran (ALN-TTR02), an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR)
Alnylam Pharmaceuticals, Inc. announced that it has earned a $7 million milestone from its partner Genzyme, a Sanofi company, for achieving Phase II success with patisiran (ALN-TTR02). [Alnylam Pharmaceuticals]
Press Release

ARMO BioSciences Initiates Phase I Clinical Trial of First-in-Class Cancer Immunotherapy
ARMO BioSciences, Inc. announced that it initiated the first cohort of patients with advanced solid tumors in a Phase I clinical trial of AM0010, a PEGylated form of recombinant human interleukin-10. [PR Newswire Association LLC] Press Release

Alliqua Receives License to Celgene Cellular Therapeutics Advanced Wound Care Products
Alliqua, Inc. entered into a licensing agreement with Celgene Cellular Therapeutics, a subsidiary of Celgene Corporation, whereby Alliqua received the right to develop and market the advanced wound care products Biovance® and Extracellular Matrix. [Alliqua Inc.] Press Release

Leading Scientists Join BioResearch Open Access Editorial Board under Editor-in-Chief Robert Lanza
Dr. Robert Lanza announced that in addition to the distinguished group of Section Editors, including Alan Russell, James Wilson, and Sir Ian Wilmut, among others, fifteen world renowned experts have joined the new Editorial Board of BioResearch Open Access, including ten members of the National Academy of Sciences and Institute of Medicine. [Mary Ann Liebert, Inc.] Press Release

Allergen Research Corporation Announces $17 Million Series A Financing
Allergen Research Corporation announced the successful completion of financing to support its upcoming Phase IIb clinical trial to evaluate peanut allergy oral immunotherapy with characterized peanut allergen, as well as related development projects. [Allergen Research Corporation] Press Release

Regeneus to Fast-Track Human Cells under New Japanese Laws
The Japanese parliament has passed new laws that provide a rapid approval process specifically designed for human stem cell therapies. Regenerative medicine company, Regeneus, is ready to move on the opportunity. These new laws give the company a unique opportunity to fast-track the clinical trial and potential approval of its new human “off-the-shelf” cell therapy to treat osteoarthritis and other inflammatory musculoskeletal conditions into the Japanese market. [Regeneus Ltd.] Press Release

New Japanese Regenerative Medicine Legislation and Commercial Opportunities for Stem Cell Products
Regenerative medicine company Mesoblast Limited gave an update on new legislation which provides a framework for accelerated approval of stem cell products in Japan, the world’s second-largest mature healthcare market. [Mesoblast Limited] Press Release

Prospective Grant of Exclusive License: The Development of Modified T-Cells for the Treatment of Multiple Myeloma (FR Doc No: 2013-27601)

Prospective Grant of Exclusive License: Development of Chitosan/IL-12 Conjugate as Immunotherapeutic Products for Human Cancers (FR Doc No: 2013-28119)

Draft Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Extension of Comment Period (FR Doc No: 2013-27769)

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NEW Postdoctoral Fellow – Liver Tissue Engineering (Harvard Medical School)

Research Associate – Stem Cells & Regenerative Medicine (King’s College London)

Assistant Director – Regenerative Medicine Clinical Center (Wake Forest Institute for Regenerative Medicine)

Postdoctoral Positions – Stem Cell, Cancer Biology, Immunology and Bioengineering (UC Irvine)

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

Postdoctoral Position – Stem Cells Research (University of Maryland Baltimore)

Postdoctoral Research Fellow – Stem Cells (Bioprocessing Technology Institute)

Scientist – Particle Chemistry (STEMCELL Technologies Inc.)

Research Technologist – Human Pluripotent Stem Cell Products (STEMCELL Technologies Inc.)

Research Associate/Scientist – Antibodies Group (STEMCELL Technologies Inc.)

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