Cell Therapy News Volume 19.26 | Jul 30 2018

TOP STORY

Simple In Vivo Gene Editing via Direct Self-Assembly of Cas9 Ribonucleoprotein Complexes for Cancer Treatment

Investigators developed a carrier-free Cas9 ribonucleoprotein delivery system for robust gene editing in vivo. For simultaneous delivery of Cas9 and a guide RNA into target cells without the aid of any transfection reagents, they established a multifunctional Cas9 fusion protein that forms a ternary complex with synthetic crRNA:tracrRNA hybrids in a simple procedure. [ACS Nano] Abstract | Graphical Abstract

(Ranked by impact factor of the journal)

Interferon Gene Therapy Reprograms the Leukemia Microenvironment Inducing Protective Immunity to Multiple Tumor Antigens

Scientists showed that monocyte-mediated gene delivery of IFNα inhibited leukemia in a mouse model. IFN gene therapy counteracted leukemia-induced expansion of immunosuppressive myeloid cells and imposed an immunostimulatory program to the tumor microenvironment, as shown by bulk and single-cell transcriptome analyses. [Nat Commun] Full Article

Repeated Intrathecal Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis

The authors assessed the safety and efficacy of two repeated intrathecal injections of autologous bone marrow-derived mesenchymal stem cells in amyotrophic lateral sclerosis. [Ann Neurol] Abstract

Reducing Ex Vivo Culture Improves the Anti-Leukemic Activity of Chimeric Antigen Receptor (CAR)-T Cells

Scientists compared the therapeutic potential of early versus late harvested CAR targeting CD19 (CART19) in a murine xenograft model of acute lymphoblastic leukemia and showed that the anti-leukemic activity inversely correlated with ex vivo culture time: day three harvested cells showed robust tumor control despite using a six-fold lower dose of CART19, whereas day nine cells failed to control leukemia at limited cell doses. [Cancer Immunol Res] Abstract

Disease-Modifying Osteoarthritis Treatment with Interleukin-1 Receptor Antagonist Gene Therapy in Small and Large Animal Models

Investigators report the development of a helper-dependent adenovirus-mediated intraarticular gene therapy approach for long-term expression of interleukin-1 receptor antagonist for sustained symptomatic and disease-modifying osteoarthritis therapy. [Arthritis Rheumatol] Abstract

Functional Tissue-Engineered Microtissue Derived from Cartilage Extracellular Matrix for Articular Cartilage Regeneration

Articular chondrocyte- or adipose-derived stem cell-based microtissues were directly implanted in vivo to repair articular osteochondral defects in a rabbit model. [Acta Biomater] Abstract

Effects of FGF21-Secreting Adipose-Derived Stem Cells in Thioacetamide-Induced Hepatic Fibrosis

Investigators established FGF21-secreting adipose derived stem cells to enhance the effects of ADSCs and transplanted them into thioacetamide-induced liver fibrosis mice via the tail vein. [J Cell Mol Med] Abstract

Endoscopic Transplantation of Mesenchymal Stem Cell Sheets in Experimental Colitis in Rats

Researchers isolated adipose-derived mesenchymal stem cells (MSCs) and bone marrow-derived mesenchymal stem cells from EGFP-transgenic rats and fabricated the cells in sheet forms using temperature-responsive culture dishes. The MSC sheets were endoscopically transplanted to the inflamed area in electrocoagulation and DNBS colitis model. [Sci Rep] Full Article

Cell Spray Transplantation of Adipose-Derived Mesenchymal Stem Cell Recovers Ischemic Cardiomyopathy in a Porcine Model

Human adipose-derived mesenchymal stem cells (ADSCs) were acquired from white adipose tissue. Porcine ischemic cardiomyopathy models were established by constriction of the left anterior descending coronary artery. ADSCs were spread over the surface of the heart via cell spray in fibrinogen and thrombin solutions. [Transplantation] Abstract

Transplanted Neural-Like Cells Improve Memory and Alzheimer-Like Pathology in a Rat Model

The authors investigated the histological and physiological effects of transplantation the neuron-like cell-derived mouse embryonic stem cells on the repair of brain lesions in an Alzheimer’s animal model in rats. [Cytotherapy] Abstract

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CAR T Cells for Infection, Autoimmunity and Allotransplantation

The authors explored the challenges and opportunities of using this technology to target infectious diseases such as with HIV and undesired immune responses such as autoimmunity and transplant rejection. [Nat Rev Immunol] Abstract

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

Neuralstem Announces Presentation of Preclinical Data Demonstrating Neurogenic Compound NSI-189 Improves Cognition and Anxiety in a Mouse Model of Alzheimer’s Disease

Neuralstem, Inc. announced the presentation of data demonstrating that oral administration of NSI-189 in a mouse model of Alzheimer’s Disease leads to a significant amelioration and/or improvement in cognition measures and anxiety. [Press release from Neuralstem, Inc. discussing research presented at the 2018 Alzheimer’s Association International Conference, Chicago] Press Release

Foundation for the National Institutes of Health and Cancer Research Institute Announce New Collaboration in Cancer Immunotherapy

The Foundation for the National Institutes of Health (FNIH) and the Cancer Research Institute (CRI) announced today that FNIH has selected CRI to be its source of landscape intelligence in immuno-oncology, also called cancer immunotherapy. [Foundation for the National Institutes of Health, Inc.] Press Release

bluebird bio’s LentiGlobin™ Gene Therapy Granted Accelerated Assessment by European Medicines Agency for the Treatment of Transfusion-Dependent β-Thalassemia

bluebird bio, Inc. announced that its investigational LentiGlobin™ gene therapy for the treatment of adolescent and adult patients with transfusion-dependent β-thalassemia and a non-β⁰/β⁰ genotype, was granted an accelerated assessment by the Committee for Medicinal Products for Human Use of the European Medicines Agency, for its upcoming marketing authorization application [bluebird bio, Inc.] Press Release

Sarepta Therapeutics Announces that Phase I/IIa Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial Placed on Clinical Hold Due to an Out-of-Specification Production Lot; No Observed Safety Events

Sarepta Therapeutics, Inc. has been notified by the Research Institute at Nationwide Children’s Hospital that they have received a letter from the FDA on July 24, 2018, stating that their Phase I/IIa DMD Micro-Dystrophin Gene Therapy Trial has been placed on clinical hold due to the presence of a trace amount of DNA fragment in research-grade third-party supplied plasmid. [Sarepta Therapeutics, Inc.] Press Release

GSK and Adaptimmune Complete Transition of NY-ESO SPEAR T-Cell Therapy Program to GSK

GlaxoSmithKline plc and Adaptimmune Therapeutics plc announced the transition of the development programme for GSK3377794, an NY-ESO SPEAR T-cell therapy, to GSK. [GlaxoSmithKline plc] Press Release

UC San Diego Researchers Receive Funding to Pursue Novel Stem Cell-Based Treatments

The governing board of the California Institute for Regenerative Medicine unanimously approved two grants worth more than $2.2 million to University of California San Diego School of Medicine researchers investigating stem cell-based therapies for a rare genetic disorder that affects the heart and a chronic, progressive affliction of the lungs. [University of California San Diego School of Medicine] Press Release

Ultragenyx Announces First Patient Dosed in Phase I/II study of DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa); FDA Grants Fast Track Designation to Gene Therapy Program

Ultragenyx Pharmaceutical Inc. announced that the first patient has been dosed in the Phase I/II study of DTX401, an adeno-associated virus vector based gene therapy for the treatment of patients with GSDIa. [Ultragenyx Pharmaceutical Inc.] Press Release

Orgenesis Announces Collaboration between Secant Group and Atvio Biotech, a Wholly Owned Subsidiary of Masthercell Global, to Develop and Commercialize Scaffold Technologies for Advanced Cell Therapies

Orgenesis Inc. announced that Atvio Biotech Ltd., which is wholly-owned by Orgenesis’ Masthercell Global subsidiary, and Secant Group are collaborating to develop and commercialize biodegradable and injectable scaffold technologies for use in Orgenesis’ advanced cell therapies. [Orgenesis Inc.] Press Release

BrainStorm Announces Grant of a New European Patent for NurOwn^®

BrainStorm Cell Therapeutics Inc. announced that the European Patent Office has granted an European-wide patent entitled ‘Mesenchymal Stem Cells for the Treatment of CNS Diseases’ for the treatment of Amyotrophic Lateral Sclerosis with NurOwn^®. [BrainStorm Cell Therapeutics Inc.] Press Release

Omeros’ OMS721 Receives European Orphan Drug Designation Positive Opinion for Treatment in Hematopoietic Stem Cell Transplantation

Omeros Corporation announced that the European Medicines Agency’s Committee for Orphan Medicinal Products issued a positive opinion recommending orphan drug designation of OMS721 for treatment in hematopoietic stem cell transplantation. [Omeros Corporation] Press Release

Celyad Announces FDA Acceptance of IND Application for CYAD-101, a First-in-Class Non-Gene Edited Allogeneic CAR-T Candidate

Celyad announced that the FDA has accepted the company’s Investigational New Drug (IND) application for CYAD-101, the first non-gene edited allogeneic clinical program. [Celyad] Press Release

Government of Canada Further Strengthens Science and Encourages Scientists to Speak about Their Work

Canadians count on federal scientists to produce the evidence needed to keep their air clean, their food safe and their water fresh. The Government of Canada is taking greater steps to ensure that this evidence is gathered without political or commercial interference. [Government of Canada] Editorial

Trump Desperately Needs a Science Adviser, Experts Say. He Just Doubled the Record for Time Without One

President Trump can add another tick in the records column: He doubled the length of time that any modern president has gone without a science adviser. [Washington Post] Editorial

UK-India Science Ministers Reinforce Strong Research Partnership as Future Collaboration Is Announced

The UK and Indian governments signaled their commitment to a deeper relationship by jointly announcing four new awards worth £4.8 million under the Civil Nuclear Energy program and seven new awards worth £10.8 million under Phase 2 of the Global research program on health. [GOV.UK] Editorial

New Evidence USCIS Policies Increased Denials of H-1B Visas

New data show that in 2017, U.S. Citizenship and Immigration Services (USCIS) denied many more H-1B petitions and issued numerous costly Requests for Evidence to stop highly educated foreign nationals from working in America. [Forbes Media LLC] Editorial

Third Retraction for Harvard Cancer Biologist

Nature has issued a retraction of a cancer study, seven years after its publication, citing issues with two figures and the unavailability of original data for them. The paper, led by Harvard Medical School biologist Sam Lee, is among several publications from his group to be retracted. [The Scientist] Editorial

NIH

Prospective Grant of an Exclusive Patent License: Development and Commercialization of Cancer Immunotherapy (FR Doc. No:2018-16058) Notice

NEW Cell Therapies and Bioengineering Conference
September 20-22, 2018
San Francisco, California, United States

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

NEW Research Assistant or Associate – Cell Therapy (California Institute for Biomedical Research)

Postdoctoral Scientist – Novel T Cell Based Cancer Therapies (Netherlands Cancer Institute – Antoni van Leeuwenhoek)

Research Associate – Neuroimmunology (Thomas Jefferson University)

Postdoctoral Fellow – DNA Damage Repair and Pediatric Sarcoma Therapy (University of Texas Health Science Center at San Antonio)

Postdoctoral Position – Corneal Diseases (University of California, Los Angeles)

PhD Student – Cellular Immunotherapy (Glycostem Therapeutics)

Research Associate/PhD Position – iPS Cell-Derived Neurons to Model Parkinson’s Disease (TU Dresden)

Postdoctoral Fellow – Cell and Gene Therapies for Cardiac Arrhythmias and Heart Failure (Duke University)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

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