Volume 16.42 | Nov 16

Issue 16.42 November 16, 2015
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Batten Disease May Benefit from Gene Therapy
In a study of dogs, scientists showed that a new way to deliver replacement genes may be effective at slowing the development of childhood Batten disease, a rare and fatal neurological disorder. The key may be to inject viruses that carry the codes for the gene products into the ventricles, which are fluid-filled compartments in the center of the brain that serve as a plumbing system. [Press release from the National Institute of Neurological Disorders and Stroke discussing online publication in Science Translational Medicine] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Decreased Amyloid-β and Increased Neuronal Hyperactivity by Immunotherapy in Alzheimer’s Models
Using in vivo two-photon imaging in mouse models, investigators found that two different antibodies to amyloid-β used for treatment were ineffective at repairing neuronal dysfunction and caused an increase in cortical hyperactivity. [Nat Neurosci] Abstract | Press Release

Disarmed Anthrax Toxin Delivers Antisense Oligonucleotides and siRNA with High Efficiency and Low Toxicity
As anthrax toxin (Atx) accesses the cytosol, the authors evaluated the potential of disarmed Atx to deliver either antisense oligonucleotides or siRNA. They hypothesized that this delivery strategy would facilitate improved transfection efficiency while eliminating the toxicity seen for many vectors due to membrane destabilization. [J Control Release] Full Article | Graphical Abstract

Generation of Functional Human Cardiac Progenitor Cells by High-Efficiency Protein Transduction
Investigators assessed highly efficient protein-based approaches to reduce or eliminate the genetic manipulations to generate cardiac progenitor cells (CPCs) for cardiac regeneration therapy. A combination of QQ-reagent-modified Gata4, Hand2, Mef2c, and Tbx5 and three cytokines rapidly and efficiently reprogrammed human dermal fibroblasts into CPCs. [Stem Cell Transl Med] Abstract | Press Release

Muscle Tissue Engineering and Regeneration through Epigenetic Reprogramming and Scaffold Manipulation
Researchers investigated the combined effects of matrices of variable rigidities and the treatment with the epigenetic modulator 5-azacytidine on reprogramming adipose-derived stromal cells into myoblast-like cells by utilizing tunable transglutaminase cross-linked gelatin in vitro and in vivo. [Sci Rep] Full Article

T-Replete Haploidentical Allogeneic Transplantation Using Post-Transplantation Cyclophosphamide in Advanced AML and Myelodysplastic Syndromes
Investigators analyzed 60 consecutive patients with refractory or high-risk CR AML or myelodysplastic syndromes who underwent post-transplantation cyclophosphamide haploidentical transplantation. [Bone Marrow Transplant] Abstract

Transplantation of Rat Embryonic Stem Cell-Derived Retinal Progenitor Cells Preserves the Retinal Structure and Function in Rat Retinal Degeneration
Investigators generated retinal progenitor cells (RPCs) from rat embryonic stem cells (rESCs) and to test their therapeutic effects in rat model. The rESCs were cultured in N2B27 medium with 2i, and differentiated to two types of RPCs following the quickly-aggregated serum-free embryonic body method with modifications. [Stem Cell Res Ther] Full Article

Co-Transplantation of Skin-Derived Precursors and Collagen Sponge Facilitates Diabetic Wound Healing by Promoting Local Vascular Regeneration
Skin-derived precursors (SKPs) were isolated and transplanted directly to the wound areas of diabetic mice in the absence and presence of collagen sponge. The effects of SKPs and/or collagen sponge on diabetic wound healing were examined histologically as well as immunostaining of isolectin and α-SMA. [Cell Physiol Biochem] Full Article

Phenotypic Modulation and Neuroprotective Effects of Olfactory Ensheathing Cells: A Promising Tool for Cell Therapy
In vitro, olfactory ensheathing cells (OECs) promote axonal growth, while in vivo they promote remyelination of damaged axons. Scientists investigated OEC potential for regeneration and functional recovery of the damaged central nervous system. [Stem Cell Rev] Abstract

Synergistic Tumor Suppression by Adenovirus-Mediated ING4/PTEN Double Gene Therapy for Gastric Cancer
Researchers found that a multiple promoter expression cassette-based recombinant adenovirus coexpressing inhibitor of growth 4 (ING4) and phosphatase and tensin homolog (PTEN) induced synergistic growth inhibition and apoptosis in vitro AGS or SNU-1 tumor cells and in vivo AGS xenografted tumors subcutaneously inoculated in athymic BALB/c nude mice. [Cancer Gene Ther] Abstract

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Chemical and Structural Modifications of RNAi Therapeutics
The authors describe recent progress in chemically and structurally modified small interfering RNAs (siRNAs) to solve their intrinsic problems and siRNA delivery formulations including siRNA conjugates, polymerized siRNA, and nucleic acid-based nanoparticles to improve in vivo delivery. [Adv Drug Deliv Rev] Abstract

Inflammation in Tissue Engineering: The Janus between Engraftment and Rejection
The authors discuss current and potential strategies for inflammation modulation to aid engraftment and regeneration, supporting the concept that quality, and not quantity, of inflammation might influence implant success. [Eur J Immunol] Abstract

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Alnylam Pharmaceuticals and The Medicines Company Announce that New ALN-PCSsc Results from Late-Breaking Presentation Confirm Potential for Bi-Annual Hypercholesterolemia Dose Regimen
Alnylam Pharmaceuticals, Inc. and The Medicines Company reported positive results from their ongoing Phase I clinical trial with ALN-PCSsc in a late-breaking oral presentation. ALN-PCSsc is an investigational RNAi therapeutic targeting PCSK9 being developed for the treatment of hypercholesterolemia. [Press release from The Medicines Company discussing research presented at American Heart Association Meeting, Orlando] Press Release

Advaxis Presents New Data Featuring Its Lm Technology™
Advaxis, Inc. evaluated whether axalimogene filolisbac could be dosed at higher levels than previous studies conducted by Advaxis in patients with persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix. [Press release from Advaxis, Inc. discussing research presented at the Society for Immunotherapy of Cancer 2015 Annual Meeting, National Harbor] Press Release

Arrowhead Presents Data Showing Robust Sustained Anti-Viral Effects with ARC-520 in Hepatitis B Infected Chimpanzees
Arrowhead Research Corporation, a biopharmaceutical company developing targeted RNAi therapeutics, presented data that ARC-520 its drug candidate against chronic hepatitis B infection (HBV), leads to robust, sustained anti-viral effects in chimpanzees with chronic HBV. [Press release from Arrowhead Research Corporation (Business Wire) discussing research presented at the American Association for the Study of Liver Diseases (AASLD) Liver Meeting 2015, San Francisco] Press Release

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European Commission Appoints Top Scientists to Fill Policy Advice Gap
Seven scientists, including a Fields medalist and the director of CERN, Europe’s premier particle physics lab, have been appointed to provide the European Commission with policy advice—bringing an end to a year of suspense since the awkward exit of Anne Glover, the first and only chief scientific adviser in the commission’s history. [ScienceInsider] Editorial

Cancer Researchers Band Together to Pool Tumor Genome Data
It’s the latest hope offered to many patients at major cancer research centers: Let us sequence your tumor, and maybe we can match it to a drug that will beat back the malignancy. But the reality is that genomic analysis still only helps a small fraction of cancer patients. Several large U.S. and European academic medical centers are hoping to change that by pooling data on patients’ tumor genomes and their clinical outcomes. [ScienceInsider] Editorial

Chile’s Scientists Take to the Streets over Funding
Faced with street protests by researchers, together with the resignation of the nation’s top science official and the publication of an open letter accusing the government of ignorance, Chile’s Congress will consider a budget increase of 150 million pesos ($210,000) for the nation’s research funding agency. [Nature News] Editorial
ImmunoCellular Therapeutics Enters into Sponsored Research Agreement with University of Texas MD Anderson Cancer Center to Advance Stem-to-T-Cell Program
ImmunoCellular Therapeutics, Ltd. announced it has entered into a sponsored research agreement with Dr. Cassian Yee at the University of Texas MD Anderson Cancer Center. The ultimate goal of this work is to enable a clinical program based on hematopoietic stem cells that are isolated from the patient, engineered in the lab and then returned to the patient to create a population of antigen-specific killer T cells that target and kill the tumor. [ImmunoCellular Therapeutics, Ltd.] Press Release

Enterome and Gustave Roussy Enter into a Microbiome-Based Collaboration to Develop Novel Immunotherapy Solutions to Treat Cancer
Enterome Bioscience SA announced that it has entered into a collaborative development agreement with Gustave Roussy. They aim to develop of a new generation of drugs and non-invasive monitoring tools of the gut microbiome in immuno-oncology. [Enterome Bioscience SA] Press Release

Cellular Dynamics Announces Collaboration with Roche
Cellular Dynamics International, Inc. announced an agreement with Roche to supply CDI’s induced pluripotent stem cell-derived iCell products to contribute to the identification of novel drug candidates at the early stages of drug discovery. [Cellular Dynamics International, Inc.] Press Release

Moffitt Cancer Center’s Physical Sciences – Oncology Center Receives $10.4 Million Grant to Study the Intersection of Evolution and Cancer Therapy
The Integrated Mathematical Oncology (IMO) Department integrates their skills with cancer biologists and oncologists in teams to use mathematical models to better understand cancer progression and treatment. This team driven science has led to pioneering work that has recently been acknowledged through a $10.4 million award from the National Cancer Institute, designating Moffitt as one of the five Physical Science – Oncology Centers. [Moffitt Cancer Center (Newswire Inc.)] Press Release

Derma Sciences Announces Results of Futility Analyses for Phase III Clinical Trials of Aclerastide in Diabetic Foot Ulcer Healing
Derma Sciences, Inc., a tissue regeneration company focused on advanced wound and burn care, announced the termination of its Phase III clinical trials with aclerastide for diabetic foot ulcer healing. [Derma Sciences, Inc.] Press Release

Will HER-2 Targeted Treatment Improve the Efficacy of Chemotherapy for Early HER-2 Positive Gastric Cancer?
EORTC trial 1203 –Integration of trastuzumab, with or without pertuzumab, into perioperative chemotherapy of HER-2 positive stomach cancer, is open for recruitment for patients with early, HER-2 positive gastric cancer. [European Organisation for Research and Treatment of Cancer (EORTC)] Press Release

Celyad Successfully Completes 30-Day Safety Follow-Up of First Patient Cohort in NKG2D CAR T-Cell Phase I Trial
Celyad announced the completion of the 30-day safety follow-up of the final patient enrolled in the first cohort of the company’s Phase I clinical trial evaluating the safety and feasibility of its NKG2D CAR T-Cell therapy in cancer patients suffering from acute myeloid leukemia or multiple myeloma. [Celyad] Press Release

Regeneus Ltd. Patent Granted for Allogeneic Stem Cell Technology Platform
Regeneus announced that the Australian Patent Office has granted a key patent covering the use of the company’s allogeneic “off-the-shelf” stem cell technology for the treatment of osteoarthritis and other inflammatory conditions for human and animal applications. [Regeneus Ltd. (ABN Newswire)] Press Release

Cellular Biomedicine Group Opens New GMP Facility in Beijing
Cellular Biomedicine Group Inc. announced the opening of its new state-of-the-art facility in Beijing. Eight hundred square meters of the 1,400 square meter site has been equipped with four independent production lines to support clinical batch production and commercial scale manufacturing. [Cellular Biomedicine Group Inc.] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-28655)
Summary Basis for Regulatory Action – COAGADEX
Food and Drug Administration (United States)

Guidance on Qualification of Biomarker-Galactomannan in Studies of Treatments of Invasive Aspergillosis; Guidance for Industry; Availability (FR Doc. No: 2015-28804)
NEW Keystone Symposia – Stem Cells and Cancer
March 6-10, 2016
Breckenridge, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Investigator – Cancer Biology (Wake Forest Baptist School of Medicine)

NEW Production Manager – UK Stem Cell Bank (Medicines and Healthcare products Regulatory Agency)

Postdoctoral Fellow – Genome Editing for Retinal Diseases (Mass Eye and Ear and Harvard Medical School)

Staff Scientist – Immunology and Clinical Research (Fred Hutchinson Cancer Research Center)

Postdoctoral Position – Scaffold Design & Drug/Gene Delivery for Neural Tissue Engineering (Nanyang Technological University)

Postdoctoral Position – Stem Cell Engineering Research (Nanyang Technological University)

Postdoctoral Position – hPSC-Derived Cardiomyocyte Grafts in Injured Hearts (University Health Network)

Group Leader – Translational Research (Cancer Research Center of Toulouse)

Group Leader Position – Cancer Research (Oslo University Hospital)

PhD Studentships – Cancer Research (Cancer Research UK Manchester Institute)

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

Research Technician – TCR Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Technician – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant/Associate/Full Professor – Biomedical Engineering (Worcester Polytechnic Institute)

Postdoctoral Position – Spinal Cord Injury Research (Indiana University-Purdue University)

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