Cell Therapy News Volume 15.48 | Dec 8 2014

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
Conjugation of small interfering RNA (siRNA) to an asialoglycoprotein receptor ligand derived from N-acetylgalactosamine facilitates targeted delivery of the siRNA to hepatocytes in vitro and in vivo. [J Am Chem Soc] Abstract | Graphical Abstract

Tolerance Induction to Human Stem Cell Transplants with Extension to Their Differentiated Progeny
Scientists show that brief induction with monoclonal antibody-mediated coreceptor and costimulation blockade enables long-term engraftment and tolerance towards murine ESCs, hESCs, human induced pluripotent stem cells and hESC-derived progenitors in outbred murine recipients. [Nat Commun] Abstract | Press Release

Mybpc3 Gene Therapy for Neonatal Cardiomyopathy Enables Long-Term Disease Prevention in Mice
Scientists show successful long-term ​cardiac myosin-binding protein C 3 (​Mybpc3) gene therapy in homozygous Mybpc3-targeted knock-in mice, which genetically mimic these human neonatal cardiomyopathies. [Nat Commun] Abstract

Anti-CD33 Chimeric Antigen Receptor Targeting of Acute Myeloid Leukemia
Scientists generated a second generation CD33-specific chimeric antigen receptor capable of redirecting cytolytic effector T cells against leukemic cells. Chimeric antigen receptor-modified T cells efficiently killed leukemia cell lines and primary tumor cells in vitro. [Haematologica] Abstract | Full Article

Ex Vivo-Expanded but Not In Vitro-Induced Human Regulatory T Cells Are Candidates for Cell Therapy in Autoimmune Diseases Thanks to Stable Demethylation of the Foxp3 Regulatory T Cell-Specific Demethylated Region
Researchers undertook a systematic comparison of expanded regulatory T cells (Tregs) and induced Tregs to recommend the most suitable for clinical implementation, and then tested their effectiveness and feasibility in rheumatoid arthritis. [J Immunol] Abstract

Chemokine-Mediated Robust Augmentation of Liver Engraftment: A Novel Approach
For in vivo studies, investigators used a nude mouse model of partial hepatectomy followed by intraparenchymal injections of chemokine ligands at various concentrations. [Stem Cells Transl Med] Abstract

Tissue Engineering of Rat Bladder Using Marrow-Derived Mesenchymal Stem Cells and Bladder Acellular Matrix
When mesenchymal stem cells (MSCs) were seeded on acellular matrix scaffold, they remained viable and proliferative while adopting a cellular phenotype consistent with their microenvironment. Upon transplantation in rats after partial cystectomy, MSC-seeded bladder acellular matrix (BAM) proved superior to unseeded BAM with animals recovering nearly 100% normal bladder capacity for up to six months. [PLoS One] Full Article

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Benefits Persist in T Cell Therapy for Children with Relapsed Leukemia
An innovative cell therapy against a highly aggressive form of acute lymphoblastic leukemia continues to show highly promising results in children treated in a pilot study. Ninety-two percent of the 39 children receiving bioengineered T cells had no evidence of cancer at one month after treatment, with this complete response persisting in some cases for more than two years. [Press release from EurekAlert discussing research presented at the annual meeting of the American Society of Hematology (ASH), San Francisco] Press Release

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Renewal of R&D Tax Credit Seems Like Sure Bet after U.S. House Vote
Better than nothing—but barely. That is how many lawmakers and business groups are reacting to a vote by the U.S. House of Representatives to retroactively revive a tax credit that allows companies to write off certain research expenses. [ScienceInsider] Editorial

FDA Approves BLINCYTO™ (Blinatumomab) Immunotherapy for the Treatment of Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia
Amgen announced that the U.S. Food and Drug Administration (FDA) has granted approval of BLINCYTO™ for the treatment of patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia. [Amgen Inc.] Press Release

Prothena Initiates NEOD001 Global Phase III Registrational Trial Based on Positive Results in Ongoing Phase I/II Study of NEOD001 in Patients with AL Amyloidosis
Prothena Corporation plc announced the initiation of the VITAL Amyloidosis Study, an international, multi-center, registrational Phase III clinical trial, based on positive results from an ongoing Phase I/II clinical study of NEOD001 in patients with AL amyloidosis and persistent organ dysfunction. [Prothena Corporation plc]
Press Release

CEL-SCI Expands Its Pivotal Phase III Immunotherapy Head and Neck Cancer Trial with Addition of Seven More Clinical Sites
CEL-SCI Corporation announced that it has added seven new clinical sites to its global pivotal Phase III head and neck cancer trial for its investigational immunotherapy Multikine. [CEL-SCI Corporation] Press Release

TxCell Announces Start of Phase IIb Clinical Trial with Ovasave^® for Refractory Crohn’s Disease
TxCell SA announced that it has enrolled the first patient in its Phase IIb clinical trial of its lead product Ovasave^® in refractory Crohn’s disease. [TxCell SA] Press Release

Juno Therapeutics Executes License for Phase I CAR T Product Candidate Targeting CD22 for Hematological Malignancies
Juno Therapeutics, Inc. announced that it has entered into an agreement to obtain a license from Opus Bio, Inc. for a CAR-T cell product candidate targeting CD22, a protein expressed on most B cell leukemias and lymphomas. [Juno Therapeutics, Inc.] Press Release

Alnylam Files Clinical Trial Application (CTA) for ALN-CC5, an RNAi Therapeutic Targeting Complement C5 in Development for the Treatment of Complement-Mediated Diseases
Alnylam Pharmaceuticals, Inc. announced that it has filed a CTA with the U.K. Medicines and Healthcare products Regulatory Agency to initiate a 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

Pfizer Expands Rare Disease Research with Establishment of Gene Therapy Platform
Pfizer Inc. announced two strategic decisions to expand the company’s rare disease research and development activities through the establishment of a gene therapy platform to investigate potential treatments for patients. [Pfizer Inc.] Press Release

The Pacific Meso Center Receives $100,000 Donation for Ongoing Stem Cell Research Program
Citing the need to invest in a long-range cure, Roger Worthington of the Worthington & Caron, P.C. donated $100,000 to The Pacific Meso Center at The Pacific Heart, Lung & Blood Institute to support its ongoing stem cell research program. [The Pacific Heart, Lung & Blood Institute] Press Release

Content and Format of Labeling for Human Prescription Drug and Biological Products; Requirements for Pregnancy and Lactation Labeling (FR Doc. No: 2014-28241)

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

NEW Scientist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

Postdoctoral Researcher – Cell Based Therapy for ALS (University of Warmia and Mazury)

Mesenchymal Stem Cell (MSC) Laboratory Technician (California Cryobank)

Clinical Laboratory Scientist – Umbilical Cord Blood Processing (California Cryobank)

Postdoctoral Researcher – Chromatin and Epigenetic Regulation (Van Andel Research Institute)

Animal Surgery – Regenerative Medicine for Cardiac and Skeletal Muscle Disease (Duke University)

Postdoctoral Fellow – Collaborative Training Program in Musculoskeletal Health Research (Western University)

Postdoctoral Position – Muscle Cell Therapy (I-Stem)

Graduate Student – Pediatric Cancers (University of Calgary)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

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