Cell Therapy News Volume 16.23 | Jun 15 2015

Arylsulfatase A Overexpressing Human iPSC-Derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy
Researchers used metachromatic leukodystrophy patient-derived induced pluripotent stem cells (iPSC) to generate long-term self-renewing neuroepithelial stem cells and astroglial progenitors for cell-based arylsulfatase A replacement. [Mol Ther] Abstract

Efficacy and Tolerability of a GD2-Directed Trifunctional Bispecific Antibody in a Preclinical Model: Subcutaneous Administration Is Superior to Intravenous Delivery
Scientists studied different application routes with respect to therapeutic efficacy and tolerability by using the surrogate trifunctional bispecific antibody Surek (anti-GD2 x anti-murine CD3) and a murine melanoma engineered to express GD2. [Mol Cancer Ther] Abstract

Systemic Gene Delivery following Intravenous Administration of AAV9 to Fetal and Neonatal Mice and Late-Gestation Nonhuman Primates
Investigators evaluated the ability of the AAV9 vector to mediate systemic gene delivery after intravenous administration to perinatal mice and late-gestation nonhuman primates. [FASEB J] Abstract

Human Embryonic Stem Cell-Derived Progenitors Assist Functional Sensory Axon Regeneration after Dorsal Root Avulsion Injury
Scientists showed that sensory functions can be restored in the adult mouse if avulsed sensory fibers are bridged with the spinal cord by human neural progenitor transplants. [Sci Rep] Full Article | Press Release

Generation of Pure Lymphatic Endothelial Cells from Human Pluripotent Stem Cells and Their Therapeutic Effects on Wound Repair
Investigators differentiated human pluripotent stem cells into the lymphatic endothelial cell (LEC) lineage, purified them with LEC markers, and evaluated their therapeutic effects. [Sci Rep] Full Article

The Impact of Repeated Autologous Infusion of Hematopoietic Stem Cells in Patients with Liver Insufficiency
Researchers assessed the clinical and biochemical effects of repeated stem cell infusion. Ninety patients with liver cirrhosis were randomized to receive either one session treatment or two sessions four months apart of autologous hematopoietic stem cells transplantation and a control group who received regular liver treatment. [Stem Cell Res Ther] Abstract | Full Article

A Method for Performing Islet Transplantation Using Tissue-Engineered Sheets of Islets and Mesenchymal Stem Cells
Cell sheets developed using tissue engineering help maintain the function of the cells themselves. Scientists describe a tissue engineering approach using islets with mesenchymal stem cell sheets to improve the therapeutic effect of islet transplantation. [Tissue Eng Part C Methods] Abstract

Enhancement of Ischemic Wound Healing by Spheroid Grafting of Human Adipose-Derived Stem Cells Treated with Low-Level Light Irradiation
Researchers investigated whether low-level light irradiation prior to transplantation of adipose-derived stromal cell spheroids in an animal skin wound model stimulated angiogenesis and tissue regeneration to improve functional recovery of skin tissue. [PLoS One] Full Article

Cytotoxic Effects of Replication-Competent Adenoviruses on Human Esophageal Carcinoma Are Enhanced by Forced P53 Expression
Scientists constructed replication-competent type 5 adenovirus of which the E1 region genes were activated by a transcriptional regulatory region of the midkine or the survivin gene, which is expressed preferentially in human tumors. [BMC Cancer] Full Article

Amelioration of Hyperbilirubinemia in Gunn Rats after Transplantation of Human Induced Pluripotent Stem Cell-Derived Hepatocytes
Investigators explored whether transplantation of hepatocyte-like cells differentiated from human induced pluripotent stem cells could ameliorate inherited liver diseases. [Stem Cell Reports] Full Article

Advancement of the Subchondral Bone Plate in Translational Models of Osteochondral Repair – Implications for Tissue Engineering Approaches
Subchondral bone plate advancement is of increasing relevance for translational models of osteochondral repair in tissue engineering. The authors summarize the knowledge on this topic gained from a total of 31 translational investigations including 1009 small and large animals. [Tissue Eng Part B Rev] Abstract

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

bluebird bio Reports New Beta-Thalassemia Major and Severe Sickle Cell Disease Data from HGB-205 Study at EHA
bluebird bio, Inc. announced long-term follow up of two patients with beta-thalassemia major and early safety and efficacy data in the first patient with severe sickle cell disease treated with LentiGlobin BB305 product candidate in the HGB-205 study. [Press release from bluebird bio, Inc. discussing research presented at the 20^th Congress of the European Hematology Association (EHA), Vienna] Press Release

NTCELL^® Clinical Study in Parkinson’s Meets Endpoints
Living Cell Technologies Limited announced results from a Phase I/IIa clinical study of NTCELL^®, an experimental regenerative cell therapy being studied as a disease-modifying agent in Parkinson’s disease. [Press release from Living Cell Technologies Limited discussing research to be presented at the 19^th International Congress of Parkinson’s Disease and Movement Disorders, San Diego ] Press Release

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Russian Researchers Protest Government Reforms
About 3000 Russian scientists rallied in Moscow to protest against government reforms of the research system and the imposition of competitive funding, which is not commonly used in the country. [ScienceInsider] Editorial

Atara Biotherapeutics Exercises Exclusive License to T-Cell Technology from Memorial Sloan Kettering Cancer Center
Atara Biotherapeutics, Inc. announced that it has exercised its exclusive option with Memorial Sloan Kettering Cancer Center to license certain clinical stage, allogeneic T-cell therapies for the treatment of cancers and persistent viral infections. [Atara Biotherapeutics, Inc.] Press Release

Cellular Biomedicine Group Enters into Definitive Agreement to Acquire CD40LGVAX Vaccine and Related Technologies and Know-How Which Will Be Used in a Pending PD-1 Combination Clinical Trial in the U.S. for Non-Small Cell Lung Cancer (NSCLC)
Cellular Biomedicine Group Inc. announced that it has entered into a definitive agreement to acquire from Blackbird Bio Finance University of South Florida’s next generation GVAX vaccine’s (CD40LGVAX) related technologies and know-how for an initial consideration of $2.5 million in cash and $1.75 million in shares of the company’s common stock. [Cellular Biomedicine Group Inc.] Press Release

Sanford Consortium, Takeda Pharmaceutical Partner in $10-Million “Innovation Alliance”
The Sanford Consortium for Regenerative Medicine and the Takeda Pharmaceutical Company Limited have agreed to establish a $10-million, five-year Innovation Alliance that would advance each organization’s research missions. [UC San Diego] Press Release

PlasmaTech Biopharmaceuticals Expands Rare Disease Pipeline with License Agreement for Gene Therapy in Fanconi Anemia & CRISPR/Cas9-Based Technology Platform for Rare Blood Disorders
PlasmaTech Biopharmaceuticals, Inc. announced that it has added another adeno-associated virus (AAV) gene therapy program to its product pipeline. The company has licensed an AAV gene therapy and IP from the University of Minnesota to treat patients with Fanconi anemia disorder and other rare blood diseases. [PlasmaTech Biopharmaceuticals, Inc.] Press Release

Cord Blood Registry’s New Disease Registry Brings Data & Families Together to Advance Stem Cell Research
Cord Blood Registry^® announced the launch of CBR’s Family Health Registry, a unique registry designed to offer insight into common diseases and conditions affecting families and to bring patients, healthcare professionals and researchers together to expedite and facilitate research related to the discovery of stem cell treatments and potential cures. [Cord Blood Registry^®] Press Release

Heat Biologics Announces Development of ComPACT, a Next Generation Combination Immunotherapy Platform
Heat Biologics, Inc. announced the development of its next-generation combination immunotherapy platform, which combines a pan-antigen T cell priming vaccine and T cell co-stimulator in a single product. [Heat Biologics, Inc.] Press Release

STEMCELL Technologies Named Company of the Year by BIOTECanada
STEMCELL Technologies Inc. will be presented with the Biotech Company of the Year award by BIOTECanada at this year’s BIO International Convention in Philadelphia. [BIOTECanada] Press Release

Creighton Researcher Earns NIH Grant to Study Gene, Stem Cell Therapy for Coronary Grafts
A Creighton University researcher has received a National Institutes of Health (NIH) grant to study the effects of gene and stem cell therapy in coronary artery bypass grafts, a first-of-its-kind undertaking that could transform the procedure, increase survival rates and dramatically reduce the possibility of re-occlusion of the grafted arteries and veins in the procedure. [Creighton University] Press Release

Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry; Availability (FR Doc. No: 2015-14261)

Cell-Based Products for Animal Use; Guidance for Industry; Availability (FR Doc. No: 2015-14360)

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NEW Postdoctoral Fellow – Stem Cell Biology (INSERM)

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

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

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

Cell Therapy GMP Manager (Paramount Recruitment)

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

Lead Device and Delivery Scientist (Cell Therapy Catapult)

Head of Stem Cells Immunotherapies Service Lab (NHS Blood and Transplant)

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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