Cell Therapy News Volume 18.21 | Jun 19 2017

TOP STORY

Enhanced Therapeutic Efficacy and Memory of Tumor Specific CD8 T Cells by Ex-Vivo PI3K-δ Inhibition

Scientists investigated the role of upstream PI3K isoforms in CD8⁺ T cell differentiation and assessed the potential use of PI3K isoform-specific inhibitors to favorably condition CD8⁺ T cells for adoptive cell therapy. [Cancer Res] Abstract

(Ranked by impact factor of the journal)

Adenoviral Production of Interleukin-2 at the Tumor Site Removes the Need for Systemic Postconditioning in Adoptive Cell Therapy

Researchers evaluated the efficacy and safety of intratumorally administered interleukin-2 (IL-2)-coding adenoviruses in combination with tumor-infiltrating lymphocyte therapy in syngeneic Syrian hamsters bearing HapT1 pancreatic tumors and with T cell receptor transgenic adoptive cell therapy in B16.OVA melanoma bearing C57BL/6 mice. [Int J Cancer] Abstract

Enhanced Chondrogenesis of Bone Marrow-Derived Stem Cells by Using a Combinatory Cell Therapy Strategy with BMP-2/TGF-β1, Hypoxia, and COL1A1/HtrA1 siRNAs

siRNAs significantly decreased mRNA and protein levels of type I collagen and HtrA1, resulting in a more typical chondrocyte phenotype, but with frequent calcification of the subcutaneously implanted constructs in a nude mouse model. [Sci Rep] Full Article

Human Myogenic Reserve Cells Are Quiescent Stem Cells that Contribute to Muscle Regeneration after Intramuscular Transplantation in Immunodeficient Mice

Fourteen days and 21 days after cell transplantation in immunodeficient mice, live human cells were significantly more numerous and the percentage of Pax7⁺/human lamin A/C⁺ cells was two fold higher in muscles of animals injected with myogenic reserve cells (MRC) compared to those injected with human myoblasts, despite that percentage of spectrin⁺ and lamin A/C⁺ human fibers in both groups MRC were similar. [Sci Rep] Full Article

Dendritic-Cell-Based Immunotherapy Evokes Potent Anti-Tumor Immune Responses in CD105⁺ Human Renal Cancer Stem Cells

The authors evaluated the effectiveness of dendritic cells pulsed with antigen derived from CD105⁺ human renal cell carcinoma cancer stem cells against renal cancer cells in vitro and in vivo. [Mol Carcinog] Abstract

Expansion of Umbilical Cord Blood Aldehyde Dehydrogenase Expressing Cells Generates Myeloid Progenitor Cells that Stimulate Limb Revascularization

Injection of expanded progeny accelerated recovery of perfusion and improved limb usage in immunodeficient mice with femoral artery ligation-induced limb ischemia. At 7 or 28 days post-transplantation, mice transplanted with expanded ALDH^hi cells showed augmented endothelial cell proliferation and increased capillary density compared to controls. [Stem Cells Transl Med] Full Article

Development of a Cell-Based Gene Therapy Approach to Selectively Turn Off Bone Formation

Delivering adenoviral vector containing the cDNA for bone morphogenetic protein 2-transduced human mesenchymal stem cells encapsulated in poly(ethylene glycol) diacrylate hydrogel promoted ectopic ossification in vivo, and the inducible caspase-9 system allowed direct control of the timing of apoptosis of the injected cells. [J Cell Biochem] Abstract

Sciatic Nerve Regeneration by Transplantation of In Vitro Differentiated Nucleus Pulposus Progenitor Cells

Investigators assessed the applicability of mouse intervertebral disc-derived nucleus pulposus progenitor cells as a cell source for sciatic nerve regeneration. [Regen Med] Abstract

Boundary Cap Neural Crest Stem Cell Transplants Contribute Mts1/S100A4-Expressing Cells in the Glial Scar

Avulsed mice received boundary cap neural crest stem cell or human neural progenitor cell transplants and their contributions to glial scar formation and sensory axon regeneration were analyzed with immunohistochemistry and transganglionic tracing. [Regen Med] Abstract

In Vivo Murine-Matured Human CD3⁺ Cells as a Preclinical Model for T Cell-Based Immunotherapies

Scientists demonstrated that in vivo murine-matured autologous CD3⁺ T cells from humans can be collected from mice, engineered with lentiviral vectors, reinfused into the mice, and detected in multiple lymphoid compartments at stable levels over 50 days after injection. [Mol Ther Methods Clin Dev] Full Article

Autologous Hematopoietic Stem Cell Transplantation for Treatment of Multiple Sclerosis

Current evidence indicates that the patients who are most likely to benefit from and tolerate autologous haematopoietic stem cell transplantation (AHSCT) are young, ambulatory and have inflammatory multiple sclerosis (MS) activity. Clinical trials are required to rigorously test the efficacy, safety and cost-effectiveness of AHSCT against highly active MS drugs. [Nat Rev Neurol] Abstract

Translational Cardiac Stem Cell Therapy: Advancing from First-Generation to Next-Generation Cell Types

Aiming at highlighting controversies, the author provides a critical overview of the translation of first-generation and second-generation cell types. They emphasize the importance of understanding the mechanisms of cardiac repair and the lessons learned from first-generation trials, in order to improve cell-based therapies and to potentially finally implement cell-free therapies. [npj Regen Med] Full Article

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Juno Therapeutics Presents TRANSCEND NHL 001 Trial Data at International Conference on Malignant Lymphoma

Juno Therapeutics, Inc. presented data from the TRANSCEND trial of JCAR017 in relapsed and refractory aggressive B cell non-Hodgkin lymphoma (NHL). [Press release from Juno Therapeutics, Inc. (Business Wire, Inc.) discussing research presented at the 2017 International Conference on Malignant Lymphoma, Lugano] Press Release

Tessa Therapeutics and Parker Institute for Cancer Immunotherapy Enter into Strategic Alliance Agreement

Tessa Therapeutics and the Parker Institute for Cancer Immunotherapy announced the establishment of a multi-year strategic alliance to combine their capabilities to jointly advance research in the field of cancer immunotherapy. [Tessa Therapeutics Pte Ltd.] Press Release

Inovio Begins Phase III Clinical Trial of VGX-3100 for the Treatment of HPV-Related Cervical Pre-Cancer

Inovio Pharmaceuticals, Inc. announced that it has commenced its Phase III clinical program to evaluate the efficacy of Inovio’s DNA-based immunotherapy, VGX-3100, to treat cervical dysplasia caused by human papillomavirus (HPV). [Inovio Pharmaceuticals, Inc.] Press Release

TxCell Appoints Lentigen Technology, Inc. to Manufacture the Lentiviral Vector for Its First CAR-Treg Program in Transplant Rejection

TxCell SA announces the signing of a strategic agreement with Lentigen Technology, Inc. This agreement appoints LTI as TxCell’s contract manufacturing organization for the GMP production of its HLA-A2 CAR lentiviral vector. [Txcell SA] Press Release

Unum Therapeutics Announces Active Investigational New Drug (IND) Application for ACTR707 in Combination with Rituximab in Patients with Relapsed/Refractory B-Cell Lymphoma

Unum Therapeutics announced that an IND application for ACTR707 in combination with rituximab for the treatment of adult patients with relapsed/refractory CD20-positive B cell non-Hodgkin lymphoma, is now active. [Unum Therapeutics Inc.] Press Release

Genexine Receives Approval to Initiate Phase Ib/II Trial of GX-188E, HPV Therapeutic DNA Vaccine, in Combination with KEYTRUDA^® (Pembrolizumab), for the Treatment of HPV-Induced Cervical Cancer

Genexine Inc. announced that the Ministry of Food and Drug Safety in Korea has granted approval to initiate a Phase Ib/II trial of GX-188E, an HPV therapeutic DNA vaccine, in combination with KEYTRUDA^®, an anti-PD-1 therapy for the treatment of patients with HPV-induced advanced non-resectable cervical cancer. [Genexine, Inc.] Press Release

Intrexon Integrates Leading Adenoviral Gene Delivery Technology with Completion of GenVec Acquisition

Intrexon Corporation announced that it has completed the acquisition of GenVec, Inc., a clinical-stage biopharmaceutical company and pioneer in the development of AdenoVerse™ gene delivery technology platform. [Intrexon Corporation] Press Release

MilliporeSigma Awarded Its First CRISPR Patent by Australian Patent Office

MilliporeSigma announced that the Australian Patent Office has granted the company patent rights relating to the use of CRISPR in a genomic integration method for eukaryotic cells. [EMD Millipore Corporation] Press Release

Fibrocell Receives Rare Pediatric Disease Designation from FDA for FCX-013 for Treatment of Localized Scleroderma

Fibrocell Science, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to FCX-013, Fibrocell’s gene therapy candidate for the treatment of moderate to severe localized scleroderma—a chronic autoimmune disease characterized by thickening of the skin and connective tissue. [Fibrocell Science, Inc.] Press Release

AveXis and REGENXBIO Announce New Exclusive Worldwide Licenses for the Treatment of Two Rare Neurological Monogenic Disorders Using NAV AAV9 Vector

AveXis, Inc. and REGENXBIO Inc. announced an exclusive worldwide license agreement for AveXis to develop and commercialize gene therapy treatments using REGENXBIO’s NAV AAV9 vector to treat two rare neurological monogenic disorders: Rett syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 gene. [AveXis, Inc.] Press Release

AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101

AveXis, Inc. announced alignment with the U.S. Food and Drug Administration (FDA) on the company’s Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 following the receipt of minutes from the Type B Chemistry Manufacturing and Controls meeting. [AveXis, Inc.] Press Release

Horizon Discovery Announces Progress of Its Gene and Cell Therapy Platform for Contract Service and Therapeutic Applications

Horizon Discovery Group plc announced progress of its gene and cell therapy platform for contract service and therapeutic applications. The new offering will make use of Horizon’s extensive suite of gene editing technologies, including CRISPR and its proprietary rAAV and Transposon technologies, to edit pluripotent stem cells and primary human lymphocytes. [Horizon Discovery Group plc] Press Release

Macron Consolidates Electoral Victory

The party of France’s recently elected president won an absolute majority in its first general elections, with an agenda that included strong support for research. [Nature News] Editorial

Biologists Debate How to License Preprints

Biology’s zeal for preprints — papers posted online before peer review — is opening up a thorny legal debate: should scientists license their manuscripts on open-access terms? Researchers have now shared more than 11,000 papers at the popular bioRxiv preprints site. But where some researchers allow their bioRxiv manuscripts to be freely redistributed and reused, others have chosen to lock them down with restrictive terms. [Nature News] Editorial

Indian Research Labs Face Financial Crisis

India’s 38 premier scientific laboratories are in a budgetary pinch. A jump in expenditures on salaries, pensions, and perks for government employees, recommended by an advisory commission, is leaving little money for new research in the budget of the Council of Scientific & Industrial Research, based in New Delhi, which oversees the labs and their 4600 scientists. [ScienceInsider] Editorial

NIH

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No:2017-12364) Notice

NEW Gordon Research Conferences: Wnt Signaling
August 6-11, 2017
Stowe, Vermont, United States

Visit our events page to see a complete list of events in the community.

NEW Postdoctoral Scientist – Systems Oncology Group (CRUK Manchester Institute)

Quality Improvement Coordinator – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Tenure-Track Professorship – Experimental Cell Therapy (University of Bonn)

Postdoctoral Position – Tumor Immunology and Cancer Immunotherapy (VIB)

Professor – Stem Cell Biology (University College London)

Postdoctoral Researcher – Cellular Therapy for Degenerative Disc Disease (NUI Galway)

Specialist – Cell Procurement (Celgene Corporation)

Head – CAR-T Operations and Technology (Celgene Corporation)

Scientist – Analytical Development (KBI Biopharma)

PhD Fellow – Experimental Genetic Cardiology (Oslo University Hospital)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center)

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