Volume 16.14 | Apr 13

Newsletter Issue
Issue 16.14 April 13, 2015
Cell Therapy News - Your Industry in an Instant
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In First Human Study, New Antibody Therapy Shows Promise in Suppressing HIV Infection
In the first results to emerge from HIV patient trials of a new generation of so-called broadly neutralizing antibodies, researchers have found the experimental therapy can dramatically reduce the amount of virus present in a patient’s blood. [Press release from The Rockefeller University discussing online prepublication in Nature]
Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
An RNAi Therapeutic Targeting Antithrombin to Rebalance the Coagulation System and Promote Hemostasis in Hemophilia
Guided by reports suggesting that co-inheritance of prothrombotic mutations may ameliorate the clinical phenotype in hemophilia, investigators developed an RNA interference (RNAi) therapeutic (ALN-AT3) targeting antithrombin (AT) as a means to promote hemostasis in hemophilia. When administered subcutaneously, ALN-AT3 showed potent, dose-dependent, and durable reduction of AT levels in wild-type mice, mice with hemophilia A, and nonhuman primates. [Nat Med] Abstract | Press Release

Faster T-Cell Development following Gene Therapy Compared to Haplo-Identical Hematopoietic Stem Cell Transplantation in the Treatment of SCID-X1
Researchers retrospectively analyzed and compared clinical outcomes and immune reconstitution in 13 consecutive severe combined immunodeficiency caused by ILR2G mutation (SCID-X1) patients having undergone haploidentical hematopoietic stem cell transplantation and 14 SCID-X1 patients treated with gene therapy over the same period at a single center level, the Necker Children’s Hospital. [Blood]
Abstract | Full Article | Press Release

ADAP and SKAP55 Deficiency Suppresses PD-1 Expression in CD8+ Cytotoxic T Lymphocytes for Enhanced Anti-Tumor Immunotherapy
Scientists identified that the ADAP-SKAP55 signaling module reduced CD8+ cytotoxic T lymphocytes cytotoxicity and enhanced PD-1 expression in a Fyn-, Ca2+-, and NFATc1-dependent manner. [EMBO Mol Med] Full Article

Long-Term Controlled Delivery of rhBMP-2 from Collagen-Hydroxyapatite Scaffolds for Superior Bone Tissue Regeneration
Investigators aimed to design and develop a delivery system to release low recombinant human bone morphogenetic protein 2 (rhBMP-2) doses from a collagen-hydroxyapatite scaffold which had been previously been optimized for bone regeneration and recently demonstrated significant healing in vivo. [J Control Release] Abstract

Ex Vivo Induced Regulatory Human/Murine Mesenchymal Stem Cells as Immune Modulators
The authors established an ex vivo immunomodulatory triple combination treatment (TCT) for mesenchymal stem cells (MSCs), using IFNγ, TGFβ and kynurenine. To test the immunomodulatory function of TCT MSCs in vivo, they used the cells as graft-versus-host disease (GvHD) prophylaxis in a GvHD mouse model. [Stem Cells] Abstract

Gene Electrotransfer of Plasmid AMEP, an Integrin-Targeted Therapy, Has Antitumor and Antiangiogenic Action in Murine B16 Melanoma
Researchers investigated the antiangiogenic and direct antitumor effectiveness of gene therapy with Plasmid AMEP (antiangiogenic metargidin peptide) on low and highly metastatic B16 melanoma variants. In vivo, antitumor effectiveness was mediated by the inhibition of proliferation, migration and invasion of melanoma cells and correlated with the expression of integrins on tumor cells after intratumor delivery. [Gene Ther] Abstract

Long-Term Results of Adipose-Derived Stem Cell Therapy for the Treatment of Crohn’s Fistula
A previous Phase II clinical trial of adipose-derived stem cell therapy for fistulae associated with Crohn’s disease, a devastating condition with a high recurrence rate, demonstrated safety and therapeutic potential with a one-year sustained response. In the present study, 41 of the 43 Phase II trial patients were followed for an additional year, regardless of response in the initial year. [Stem Cells Transl Med] Abstract

Delayed Minimally Invasive Injection of Allogenic Bone Marrow Stromal Cell Sheets Regenerates Large Bone Defects in an Ovine Preclinical Animal Model
Researchers developed a unique treatment concept using the delayed injection of allogenic bone marrow stromal cell sheets to regenerate a critical-sized tibial defect in sheep to study the effect of the cells’ regeneration potential when introduced at a postinflammatory stage. [Stem Cells Transl Med] Abstract

The Critical Role of Induced CD4+ FoxP3+ Regulatory Cells in Suppression of Interleukin-17 Production and Attenuation of Mouse Orthotopic Lung Allograft Rejection
Researchers established mouse orthotopic lung transplantation models to investigate the importance of interleukin (IL)-17 and IL-17-producing cell types in acute lung allograft rejection and the efficacy of the adoptive transfer of induced regulatory T cells in attenuating pathologic lesions of lung allografts. [Transplantation] Abstract

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Immune Checkpoint Targeting in Cancer Therapy: Toward Combination Strategies with Curative Potential
The authors discuss the evolution of research in cancer signaling pathways and immune checkpoints and propose that intercrossing them and increasing funding to guide research of combination of agents represent a path forward for the development of curative therapies for the majority of cancer patients. [Cell] Abstract | Press Release

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

Development of Simplified and Defined Cell Culture for hPSCs - Watch Now!
Dicerna Presents New β-Catenin Data from Multiple Tumor Models
Dicerna Pharmaceuticals, Inc. announced promising new in vivo data for CTNNB1 Dicer substrate short interfering RNA in multiple patient-derived xenograft and other models of diverse tumor types. [Press release from Dicerna Pharmaceuticals, Inc. discussing research presented at the at the 2015 RNA & Oligonucleotide Therapeutics Meeting, Cold Spring Harbor] Press Release

Benitec Provides Update on TT-034 Trial
Benitec Biopharma, a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi, announced that laboratory results from liver biopsies in the company’s ‘first in man’, Phase I/IIa clinical trial of TT-034 for hepatitis C confirmed that the trial is proceeding according to expectations. [Benitec Biopharma Limited] Press Release

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Research Advocates Watch, Warily, as Congress Tries to Finish Its Budget Outline
Congress returned to work after a two-week recess, and a top priority for House of Representatives and Senate Republican leaders is reconciling their versions of a largely symbolic but politically sensitive budget plan. Science boosters loathe the spending blueprints approved by the House and Senate. That’s because they would, if implemented, squeeze federal funding for civilian research over the long term. [ScienceInsider] Editorial

Synthetic Biologists Seek Standards for Nascent Field
Representatives from industry, academic institutions and government met at Stanford University to launch the Synthetic Biology Standards Consortium, an initiative led by the US National Institute of Standards and Technology to address issues preventing the field from reaching its potential. [Nature News] Editorial
Advaxis and Merck Announce Initiation of Enrollment in the Phase I/II Study of ADXS-PSA in Combination with Anti-PD-1 Therapy, KEYTRUDA® (Pembrolizumab), for the Treatment of Advanced Prostate Cancer
Advaxis, Inc. and Merck announced that enrollment has initiated in the Phase I/II clinical trial evaluating the combination of ADXS-PSA (ADXS31-142), an investigational Lm-LLO immunotherapy, and KEYTRUDA®, the first anti-PD-1 (programmed death receptor-1) therapy approved in the United States, in patients with previously treated, metastatic castration-resistant prostate cancer. [Advaxis, Inc.] Press Release

Taxus Cardium Announces Agreement with Dr. Reddy’s Laboratories to Co-Develop, Market & Sell Generx Angiogenic Microvascular Gene Therapy in Certain International Markets
Taxus Cardium Pharmaceuticals Group Inc. announced that it has entered into a binding term sheet with Dr. Reddy’s Laboratories Ltd. for an exclusive territorial license covering the co-development, marketing and sales of the Generx® angiogenic microvascular gene therapy Phase III product candidate for patients with refractory angina, and myocardial ischemia due to cardiac microvascular insufficiency. [Taxus Cardium Pharmaceuticals Group Inc.] Press Release

MD Anderson, Astellas Pharma Sign Option Agreement for Monoclonal Antibody Drug Targeting Acute Myeloid Leukemia
Astellas Pharma Inc. and The University of Texas MD Anderson Cancer Center announced the signing of an option agreement to research and develop a new treatment for patients with acute myeloid leukemia. [The University of Texas MD Anderson Cancer Center] Press Release

Mount Sinai Receives $8.8 Million Grant to Further Stem Cell Research
An $8.8 million grant from the New York State Stem Cell Science Program will accelerate efforts by the Icahn School of Medicine at Mount Sinai to develop new stem-cell-based treatments for chemotherapy-resistant blood cancer and other genetic blood disorders. [Icahn School of Medicine at Mount Sinai] Press Release

FDA Modifies Partial Clinical Hold on Tekmira’s TKM-Ebola IND to Allow Multiple Dosing of Healthy Volunteers
Tekmira Pharmaceuticals Corporation announced that the U.S. Food and Drug Administration (FDA) has notified the company that the partial clinical hold on the company’s Investigational New Drug application (IND) for TKM-Ebola has been modified to permit repeat dosing of healthy volunteers at a dose of 0.24 mg/kg/day. [Tekmira Pharmaceuticals Corporation] Press Release

Purdue Startup Is Developing Self-Assembling, Bioinstructive Collagen Materials for Research, Medical Applications
A Purdue University researcher and entrepreneur is commercializing her laboratory’s innovative collagen formulations that self-assemble or polymerize to form fibrils that resemble those found in the body’s tissues. [Purdue University] Press Release

Cytori and Lorem Vascular Receive Regulatory Clearance in China
Cytori Therapeutics, Inc. announced that its exclusive licensee, Lorem Vascular, has been granted regulatory clearance for the Cytori Celution® System by the State Food and Drug Administration of the People’s Republic of China. [Cytori Therapeutics, Inc.] Press Release

Editing HIV Out of Our Genome with CRISPR
In an attempt to render latent HIV completely harmless, researchers at the University of Massachusetts Medical School are using Cas9/CRISPR, a powerful gene editing tool, to develop a novel technology that can potentially cut the DNA of the latent virus out of an infected cell. [University of Massachusetts Medical School] Press Release
Office of the Director, Office of Science Policy, Office of Biotechnology Activities; Notice of Meeting (FR Doc. No: 2015-07981)
ALBAsure QC Kit
Food and Drug Administration (United States)

Risk Evaluation and Mitigation Strategies: Modifications and Revisions; Guidance for Industry; Availability (FR Doc. No: 2015-08015)
NEW The 2015 Tissue Engineering Congress
September 8-10, 2015
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Professor – Gene Therapy (University of Massachusetts)

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

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Research Scientist – Cell and Gene Therapy Group (Biogen Idec)

Lecturer – Gene Therapy (University College London)

Postdoctoral Position – Cancer Imaging (King’s College London)

Research Associate – Cell Culture Specialist (Tisch MS Research Center of New York)

Manufacturing Manager – Cell Therapy (Opexa Therapeutics)

Postdoctoral Positions – Developmental Biology (Joslin Diabetes Center)

Cell and Gene Therapy Formulation Scientist (Stelfox)

Associate Director, Center for Gene Therapy (City of Hope)

Assistant Professor – Neuroscience (Georgia State University)

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