Cell Therapy News Volume 17.37 | Oct 17 2016

TOP STORY

Differentiation of Human Embryonic Stem Cells to HOXA⁺ Hemogenic Vasculature that Resembles the Aorta-Gonad-Mesonephros

The ability to generate hematopoietic stem cells from human pluripotent cells would enable many biomedical applications. Scientists found that hematopoietic CD34⁺ cells in spin embryoid bodies derived from human embryonic stem cells lack HOXA expression compared with repopulation-competent human cord blood CD34⁺ cells, indicating incorrect mesoderm patterning. [Nat Biotechnol] Abstract

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Purification of Functional Human ES and iPSC-Derived Midbrain Dopaminergic Progenitors Using LRTM1

Scientists employed a double selection strategy for cells expressing both CORIN and LMX1A::GFP, and report a cell surface marker to enrich mDA progenitors, LRTM1. When transplanted into 6-OHDA-lesioned rats, human induced pluripotent stem cells (iPSCs)-derived LRTM1⁺ cells survive and differentiate into mDA neurons in vivo, resulting in a significant improvement in motor behavior without tumor formation. [Nat Commun] Full Article

Specification of Hematopoietic Stem Cell Fate via Modulation of Mitochondrial Activity

Investigators showed that enforced modulation of energy metabolism impacts hematopoietic stem cells (HSCs) self-renewal. Lowering the mitochondrial activity of HSCs by chemically uncoupling the electron transport chain drives self-renewal under culture conditions that normally induce rapid differentiation. [Nat Commun] Full Article

Islet-Like Organoids Derived from Human Pluripotent Stem Cells Efficiently Function in the Glucose Responsiveness In Vitro and In Vivo

The authors report that pancreatic islet-like clusters derived from human pluripotent stem cells (hPSCs) are functionally capable of glucose-responsive insulin secretion as well as therapeutic effects. Pancreatic hormone-expressing endocrine cells were differentiated from hESCs using a step-wise protocol. [Sci Rep] Full Article

Effective Gene Expression in the Rat Dorsal Root Ganglia with a Non-Viral Vector Delivered via Spinal Nerve Injection

Researchers aimed to develop a spinal nerve injection strategy to deliver polyethylenimine mixed with plasmid (PEI/DNA polyplexes) containing green fluorescent protein. Using this spinal nerve injection approach, PEI/DNA polyplexes were delivered to dorsal root ganglia neurons without nerve injury. [Sci Rep] Full Article

Adoptively Transferred Donor IL-17-Producing CD4⁺ T Cells Augment, but IL-17 Alleviates, Acute Graft-versus-Host Disease

Researchers carried out studies in a murine acute graft-versus-host disease (GVHD) model of fully major histocompatibility complex-mismatched myeloablative bone marrow transplantation. They showed that donor wild-type CD4⁺ T cells exacerbated acute GVHD compared with IL-17^−/− CD4⁺ T cells, while IL-17 reduced the severity of acute GVHD. [Cell Mol Immunol] Abstract

Gene Therapy with Plasmids Encoding IFN-β or IFN-α14 Confers Long-Term Resistance to HIV-1 in Humanized Mice

Because endogenous interferon type I (IFN-I) produced by HIV-1 infection might complicate the analysis of therapeutically administered IFN-I, researchers tested different humanized mouse models for induction of IFN-I during HIV-1 infection. While HIV-1 induced high levels of IFN-α in BLT mice, IFN-I was undetectable following infection in the Hu-PBL mouse model, in which only T cells expand. [Oncotarget] Full Article

AAV Delivery of Endothelin-1 shRNA Attenuates Cold-Induced Hypertension

Investigators determined if upregulation of endothelin-1 (ET1) contributes to cold-induced hypertension. In vivo RNAi silencing of the ET1 gene was achieved by AAV2 delivery of ET1 short-hairpin siRNA. Four groups of male rats were used. [Hum Gene Ther] Abstract

A Regenerative Approach with Dermal Micrografts in the Treatment of Chronic Ulcers

Scientists demonstrated the efficacy of dermal micrografts in the treatment of ulcers with different etiologies. They also investigated in vitro the action of micrografts in the regenerative process. [Stem Cell Rev] Abstract

T Cell Receptor Excision Circle (TREC) Monitoring after Allogeneic Stem Cell Transplantation; a Predictive Marker for Complications and Clinical Outcome

The authors discuss the role of TREC analysis in the prediction of clinical outcome after allogeneic hematopoietic stem cell transplantation (HSCT). Due to the pivotal role of T cell reconstitution we propose that TREC analysis should be included as a key indicator in the post-HSCT follow-up. [Int J Mol Sci] Full Article

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New Data Presented Support Potential Benefit of Investigational Treatment Nusinersen in Spinal Muscular Atrophy

New data from the clinical program for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), were presented by Biogen and Ionis Pharmaceuticals. The presentations included safety results from the interim analysis of the Phase III ENDEAR study in infantile-onset SMA, encouraging preliminary results from NURTURE, a Phase II open-label study in pre-symptomatic infants, and a recent analysis of the ongoing Phase II open-label study in patients with later-onset SMA. [Press release from Biogen discussing research presented at 2016 World Muscle Society Congress, Grenada] Press Release

AUM LifeTech’s Next Generation FANA RNA Silencing Technology Showcased in Several Studies

AUM LifeTech, Inc. has successfully developed multiple applications for its next generation FANA RNA silencing technology to advance biomedical research and therapeutic development. New encouraging data on the use of FANA RNA silencing technology was recently presented by several of AUM’s academic collaborators from North America and Europe. [Press release from AUM LifeTech, Inc. (PR Newswire Association LLC.) discussing research presented at the annual Oligonucleotide Therapeutics Society (OTS), Montreal] Press Release

Gamida Cell Receives FDA Breakthrough Therapy Designation for NiCord^®

Gamida Cell announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to the company’s lead product candidate, NiCord^®, in development as a novel graft modality for bone marrow transplantation in patients with high risk hematological malignancies such as leukemia and lymphoma. The international, multi-center Phase III registration study of NiCord is planned to begin before the end of the year. [Gamida Cell] Press Release

Agilis Biotherapeutics Announces Authorization of Phase IIb Clinical Study of Gene Therapy for AADC Deficiency

Agilis Biotherapeutics, LLC announced that a Phase IIb clinical study for its gene therapy treatment of Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, AGIL-AADC, has been authorized by the Taiwan Food and Drug Administration and the ethics committee at National Taiwan University. AGIL-AADC is an adeno-associated virus vector containing the human gene for the AADC enzyme. [Agilis Biotherapeutics, LLC] Press Release

Biostage Provides Regulatory Update of Cellspan™ Esophageal Implant

Biostage, Inc. announced a regulatory update following its planned pre-Investigational New Drug meeting with the U.S. Food and Drug Administration for the advancement of its lead product candidate, Cellspan Esophageal Implant, into human clinical studies. [Biostage, Inc.] Press Release

WuXi AppTec Unveils Manufacturing Center for Cutting Edge Cell and Gene Therapies

WuXi AppTec celebrated the grand opening of their new 150,000 square foot biomanufacturing facility in an event attended by Pennsylvania Lieutenant Governor Mike Stack and WuXi AppTec Chairman and Chief Executive Officer, Dr. Ge Li. [WuXi AppTec] Press Release

Capricor Therapeutics Completes Enrollment in Phase II ALLSTAR Clinical Trial

Capricor Therapeutics, Inc. announced that its Phase II ALLSTAR clinical trial has completed patient enrollment. ALLSTAR (ALLogeneic Heart STem Cells to Achieve Myocardial Regeneration) is evaluating CAP-1002 in adults with cardiac dysfunction following a large heart attack. [Capricor Therapeutics, Inc.] Press Release

SELLAS™ Life Sciences Announces Positive Interim Phase II Results from its WT1 Immunotherapeutic Anti-Cancer Treatment in Multiple Myeloma Patients

SELLAS Life Sciences Group reported positive results from the company’s Phase II study of its WT1 first-in-class immunotherapeutic anti-cancer treatment in multiple myeloma patients following autologous stem cell transplantation. [SELLAS Life Sciences Group] Press Release

Regenicin, Inc. Announces Scheduled Pre-IND Meeting with U.S. Food and Drug Administration (FDA) for Its New Autologous Cultured Skin Substitute, NovaDerm^®

Regenicin, Inc. announced that they have scheduled a Pre-IND Meeting via teleconference with members of the FDA Office of Cellular, Tissue, and Gene Therapies in the Center for Biologics Evaluation and Research regarding its product NovaDerm^®. NovaDerm^®, which recently received its orphan-drug designation from the FDA, is a regenerative cell therapy that has the potential to re-grow a patient’s own skin in a laboratory by harvesting a small, stamp-size skin biopsy. [Regenicin, Inc. (PR Newswire Association LLC.)] Press Release

Mount Sinai Receives $10 Million Grant to Study Graft vs. Host Disease

Researchers from the Icahn School of Medicine at Mount Sinai have been awarded a $10 million from the National Cancer Institute to explore the cellular and molecular mechanisms of acute graft-versus-host disease, a common side effect that occurs after allogeneic bone marrow transplantation (BMT), and to develop novel therapeutic strategies for BMT patients with cancer that begin in the cells of blood-forming tissue or hematologic malignancies. [Mount Sinai Health System] Press Release

Biden’s Moonshot Cancer Plan Calls for More Data Sharing

Vice President Joe Biden released his vision for doubling progress against cancer over 5 years. It includes numerous policy recommendations and a laundry list of projects by the National Cancer Institute and other federal agencies that would require additional funding. [Science Insider] Editorial

U.S. and Cuban Biomedical Researchers are Free to Collaborate

The U.S. Department of the Treasury has authorized U.S. scientists to freely collaborate with Cuban counterparts on everything from cancer therapies to combating the Zika virus. [Science Insider] Editorial

FDA

Additions and Modifications to the List of Drug Products That Have Been Withdrawn or Removed From the Market for Reasons of Safety or Effectiveness (FR Doc. No:2016-24333) Notice

Abbreviated New Drug Applications and 505(b)(2) Applications (FR Doc. No:2016-22690) Notice

NEW Cancer Immunotherapy
November 7-8, 2016
San Francisco, United States

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NEW Postdoctoral Fellow – Anti-Tumor Immunity in Tetraspannin Deficient Mice (Radboud University Medical Center)

Postdoctoral Fellowship – Genetic Medicine (Weill Cornell Medical College)

Research Fellow in Pluripotent Stem Cells Self-Renewal Program and Cell Cycle (KU Leuven)

Faculty Position – Stem Cell Biology and Regenerative Medicine (Stanford University)

Research Associate – Hematology (Editas Medicine)

Faculty Position – Stem Cell Research (Children’s Research Institute at UTSW)

Assistant Professorship – Stem Cells and Regenerative Medicine (University of Southern California)

Postdoctoral Positions – Stem Cell Research, Hematopoiesis, Cancer, Genome Editing (Icahn School of Medicine)

Faculty Position – Cancer Research (Children’s Research Institute at UTSW)

Assistant Professor – Molecular and Cellular Biology (Harvard University)

Postdoctoral Fellow – Novel Systems for Treatment of Type 1 Diabetes (Toronto General Research Institute & McEwen Centre for Regenerative Medicine)

Assistant Professor – Developmental Biology (Florida State University)

Research Associate – Cellular Therapy (California Institute for Biomedical Research)

Postdoctoral Fellow – Pluripotent Stem Cell Technology (University of California Davis)

Associate Member – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – Pathogenic Mechanisms of Rare Bone Disorders (University of Connecticut Health)

Postdoctoral Fellow – Oxidative Lung Injury (Temple University)

Postdoctoral Fellow – Lentivirus-Mediated FOXP3 Gene Transfer (Stanford University School of Medicine)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

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

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