Cell Therapy News Volume 17.00 | Jan 4 2016

In Vivo Gene Editing in Dystrophic Mouse Muscle and Muscle Stem Cells
Researchers developed and tested a direct gene-editing approach to induce exon deletion and recover dystrophin expression in the mdx mouse model of Duchenne muscular dystrophy. [Science] Abstract

TG4010 Immunotherapy and First-Line Chemotherapy for Advanced Non-Small-Cell Lung Cancer (TIME): Results from the Phase IIb Part of a Randomized, Double-Blind, Placebo-Controlled, Phase IIb/III trial
In this Phase IIb part of the Phase IIb/III TIME trial, researchers assessed TG4010 in combination with first-line chemotherapy and use of the triple-positive activated lymphocytes biomarker in this setting. [Lancet Oncol] Abstract | Press Release

Development of Non-Viral Vectors Targeting the Brain as a Therapy for Parkinson’s Disease and Other Brain Disorders
Researchers report the development of non-viral vectors specific for neuronal cells that can deliver short interfering RNA against the α-synuclein gene, and prevent Parkinson’s disease-like symptoms both in vitro and in vivo. [Mol Ther] Abstract

Serial Activation of the Inducible Caspase 9 Safety Switch after Human Stem Cell Transplantation
The authors report a patient who received an infusion of haploidentical inducible caspase 9-T cells after HSCT and subsequently received three treatments with AP1903. There was a mild and transient pancytopenia following each AP1903 administration but no non-hematological toxicity. [Mol Ther] Abstract

Fetal Hematopoietic Stem Cell Transplantation Fails to Fully Regenerate the B-Lymphocyte Compartment
Researchers showed that, similar to adult hematopoietic stem cells (HSCs), fetal HSCs selectively fail to regenerate B-1a. They also showed that, in humanized mice, human fetal liver regenerates tissue B cells that are phenotypically similar to murine B-1a, raising the question of whether human HSC transplantation, the mainstay of such models, is sufficient to regenerate human B-1a. [Stem Cell Rep] Full Article

Transplantation of Placenta-Derived Mesenchymal Stem Cells Reduces Hypoxic-Ischemic Brain Damage in Rats by Ameliorating the Inflammatory Response
Investigators explored the immunoregulatory mechanism of placenta-derived mesenchymal stem cells in hypoxic-ischemic brain damage treatment. [Cell Mol Immunol] Abstract

AAV_1/2– Mediated BDNF Gene Therapy in a Transgenic Rat Model of Huntington’s Disease
To investigate the potential therapeutic properties of BDNF for Huntington’s disease (HD), scientists examined the effect of bilateral AAV_1/2-mediated BDNF expression in the striatum of a transgenic rat model of HD. [Gene Ther] Abstract

Prevalence of AAV1 Neutralizing Antibodies and Consequences for a Clinical Trial of Gene Transfer for Advanced Heart Failure
This study was designed to determine characteristics of adeno-associated virus (AAV) neutralizing antibodies (NAbs) in human subjects, prevalence of AAV1 NAbs in heart failure patients and utility of aggressive immunosuppressive therapy in reducing NAb seroconversion in an animal model. [Gene Ther] Abstract

Allogeneic Stem Cell Transplantation for Multiple Myeloma: Is There a Future?
The authors provide an in-depth review of literature for allogeneic hematopoietic cell transplantation (allo-SCT) and other immunotherapy options, as well as the authors’ approach to using allo-SCT in multiple myeloma. [Bone Marrow Transplant] Abstract

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Canada’s Top Scientist Faces Tough Challenge
Kirsty Duncan, the medical geographer who last month became Canada’s first Minister of Science, has a big mandate: to ensure that scientific considerations again figure into public-policy decisions. [Nature News] Editorial

NAS Panel Tackles—and Is Tackled by—Genome Editing in Animals
A two-day National Academy of Sciences (NAS) workshop here last week exposed just how far scientists, ethicists, and regulators are from agreeing on the best way to move forward with genome editing in animals. [ScienceInsider] Editorial

E.U. Frees Up Data for Science
Scientists in Europe have won a major battle over access to personal health data. A research coalition had worried that draft E.U. legislation would have sharply restricted scientific use of such data. [ScienceInsider] Editorial

Seattle Genetics and Bristol-Myers Squibb Announce Initiation of Phase I/II Clinical Trial of ADCETRIS^® (Brentuximab Vedotin) in Combination with Opdivo^® (Nivolumab) in Relapsed or Refractory Non-Hodgkin Lymphoma
Seattle Genetics, Inc. and Bristol-Myers Squibb announced that the companies have initiated a Phase I/II clinical trial of ADCETRIS in combination with Opdivo for patients with CD30-expressing relapsed or refractory B-cell and T-cell non-Hodgkin lymphomas, including diffuse large B-cell lymphoma, peripheral T-cell lymphoma and cutaneous T-cell lymphoma. [Seattle Genetics, Inc.] Press Release

Alnylam Initiates Dosing in PNH Patients in Phase I/II Clinical Trial for ALN-CC5
Alnylam Pharmaceuticals, Inc. announced that it initiated the final phase of its Phase I/II clinical trial with ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. [Alnylam Pharmaceuticals, Inc.] Press Release

Cellectis Files First Clinical Trial Application for UCART19, an Allogeneic Gene Edited CAR T-Cell Product for Hematological Malignancies
Cellectis announced the submission of a clinical trial application to the Medicines & Healthcare products Regulatory Agency requesting approval to initiate UCART19 First-in-Human clinical investigation in leukemia in the United Kingdom. [Cellectis] Press Release

Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Sarepta Therapeutics, Inc. announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s new drug application for eteplirsen, on January 22, 2016 [Sarepta Therapeutics] Press Release

Dicerna Begins Dosing in First Clinical Trial of Therapy for Primary Hyperoxaluria Type 1 (PH1) in Healthy Volunteers
Dicerna Pharmaceuticals, Inc. announced it has initiated dosing in the first clinical trial of DCR-PH1, its investigational therapy for the treatment of PH1. [Dicerna Pharmaceuticals, Inc.] Press Release

Regen BioPharma, Inc. Identifies Small Molecule Drug Candidates Targeting Its Proprietary Immune Checkpoint NR2F6
Regen BioPharma, Inc., announced identification of a series of compounds that have been identified by biochemical testing which interact with the company’s newly identified immune checkpoint NR2F6. [Regen BioPharma, Inc. (PR Newswire Association LLC)] Press Release

Regen BioPharma, Inc. Receives FDA Clearance to Initiate Clinical Trial of HemaXellerate
Regen BioPharma, Inc., announced receipt of a communication from the U.S. Food and Drug Administration, allowing for initiation of clinical trials under its investigational new drug (IND) #15376. The clearance of the IND allows the company to initiate clinical trials of HemaXellerate, a personalized immune-modulatory cell therapy that has demonstrated benefit in animal models of aplastic anemia. [Regen BioPharma, Inc. (PR Newswire Association LLC)] Press Release

VistaGen Secures Exclusive Worldwide Commercial Rights to Three Proprietary Stem Cell Technologies from University Health Network
VistaGen Therapeutics, Inc. announced that it has secured exclusive worldwide commercial rights to three patent-pending stem cell technologies from University Health Network. [VistaGen Therapeutics, Inc.] Press Release

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-32495)

Draft Guidances Relating to the Regulation of Human Cells, Tissues, or Cellular or Tissue-Based Products; Public Hearing; Request for Comments; Correction (FR Doc. No: 2015-32686)

Deviation Reporting for Human Cells, Tissues, and Cellular and Tissue-Based Products; Draft Guidance for Industry; Availability (FR Doc. No: 2015-32323)

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NEW Postdoctoral Researcher – Prostate Cancer Biology (Roswell Park Cancer Institute)

NEW Postdoctoral Research Scientist – Follicular Lymphoma (Herbert Irving Comprehensive Cancer Center)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

Professor – Regenerative Vision Research (University of Montreal)

Assistant Professor – Regenerative Kidney Research (University of Montreal)

PhD/MSc Position – Cancer Research and Wound Healing (Technion, Israel Institute of Technology)

Postdoctoral Position – Cell Mechanobiology (Washington University in St. Louis)

Investigator – Cancer Biology (Wake Forest Baptist School of Medicine)

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

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

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