Cell Therapy News Volume 15.44 | Nov 10 2014

Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells Using CRISPR/Cas9
Researchers report CRISPR/Cas9 targeting of two clinically relevant genes, B2M and CCR5, in primary human CD4⁺ T cells and CD34⁺ hematopoietic stem and progenitor cells. [Cell Stem Cell]
Abstract | Graphical Abstract | Press Release

Transdifferentiation of Human Fibroblasts to Endothelial Cells: Role of Innate Immunity
Scientists show that toll-like receptor 3 agonist Poly I:C, combined with exogenous EC growth factors, transdifferentiated human fibroblasts into ECs. Induced ECs significantly improved limb perfusion and neovascularization in the murine ischemic hindlimb. [Circulation] Abstract | Press Release

Pravastatin Reverses Obesity-Induced Dysfunction of Induced Pluripotent Stem Cell-Derived Endothelial Cells via a Nitric Oxide-Dependent Mechanism
Metabolic abnormalities of induced pluripotent stem cell-derived endothelial cells (iPSC-ECs) from obese individuals could potentially limit their therapeutic efficacy for peripheral vascular disease. Investigators compared the function of iPSC-ECs from normal and diet-induced obesity mice using comprehensive in vitro and in vivo assays. [Eur Heart J] Abstract

Adiposity Induces Lethal Cytokine Storm after Systemic Administration of Stimulatory Immunotherapy Regimens in Aged Mice
Investigators determined the effects of adiposity on systemic immunotherapy (IT) tolerance in aged compared with young obese mice. Both young ob/ob- and diet-induced obese-generated proinflammatory cytokine levels and organ pathologies are comparable to those in aged ad libitum mice after IT, culminating in lethality. [J Exp Med] Abstract | Press Release

Intravenous Autologous Bone Marrow Mononuclear Stem Cell Therapy for Ischemic Stroke: A Multicentric, Randomized Trial
Investigators explored the efficacy and safety of autologous bone marrow mononuclear stem cells (BMSCs) in subacute ischemic stroke. This was a Phase II, multicenter, parallel group, randomized trial with blinded outcome assessment that included 120 patients. [Stroke] Abstract

NOTCH-Mediated Maintenance and Expansion of Human Bone Marrow Stromal/Stem Cells: A Technology Designed for Orthopedic Regenerative Medicine
Researchers demonstrate that transient JAGGED1-mediated NOTCH signaling promotes human bone marrow-derived stromal/stem cell maintenance and expansion while increasing their skeletogenic differentiation capacity, both ex vivo and in vivo. [Stem Cells Transl Med] Abstract

Optimized Cell Survival and Seeding Efficiency for Craniofacial Tissue Engineering Using Clinical Stem Cell Therapy
Using cell therapy, researchers report the upper jaw reconstruction of a patient who lost teeth and 75% of the supporting jawbone following injury. A mixed population of bone marrow-derived autologous stem and progenitor cells was seeded onto β-tricalcium phosphate, which served as a scaffold to deliver cells directly to the defect. [Stem Cells Transl Med] Abstract

A Rationally Designed Nanoparticle for RNA Interference Therapy in B-Lineage Lymphoid Malignancies
Researchers provide unprecedented experimental evidence for a previously unrecognized causal link between dysfunctional CD22 due to deletion of Exon 12 (CD22ΔE12) and aggressive biology of B-precursor acute lymphoblastic leukemia cells by demonstrating that siRNA-mediated knockdown of CD22ΔE12 in primary BPL cells is associated with a marked inhibition of their clonogenicity. [EBioMedicine] Full Article | Press Release

Prostate Cancer Immunotherapy: Beyond Immunity to Curability
The authors present advances in the past two decades of basic, translational, and clinical research in prostate cancer, including bioengineering B-cell and T-cell responses, and ongoing prostate cancer immunotherapy trials. [Cancer Immunol Res] Abstract

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bluebird bio to Present LentiGlobin Clinical Data
bluebird bio, Inc., a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, announced that data from its ongoing Phase I/II HGB-205 and Northstar Studies of LentiGlobin BB305 Drug Product will be presented. [Press release from bluebird bio, Inc. discussing research to be presented at the 56^th Annual Meeting of the American Society of Hematology, San Francisco] Press Release

Galena Biopharma Presents GALE-301 (Folate Binding Protein Immunotherapy) Phase IIa Clinical Trial Data
Galena Biopharma, Inc. announced that data from the company’s Phase I/IIa trial of GALE-301, a Folate Binding Protein-derived immunotherapy, was presented. GALE-301 is a peptide immunotherapy given as an adjuvant treatment to prevent recurrences in high-risk, ovarian and endometrial cancer patients rendered disease-free after completing standard of care therapy. [Press release from Galena Biopharma, Inc. discussing research presented at the Society for Immunotherapy of Cancer (SITC) 29^th Annual Meeting, National Harbor] Press Release

OncoSec Medical to Present New Clinical Data at Melanoma Bridge 2014
OncoSec Medical Inc. will present new data from clinical studies evaluating the company’s ImmunoPulse immunotherapy in patients with melanoma. Data from the two clinical studies evaluating the safety and efficacy of intratumoral electroporation of plasmid interleukin-12 in patients with metastatic melanoma will be featured in separate abstract discussion sessions. [Press release from OncoSec Medical Inc. discussing research to be presented at Melanoma Bridge 2014, Naples] Press Release

China’s Plans for Research-Grant Reforms Get Cautious Welcome
China quietly announced a reform plan aimed at overhauling its competitive scientific funding system. The new system, although only vaguely defined, will take the bulk of competitive funding and redirect it through five new channels. [Nature News] Editorial

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Felicitex Therapeutics and Selvita Initiate Strategic Collaboration to Target Cancer Quiescence
Felicitex Therapeutics and Selvita announced that they have entered into a strategic collaboration to develop breakthrough personalized cancer therapeutics for some of the deadliest and most resistant cancers, such as pancreatic, colon, ovarian, lung and hematopoietic tumors. [Felicitex Therapeutics Inc.] Press Release

Case Western Reserve University to Study Pluristem’s PLX-RAD Cells in Umbilical Cord Blood Transplants for the Treatment of Blood Cancers and Genetic Diseases
Pluristem Therapeutics Inc. announced that researchers at Case Western Reserve University will conduct a preclinical study of the company’s PLacental eXpanded (PLX)-RAD cells. The study will evaluate whether PLX-RAD cells increase the success rate of human umbilical cord blood transplantation. [Pluristem Therapeutics Inc.] Press Release

Seattle Children’s Begins Recruiting Patients for Immunotherapy Research Trial for Neuroblastoma, One of the Deadliest Forms of Childhood Cancer
Seattle Children’s announced the opening of patient enrollment for its new cellular immunotherapy clinical research trial designed to induce remission in children suffering from neuroblastoma, one of the deadliest forms of childhood cancer. [Seattle Children’s Hospital] Press Release

Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65
Spark Therapeutics announced that its lead product candidate, SPK-RPE65, has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA). SPK-RPE65 targets a group of blinding conditions known as inherited retinal dystrophies caused by autosomal recessive mutations in the RPE65 gene, and for which there is currently no pharmacologic treatment available. [Spark Therapeutics, Inc.] Press Release

ViaCyte Receives U.S. Patent Claiming Alternative Methods to Manufacture Pancreatic Progenitor Cells In Vitro
ViaCyte, Inc. announced it has been granted U.S. patent 8,859,286, which describes methods for manufacture of human pancreatic progenitor cells from definitive endoderm cells, by the U.S. Patent and Trademark Office. [ViaCyte, Inc.] Press Release

Advaxis Submits Investigational New Drug Application for ADXS31-142 (ADXS-PSA) for the Treatment of Metastatic Castration Resistant Prostate Cancer
Advaxis, Inc. announced that the company has submitted an Investigational New Drug application to the United States Food and Drug Administration to conduct the first-in-human study of ADXS31-142 for the treatment of metastatic castration resistant prostate cancer. [Advaxis, Inc.] Press Release

FDA Issues IND Number for Regen BioPharma’s dCellVax Therapeutic Application, a Proposed Treatment for Breast Cancer
Regen BioPharma, Inc. announced issuance of IND (Investigational New Drug Application) number 16200 from the FDA for a proposed Phase I/II clinical trial assessing safety with signals of efficacy of the dCellVax gene silenced dendritic cell immunotherapy for treating breast cancer. [Regen BioPharma, Inc. (Marketwire L.P.)] Press Release

Nuvilex Applies in Australia for the Orphan Drug Designation for Pancreatic Cancer Treatment
Nuvilex, Inc. announced that it has submitted, through its subsidiary Nuvilex Australia, an application to the Therapeutic Goods Administration in Australia to obtain the Orphan Drug Designation for its pancreatic cancer treatment. [Nuvilex, Inc.] Press Release

Specification of the Unique Facility Identifier System for Drug Establishment Registration; Guidance for Industry; Availability (FR Doc. No: 2014-26397)

Agency Information Collection Activities; Proposed Collection; Comment Request; Investigational New Drug Applications (FR Doc. No: 2014-26304)

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Postdoctoral Fellow – Developmental, Stem Cell, and/or Cancer Biology (North Carolina State 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)

Postdoctoral Position – Leukemic Stem Cells (BC Cancer Agency)

Postdoctoral Fellowship – AAV Gene Therapy (Icahn School of Medicine at Mount Sinai)

Postdoctoral Position – Novel Mediators of Cardiovascular Disease and Novel Therapeutic Strategies (Monzino Cardiology Center)

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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