Volume 14.02 | Jan 21

Issue 14.02 January 21, 2013
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Engineered AAV Vector Minimizes In Vivo Targeting of Transduced Hepatocytes by Capsid-Specific CD8+ T Cells
Recent clinical trials have shown that evasion of CD8+ T cell responses against viral capsid is critical for successful liver-directed gene therapy with adeno-associated viral (AAV) vectors for hemophilia. AAV8 gene transfer resulted in prolonged susceptibility to CD8+ T cells, consistent with recent clinical findings. In contrast, using an AAV2(Y-F) mutant capsid, which is known to be less degraded by proteasomes, preserved transgene expression and largely avoided hepatotoxicity. [Blood] Abstract

TeSR-E8 Is Here

PUBLICATIONS (Ranked by impact factor of the journal)

Peptide Nanofibrils Boost Retroviral Gene Transfer and Provide a Rapid Means for Concentrating Viruses
Scientists report the development of nanofibrils that self-assemble in aqueous solution from a 12-residue peptide, termed enhancing factor C. These artificial nanofibrils enhance retroviral gene transfer substantially more efficiently than semen-derived fibrils or other transduction enhancers. [Nat Nanotechnol] Abstract

Reprogramming of Adult Rod Photoreceptors Prevents Retinal Degeneration
Researchers hypothesized that converting adult rods into cones, via knockdown of the rod photoreceptor determinant Nrl, could make the cells resistant to the effects of mutations in rod-specific genes, thereby preventing secondary cone loss. Reprogramming of adult rods achieved cellular and functional rescue of retinal degeneration in a mouse model of retinitis pigmentosa. [Proc Natl Acad Sci USA] Abstract

Focused Ultrasound Delivers Targeted Immune Cells to Metastatic Brain Tumors
NK-92, a human natural killer (NK) cell line, may be targeted to tumor-associated antigens in solid malignancies where it exhibits antitumor efficacy, but its clinical utility for treating brain tumors is limited by an inability to cross the blood-brain barrier. Researchers investigated the potential for focused ultrasound to deliver targeted NK-92 cells to the brain using a model of metastatic breast cancer. [Cancer Res] Abstract

Human Cartilage Repair with a Photoreactive Adhesive-Hydrogel Composite
A model in vitro system demonstrated deposition of cartilage-specific extracellular matrix in the hydrogel biomaterial and stimulation of adjacent cartilage tissue development by mesenchymal stem cells. For translation to the joint environment, a chondroitin sulfate adhesive was applied to covalently bond and adhere the hydrogel to cartilage and bone tissue in articular defects. After preclinical testing in a caprine model, a pilot clinical study was initiated where the biomaterials system was combined with standard microfracture surgery in 15 patients with focal cartilage defects on the medial femoral condyle. [Sci Transl Med] Abstract | Press Release

A Thermoresponsive and Chemically Defined Hydrogel for Long-Term Culture of Human Embryonic Stem Cells
Scientists report the identification of a family of chemically defined thermoresponsive synthetic hydrogels based on 2-(diethylamino)ethyl acrylate, which support long-term human embryonic stem cell growth and pluripotency over a period of two-to-six months. [Nat Commun] Full Article | Press Release

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy
To test whether small gene size constitutes a fundamental design error for large mammalian muscle, researchers performed a comprehensive study using 22 dogs. They delivered the ΔR2-15/ΔR18-19/ΔR20-23/ΔC microdystrophin gene to eight extensor carpi ulnaris muscles in six dystrophic dogs using Y713F tyrosine mutant adeno-associated virus-9. [Mol Ther] Abstract | Press Release

Muscle-Derived Stem Cell Sheets Support Pump Function and Prevent Cardiac Arrhythmias in a Model of Chronic Myocardial Infarction
Researchers evaluated whether the transplantation of a muscle-derived stem cell (MDSC) sheet could improve the regenerative capacity of MDSCs in a chronic model of myocardial infarction. [Mol Ther] Abstract

A Mutant Tat Protein Provides Strong Protection from HIV-1 Infection in Human CD4+ T Cells
Nullbasic is a mutant form of the HIV-1 Tat protein that was previously shown to strongly inhibit HIV-1 replication in non-hematopoietic cell lines by targeting three steps of HIV-1 replication: reverse transcription, transport of viral mRNA and transactivation of HIV-1 gene expression. Here researchers investigated gene delivery of Nullbasic using lentiviral and retroviral vectors. [Hum Gene Ther] Abstract | Full ArticlePress Release

GMP-Compliant, Large-Scale Expanded Allogeneic Natural Killer Cells Have Potent Cytolytic Activity against Cancer Cells In Vitro and In Vivo
Investigators established a simplified and efficient method for the large-scale expansion and activation of natural killer (NK) cells from healthy donors under good manufacturing practice (GMP) conditions. Compared with freshly isolated NK cells, these expanded NK cells showed robust cytokine production and potent cytolytic activity against various cancer cell lines. [PLoS One] Full Article

Engineered Stem Cell-Derived Microglia as Therapeutic Vehicle for Experimental Autoimmune Encephalomyelitis
Researchers showed that genetically engineered embryonic stem cell-derived microglia (ESdM) are a suitable therapeutic vehicle for neurotrophin-3 (NT3) in experimental autoimmune encephalomyelitis (EAE). The intravenously transplanted ESdM migrated into the inflammatory central nervous system lesions and engrafted there as microglial cells. EAE afflicted mice treated with ESdM that were genetically modified to express NT3 showed stable recovery from disease symptoms. [Gene Ther] Abstract

Free Nature Reviews Immunology Poster: The Immune Response to HIV


Adult Salivary Gland Stem Cells and a Potential Therapy for Xerostomia
Researchers review the current state of knowledge of salivary gland stem cells and their potential for stem cell-based therapies that may provide a more durable cure for hyposalivation. [Stem Cells] Abstract | Full Article

Mesenchymal Stem Cells and the Lung
In this review the authors outline what is known about mesenchymal stem cell (MSC) biology with particular emphasis on lung biology, and also highlight the therapeutic potential of MSC-based cell therapy. [Respirology] Abstract

New: Directed Differentiation of Pluripotent Stem Cells. A Cell Stem Cell Poster


Neuralstem Receives FDA Approval to Commence Spinal Cord Injury Trial
Neuralstem, Inc. announced that it received approval from the United States Food and Drug Administration (FDA) to commence a Phase I safety trial of its lead cell therapy candidate, NSI-566, in chronic spinal cord injury patients. The primary objective of the study is to determine the safety and toxicity of human spinal stem cell transplantation for the treatment of paralysis and related symptoms due to chronic spinal cord injury. [Neuralstem, Inc.] Press Release

SanBio Announces Enrollment of the Second Cohort of Patients in Its Clinical Trial of Stem Cell Therapy for Chronic Stroke
SanBio Inc. announced the successful enrollment of the second dose cohort of patients in its Phase I/IIa clinical trial testing the safety and efficacy of a novel allogeneic stem cell therapy product, SB623, in patients suffering from chronic deficits resulting from previous stroke injuries. The first 12 patients, of a planned total of 18, have been successfully administered SB623. [SanBio Inc.] Press Release

Cell Culture World Congress 2013


Funding for Texas Cancer Research Agency in Limbo
Texas legislators are threatening to cut off funding for the state’s troubled $3 billion cancer research agency unless the organization can resolve problems with how it awards money. [House of Representatives, United States] Press Release

Japan’s Stimulus Propels Science Spending to New Heights
The economic stimulus the Japanese government announced includes $11 billion for science and technology according to a preliminary analysis released by the Cabinet office. Combined with previously planned spending, total national and local government support for science for the fiscal year through March will reach $57 billion, a new record. Much of the new money will go to applied research. [Japanese Government, Japan] Press Release


NeoStem’s Subsidiary, Progenitor Cell Therapy, Enters into a Cell Therapy Manufacturing Services Agreement with Adaptimmune
NeoStem, Inc. and its subsidiary, Progenitor Cell Therapy LLC (PCT), together with Adaptimmune Limited and Adaptimmune LLC (Adaptimmune), announced a Services Agreement under which PCT will provide services to support Adaptimmune’s NYESO-1c259-T cell therapy product being developed for multiple oncology indications. [NeoStem, Inc.] Press Release

DSM Announces License Agreement with Amgen for Use of DSM’s XD® Cell Culture Patents
DSM Pharmaceutical Products announced that it has signed a non-exclusive license agreement with Amgen Inc. for access to DSM’s proprietary XD® high cell density process patents. [DSM Pharmaceutical Products]
Press Release

Apeiron Out-Licenses Antibody-Based Immunotherapy
Apeiron Biologics closed its first transaction in its ongoing initiative to out-license APN311, an antibody-based immunotherapy in development for the treatment of children with high-risk neuroblastoma. Paladin Labs entered into an exclusive licensing agreement with Apeiron, under which Canada-based Paladin receives the rights to market and sell APN311 in Canada and Sub-Saharan Africa, including South Africa. [Genetic Engineering & Biotechnology News] Press Release

UB to Share $12.1 Million State Grant for Stem Cell Study in MS
The grant, funded by the Empire State Stem Cell Board, will allow scientists from University at Buffalo (UB), SUNY Upstate Medical Center and the University of Rochester Medical Center to test the safety and effectiveness of implanting stem cells that can reproduce myelin into the central nervous system of multiple sclerosis (MS) patients. [UB School of Medicine and Biomedical Sciences] Press Release

Northwestern Medicine Plans $1 Billion Research Investment
Leaders of Northwestern Medicine, comprising Northwestern University Feinberg School of Medicine, Northwestern Medical Faculty Foundation and Northwestern Memorial HealthCare, announced they have made a transformational commitment of more than $1 billion toward creating a leading medical research enterprise on Northwestern’s campus in Chicago’s Streeterville neighborhood. [Northwestern University] Press Release

Argos Therapeutics Enrolls Initial Patients in Pivotal Phase III ADAPT Study
Argos Therapeutics Inc. announced that the first patients have been enrolled in the ADAPT Phase III clinical study for AGS-003, its most advanced product candidate. AGS-003 is an investigational, fully personalized, dendritic-cell based immunotherapy. The ADAPT study is designed to examine the potential for AGS-003 plus standard targeted drug therapy to extend overall survival versus standard therapy alone in newly diagnosed patients with unfavorable risk metastatic renal cell carcinoma. [Argos Therapeutics Inc.] Press Release

Pluristem Receives Approval to Expand Its Phase II Clinical Trial in Intermittent Claudication to Germany
Pluristem Therapeutics, Inc. announced that the Paul-Ehrlich-Institute has approved the company’s request to initiate a Phase II study using PLX-PAD cells in patients suffering from Intermittent Claudication. [Pluristem Therapeutics, Inc.] Press Release

Cellular Dynamics Announces the Production of Human iPSC Lines Under cGMP Conditions
Cellular Dynamics International, Inc. announced that it is producing human induced pluripotent stem cell (iPSC) master cell banks from five individual donors under current Good Manufacturing Practices (cGMPs). Manufacturing human iPSCs and ultimately human cells under cGMPs enables the possible use of iPSC-derived human cells within a clinical setting. [Cellular Dynamics International, Inc.] Press Release


National Center for Advancing Translational Sciences; Notice of Closed Meetings (FR Doc No: 2013-01101)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-01096)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-01099)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meetings (FR Doc No: 2013-00879)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2013-00618)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2013-00738)


European Medicines Agency (European Union)

eSubmission Gateway Release II and eSubmission Web Client Now Live for All Applications for Human Medicines

Food and Drug Administration (United States)

Draft Guidance for Industry and Food and Drug Administration Staff; Submissions for Postapproval Modifications to a Combination Product Approved Under Certain Marketing Applications; Availability (FR Doc No: 2013-01069)

Current Good Manufacturing Practice Requirements for Combination Products (FR Doc No: 2013-01068)

Therapeutic Goods Administration (Australia)

Australian Therapeutic Goods Advisory Council
The Australian Government announced that it would establish an Australian Therapeutic Goods Advisory Council to provide broad strategic advice to the TGA, with an emphasis on improving communications between the regulator and its key stakeholders. Press Release

Regulatory Framework for Biologicals


NEW The Jackson Laboratory: Everything but the Experiment – Tips for Working with Diabetic Mice
January 31, 1:00PM (ET in US)

NEW International Symposium on Development of Medical Technologies for Treating Intractable Cancers and Cardiovascular Diseases
March 1-2, 2013
Tokyo, Japan

NEW Canadian Blood and Marrow Transplant Group (CBMTG) Annual Meeting 2013
April 10-12, 2013
Winnipeg, Canada

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.


Scientist or Engineer – hPSC Bioengineer (STEMCELL Technologies, Inc.)

Senior QC Analyst/Assay Development Associate (StemCells, Inc.)

Postdoctoral Position (Sandra Rotman Centre for Global Health)

Postdoctoral Fellow – Molecular Biology (Cleveland Clinic Foundation)

Faculty Position – Stem Cell, Vascular and Developmental Biology in Ophthalmology (Zhongshan Ophthalmic Center, Sun Yat-Sen University)

Postdoctoral Position – Cardiovascular Regenerative Therapies (University of California, Los Angeles)

Postdoctoral Researcher (The University of Texas Health Science Center at San Antonio)

Postdoctoral Fellow – Transcriptional Regulation in CD8 T lymphocytes (Université Libre de Bruxelles – Institute for Medical Immunology)

Postdoctoral Researcher Position in Bioinformatics (University of Oulu)

Research Associate (Imperial College London)

Marie Curie Postdoctoral Position (Institute for Bioengineering of Catalonia)

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