Cell Therapy News Volume 19.41 | Dec 10 2018

TOP STORY

Identification and Modulation of Microenvironment Is Crucial for Effective MSC Therapy in Acute Lung Injury

The lung proteome was profiled in three models of injury induced by acid instillation and/or mechanical ventilation in mice. The human gene glutathione peroxidase-1 was delivered prior to mesenchymal stromal cell (MSC) administration; or MSCs carrying the human gene interleukin-10 or hepatocyte growth factor were administered after lung injury. [Am J Respir Crit Care Med] Abstract

(Ranked by impact factor of the journal)

Bioreactor-Based Mass Production of Human iPSC-Derived Macrophages Enables Immunotherapies against Bacterial Airway Infections

Bioreactor-derived induced pluripotent stem cells (iPSC)-macrophages rescued mice from Pseudomonas aeruginosa-mediated acute infections of the lower respiratory tract within four to eight hours post intra-pulmonary transplantation and reduced bacterial load. [Nat Commun] Full Article

A Dual‐AAV Approach Restores Fast Exocytosis and Partially Rescues Auditory Function in Deaf Otoferlin Knock‐Out Mice

Scientists generated two adeno‐associated viruses (AAVs), each containing half of otoferlin cDNA. Co‐injecting these dual‐AAV2/6 half‐vectors into the cochleae of six to seven‐day‐old otoferlin knock‐out mice led to the expression of full‐length otoferlin in up to 50% of inner hair cells. [EMBO Mol Med] Full Article | Graphical Abstract

Human Stem Cells Transplanted into the Rat Stroke Brain Migrate to Spleen via Lymphatic and Inflammation Pathways

Investigators tested the hypothesis that human bone marrow mesenchymal stromal cells intracerebrally transplanted in the brain of adult rats subjected to experimental stroke can migrate to the spleen, a vital organ that confers peripheral inflammation after stroke. [Haematologica] Abstract | Full Article

Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates

Researchers evaluated the utility of magnetic resonance (MR) imaging to monitor intrathecal infusions in nonhuman primates. Adeno-associated virus (AAV) spiked with gadoteridol, a gadolinium-based MR imaging contrast agent, enabled real-time visualization of infusions delivered either via cerebromedullary cistern, lumbar, cerebromedullary and lumbar, or intracerebroventricular infusion. [Mol Ther Methods Clin Dev] Abstract | Full Article

AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver

Systemic delivery of adeno-associated viral (AAV) vectors has been evaluated for the treatment of several liver diseases, including homozygous familial hypercholesterolemia, ornithine transcarbamylase deficiency, and hemophilia. The authors evaluated this approach for the treatment of Crigler-Najjar syndrome. [Mol Ther Methods Clin Dev] Full Article

Selective Ablation of Tumorigenic Cells following Human Induced Pluripotent Stem Cell‐Derived Neural Stem/Progenitor Cell Transplantation in Spinal Cord Injury

In vitro, the herpes simplex virus type 1 thymidine kinase system successfully ablated immature proliferative neural cells while preserving mature postmitotic neuronal cells. Similar results were observed in vivo following transplantation into the injured spinal cords of immune‐deficient mice. [Stem Cells Transl Med] Full Article

Immunosuppression Overcomes Insulin- and Vector-Specific Immune Responses that Limit Efficacy of AAV2/8-Mediated Insulin Gene Therapy in NOD Mice

Scientists report the restoration of euglycaemia in chemically induced diabetic C57BL/6 mice and spontaneously diabetic non obese diabetic (NOD) mice by intravenous systemic administration of a single-stranded adeno-associated virus (AAV) codon optimised vector encoding furin cleavable human proinsulin under a liver-specific promoter. [Gene Ther] Full Article

Enhanced Safety and Efficacy of Oncolytic Vesicular Stomatitis Virus Therapy by Combination with T Cell Receptor Transgenic T Cells as Carriers

Loading vesicular stomatitis virus onto CD8⁺ T central memory cells not only improved the safety compared to systemic administration of naked virus, but this approach also allowed for an effective delivery of virus to its tumor target, resulting in an effective combination therapy in NSG mice bearing subcutaneous human acute myeloid leukemia tumors. [Mol Ther Oncolytics] Abstract | Full Article

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Biomaterial-Guided Delivery of Gene Vectors for Targeted Articular Cartilage Repair

The authors provide an overview of advances in gene vector-loaded biomaterials for articular cartilage repair. Such systems enable the sustained release of gene therapies while maintaining transduction efficacy. [Nat Rev Rheumatol] Abstract

New Technologies to Enhance In Vivo Reprogramming for Regenerative Medicine

Recent advances in transcriptional control and improved transmembrane delivery strategies now offer exciting potential to more efficiently reprogram cell fates as well as to control the reprogramming timeline and scale of delivery to improve safety. [Trends Biotechnol] Abstract

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

Donald B. Kohn, MD, Orchard Therapeutics Scientific Advisory Board Member, Presents Clinical Proof-of-Concept Data for OTL-102 Gene Therapy for the Treatment of Patients with X-CGD

Orchard Therapeutics announced that scientific advisory board member, Donald B. Kohn, MD, presented clinical proof-of-concept data from an ongoing academic clinical trial evaluating OTL-102 for the treatment of X-linked chronic granulomatous disease (X-CGD). [Press release from Orchard Therapeutics discussing research presented at the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition, San Diego] Press Release

iCell Gene Therapeutics Presents First-in-Human Data of CLL1-CD33 Compound CAR T in Refractory Acute Myeloid Leukemia

iCell Gene Therapeutics, LLC announced results from a study ongoing at Chengdu Military General Hospital of ICG144, the first CLL1-CD33 Compound CAR T-cell in clinical study, in patients with particularly difficult to treat acute myeloid leukemia. [Press release from iCell Gene Therapeutics, LLC (Business Wire, Inc.) discussing research presented at the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition, San Diego] Press Release

Turnstone Biologics Unveils Novel Vaccinia Oncolytic Viral Immunotherapy Platform

Turnstone Biologics presented pre-clinical data supporting the development of a new Vaccinia therapeutic platform. [Press release from Turnstone Biologics (Business Wire, Inc.) discussing research presented at the American Association for Cancer Research (AACR) Tumor Immunology and Immunotherapy Meeting, Miami] Press Release

BlueRock Therapeutics Strengthens Collaboration with the McEwen Stem Cell Institute

BlueRock Therapeutics, LP announced the strengthening of its ongoing strategic collaboration with the McEwen Stem Cell Institute at the University Health Network in Toronto. [BlueRock Therapeutics, LP] Press Release

4D Molecular Therapeutics Announces Collaboration to Advance Gene Therapies for Retinal Diseases with Oregon Health & Science University’s Casey Eye Institute and Oregon National Primate Research Center

4D Molecular Therapeutics announced a research and development collaboration with the Casey Eye Institute and the Oregon National Primate Research Center at Oregon Health & Science University. [4D Molecular Therapeutics] Press Release

Evox Therapeutics to Expand Its Work on Cell-Targeted Delivery of Nucleic Acid-Based Drugs

Evox Therapeutics Ltd announced that it has secured an undisclosed amount of funding from the Bill & Melinda Gates Foundation. The funds will be used to support the company’s development of cell-targeted exosome-based approaches to more effectively deliver a nucleic acid-based anti-infective drug. [Evox Therapeutics Limited] Press Release

Voyager Therapeutics Announces First Patient Dosed in RESTORE-1, a Phase II Trial of VY-AADC Gene Therapy for the Treatment of Parkinson’s Disease

Voyager Therapeutics, Inc. announced dosing of the first patient in RESTORE-1, a Phase II, randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of VY-AADC for the treatment of Parkinson’s disease in patients with motor fluctuations that are refractory to medical management. [Voyager Therapeutics, Inc.] Press Release

Wave Life Sciences Announces Positive Phase I Results for WVE-210201 in Duchenne Muscular Dystrophy (DMD)

Wave Life Sciences Ltd. announced that the safety and tolerability data from the WVE-210201 Phase I clinical trial in boys with DMD who are amenable to exon 51 skipping support initiation of a Phase II/III clinical trial. [Wave Life Sciences Ltd.] Press Release

Birmingham to Lead Ground-Breaking Research into Patient Experience of Cell Therapies

Birmingham health researchers, with industry partners including health technology companies Dignio and Datatrial, have been awarded £1.1 million by UK Research & Innovation’s Innovate UK, to investigate patients’ experience of cell and gene therapies. The funding has been provided as part of the Industrial Strategy Challenge Fund’s Medicines Manufacturing program. [Birmingham Health Partners] Press Release

Despite CRISPR Baby Controversy, Harvard University Will Begin Gene-Editing Sperm

Even as a furious debate broke out in China over gene-edited babies, some scientists in the US are also hoping to improve tomorrow’s children. [MIT Technology Review] Editorial

South Africa’s Science Academy Urges Update to Nation’s Genetics Laws

South Africa needs to overhaul the laws and ethical guidelines that govern its genetics research, testing and databases, says its national science academy — especially given the country’s troubled history of race-based discrimination. [Nature News] Editorial

Despite Growing Budget, U.S. Cancer Institute Slashes Operating Budget by 5%

A good year for biomedical research funding has turned sour at the National Cancer Institute (NCI) in Bethesda, Maryland, where the director is cutting operating budgets by 5% across the agency. Despite a rising overall budget, NCI’s funds are being stretched thin by various priorities and a ballooning number of funding applications, explains NCI Director Norman “Ned” Sharpless. [ScienceInsider] Editorial

FDA

Bacterial Risk Control Strategies for Blood Collection Establishments and Transfusion Services to Enhance the Safety and Availability of Platelets for Transfusion; Draft Guidance for Industry; Availability (FR Doc. No:2018-26477) Notice

NIH

Laboratory Animal Welfare: Draft Report on Recommendations to Reduce Administrative Burden on Researchers (FR Doc. No:2018-26557) Notice

NEW Therapeutic Genome Editing Congress
March 20-21, 2019
Boston, Massachusetts, United States

NEW Tissue Engineering & Regenerative Medicine International Society – AP Chapter and the 7^th Asian Biomaterials Congress
October 14-17, 2019
Brisbane, Australia

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

NEW Assistant/Associate or Professor – Cancer Immunotherapy/Tumor Immunology (University of California, Irvine)

Faculty Position – Cancer Bioengineering (Ecole Polytechnique Fédérale de Lausanne)

Postdoctoral Position – Regenerative Medicine, Stem Cells and Bioengineering (Igenomix Foundation)

Postdoctoral Associate – Gene Therapy (University of Massachusetts)

Research Scientist – Cellular Manufacturing (California Institute for Biomedical Research)

Assistant Project Scientist – Hematology and Oncology (University of California, Davis)

Executive Director – Cell and Gene Medicine (Stanford University)

Faculty Positions – Hematology and Oncology (University of California Davis)

Lab Technologist – Cell Therapy (University of Maryland Medical Center)

Open Rank Professor – Biopharmaceutical Discovery (University of Delaware)

Assistant Professor – Biology (University of Delaware)

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