Cell Therapy News 19.30 September 10, 2018 | |
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TOP STORYIn Vivo Reprogramming of Wound-Resident Cells Generates Skin Epithelial Tissue Researchers generated expandable epithelial tissues using in vivo reprogramming of wound-resident mesenchymal cells. Transduction of four transcription factors that specify the skin-cell lineage enabled efficient and rapid de novo epithelialization from the surface of cutaneous ulcers in mice. [Nature] Abstract | Press Release | |
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PUBLICATIONS(Ranked by impact factor of the journal)Gene Editing Restores Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy Scientists used adeno-associated viruses to deliver CRISPR gene editing components to four dogs and examined dystrophin protein expression six weeks after intramuscular delivery or eight weeks after systemic delivery. After systemic delivery in skeletal muscle, dystrophin was restored to levels ranging from 3 to 90% of normal, depending on muscle type. [Science] Abstract | Press Release | Editorial Argininosuccinic Aciduria Fosters Neuronal Nitrosative Stress Reversed by Asl Gene Transfer Investigators showed that cerebral disease in argininosuccinic aciduria involves neuronal oxidative/nitrosative stress independent of hyperammonemia. Intravenous injection of adeno-associated virus serotype 8 vector into adult or neonatal argininosuccinate lyase (ASL)-deficient mice demonstrates long-term correction of the hepatic urea cycle and the cerebral citrulline-nitric oxide cycle, respectively. [Nat Commun] Full Article Researchers report generation of a high-fidelity CRISPR/Cas9-based gene-specific dioxygenase by fusing an endonuclease deactivated high-fidelity Cas9 to TET3 catalytic domain, targeted to specific genes by guiding RNAs. [Nat Commun] Full Article Following gene therapy with systemic administration of recombinant adeno-associated virus serotype-6 (AAV6)-microdystrophin to mdx/mTRG2 mice, scientists demonstrated the development of antibodies targeting dystrophin and AAV6 capsid in control mice. [Proc Natl Acad Sci USA] Abstract | Press Release In vitro generated 3D tissues exhibited stable phenotype for over one year in culture, providing an attractive resource for long-term in vitro studies. 3D derived tissue provided critical liver support in two animal models, including immunocompetent recipients. [Arch Toxicol] Abstract | Press Release In vivo i.v. administration of intercellular adhesion molecule-nanostructured lipid carrier/protamine/angiopoietin significantly attenuated pulmonary TNF-α and IL-6 levels, inflammatory cell infiltration, and led to positive histological improvements in lipopolysaccharide-induced acute lung injury mice. [Nanomedicine] Abstract Menstrual blood-derived mesenchymal stem cells (MenSCs) were transplanted into incomplete thoracic spinal cord injury rats, and all rats were sacrificed at 7, 14, and 28 days after surgery. Investigators found that MenSC transplantation improved hind limb motor function. [Cell Death Dis] Full Article Scientists investigated migration, differentiation, and synaptic connections of transplanted otic epithelial progenitors derived from human induced pluripotent stem cells in mouse cochlea. [Stem Cell Res Ther] Full Article Investigators observed the curative effect of surgery combined with gene therapy on small hepatocellular carcinoma. 54 patients underwent gene therapy one or two times before operation, whereas 23 patients underwent surgery alone selected by themselves. [Cancer Gene Ther] Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSRNAi/CRISPR Screens: From a Pool to a Valid Hit Scientists provide a critical assessment of the various RNAi/CRISPR approaches to pooled screens and discuss their advantages and pitfalls. They specify a set of best practices for key parameters enabling a reproducible screen and provide a detailed overview of analysis methods and repositories for identifying the best candidate gene hits. [Trends Biotechnol] Abstract The Biological Basis and Clinical Symptoms of CAR-T Therapy-Associated Toxicites The authors review the wide spectrum of effects associated with CAR-T cell therapy with a major focus on the pathogenesis of cytokine release syndrome and neurotoxicity as the most common, potentially life-threatening effects of this treatment. [Cell Death Dis] Full Article Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSSangamo Therapeutics, Inc. reported 16 week reductions in urinary glycosaminoglycans, a key biomarker of Mucopolysaccharidosis Type II (MPS II) disease pathophysiology, in Cohort 2 of the Phase I/II CHAMPIONS Study evaluating SB-913. [Press release from Sangamo Therapeutics, Inc. discussing research presented at the 2018 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), Athens] Press Release bluebird bio, Inc. announced updated results from the Phase II/III Starbeam study (ALD-102) of its investigational Lenti-Dâ„¢ gene therapy in boys 17 years of age and under with cerebral adrenoleukodystrophy (CALD), and initial data from ALD-103, the ongoing observational study of outcomes from allogeneic hematopoietic stem cell transplant in boys 17 years of age and under with CALD. [Press release from bluebird bio, Inc. discussing research presented at the 2018 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), Athens] Press Release | |
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INDUSTRY NEWSCCRM and JSRM Sign MOU to Accelerate Canadian and Japanese Advances in RM CCRM and the Japanese Society for Regenerative Medicine (JSRM) have agreed to collaborate to advance the field of regenerative medicine (RM) and cell and gene therapies in Canada and Japan, signing a Memorandum of Understanding (MOU) this week. [CCRM] Press Release Deloitte and Vineti Will Team on End-to-End Solution to Support Personalized Medicine Deloitte and Vineti Inc. announced that they have entered into a teaming agreement, announcing their intent to work together to support life sciences innovators by integrating and scaling their respective supply chain and patient engagement platforms to enable more efficient access to next-generation cellular therapies. [Deloitte (PR Newswire Association LLc.)] Press Release Fibrocell Science, Inc. announced that the FDA has granted Fast Track Designation to FCX-013, the company’s clinical stage candidate for the treatment of moderate to severe localized scleroderma—a chronic, debilitating genetic skin disorder with no FDA approved therapies. [Fibrocell Science, Inc.] Press Release Kite announced that the European Commission has granted Marketing Authorization for Yescarta® as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy. [Gilead Sciences, Inc.] Press Release First CRISPR Clinical Trial Backed by U.S. Companies Launches The first clinical trial of CRISPR-Cas9 sponsored by U.S. companies has launched, testing the genome-editing technique in patients with the blood disorder beta thalassemia, according to an announcement on the U.S. clinical trials website. [STAT News] Editorial First iPS Cell Trial for Heart Disease Raises Excitement, Concern Early next year, a small clinical trial will begin in Japan, marking the first time reprogrammed stem cells will be deployed to help regenerate injured hearts. A team led by Osaka University cardiac surgeon Yoshiki Sawa will implant sheets—each consisting of 100 million stem-cell derived cardiomyocytes—onto the hearts of three patients with advanced heart failure. [The Scientist] Editorial 2018 António Champalimaud Vision Award Winners Announced This year recognises the research teams led by Jean Bennett, Albert Maguire, Robin Ali, James Bainbridge, Samuel Jacobson and William Hauswirth, for the development of gene therapy for the Leber Congenital Amaurosis, a genetic form of childhood blindness, whose genetic cause was discovered by Michael Redmond, also awarded. Their work resulted in the first gene therapy cure for an inherited human disease. [Champalimaud Foundation] Press Release UK Rejects Gilead’s CAR-T Cancer Cell Therapy as Too Expensive A cutting-edge CAR-T cell therapy for otherwise untreatable forms of blood cancer is too expensive to justify its use on Britain’s state-funded health service, the country’s healthcare cost agency NICE said. [Reuters] Press Release Immusoft Receives $3.5 Million Grant to Expand upon Its Cell Therapy Platform Immusoft Corporation announced that it has received a Phase II Small Business Innovation Research grant from the National Institute of General Medical Sciences, part of the National Institutes of Health. [Immusoft Corporation (Business Wire, Inc.)] Press Release Cynata CYP-001 Stem Cell Therapy Meets All Safety and Efficacy Endpoints in Phase I Trial in GvHD Cynata Therapeutics Limited announced that CYP-001, its lead Cymerusâ„¢ mesenchymal stem cell product candidate, met all clinical endpoints in a Phase I trial for the treatment of steroid-resistant acute graft-versus-host disease (GvHD). [Cynata Therapeutics Limited (GlobeNewswire, Inc.)] Press Release Reflection Biotechnologies Limited announced that the FDA has granted Orphan Drug Designation to ReflectionBio’s RBIO-101 program, an AAV-based gene therapy product for treating BCD. [Reflection Biotechnologies Limited (Business Wire, Inc.)] Press Release Lysogene announced that the FDA has granted an Investigational New Drug (IND) clearance to proceed in the U.S. with its international, Phase II-III clinical trial of LYS-SAF302 for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA). [Lysogene] Press Release Poseida Therapeutics Inc. announced data results from the first eleven patients treated in its ongoing Phase I study of its P-BCMA-101 stem cell memory CAR-T product in relapsed/refractory multiple myeloma. [Poseida Therapeutics Inc.] Press Release Celsion Corporation announced it has begun dosing patients in the recently initiated OVATION II Study, the company’s randomized, Phase I/II clinical trial of GEN-1, its DNA-based immunotherapy for the localized treatment of ovarian cancer as an adjuvant to chemotherapy current standard of care. [Celsion Corporation] Press Release | |
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POLICY NEWSPivotal CRISPR Patent Battle Won by Broad Institute A fierce and unprecedented patent battle between two educational institutions may be nearing a close, after a US appeals court issued a decisive ruling on the rights to CRISPR-Cas9 gene-editing. [Nature News] Editorial Peer Reviewers in Developing Nations Are Underrepresented, Report Suggests Combined, scientists spend nearly 70 million hours peer reviewing manuscripts for scholarly journals every year, a new report says. But too much of the onus rests on researchers in wealthy countries, according to the report by the company Publons—based in London and Wellington—which enables researchers to track and claim credit for the peer reviews they perform. [ScienceInsider] Editorial
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REGULATORYFDADraft Guidances Relating to the Development of Human Gene Therapy Products; Availability; Extension of Comment Period (FR Doc. No:2018-19303) Notice
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EVENTSNEW Cell & Gene Therapy Conference Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Professorship – Somatic Gene Therapy (University of Zurich) NEW Postdoctoral Staff Fellow – Biologist/Biomedical Engineer (Food and Drug Administration) NEW Associate Professor – Cancer Research (University of Maryland) Molecular Biologist (STEMCELL Technologies Inc.) Senior Scientist – CAR-T Cell Research (CELLECTIS Inc.) Postdoctoral Fellow – Immuno-Oncology (CELLECTIS Inc.) Postdoctoral Position – Gene Therapy (Genethon) Research Assistant or Associate – Cell Therapy (California Institute for Biomedical Research) Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope) Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.
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