Cell Therapy News 20.14 April 22, 2019 | |
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TOP STORYGene Therapy Restores Immunity in Infants with Rare Immunodeficiency Disease A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. [Press release from the National Institute of Allergy and Infectious Diseases discussing online prepublication in The New England Journal of Medicine] Press Release 1 | Abstract | Press Release 2 | |
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PUBLICATIONS(Ranked by impact factor of the journal)A Safe and Potent Anti-CD19 CAR T Cell Therapy Scientists performed a Phase I trial of CD19-BBz(86) CAR T cell therapy in patients with B cell lymphoma. Complete remission occurred in 6 of 11 patients who each received a dose of 2×108–4×108 CD19-BBz(86) CAR T cells. [Nat Med] Abstract | Press Release In Utero Gene Editing for Monogenic Lung Disease Using a CRISPR fluorescent reporter system, investigators demonstrated that precisely timed in utero intra-amniotic delivery of CRISPR-Cas9 gene editing reagents during fetal development results in targeted and specific gene editing in fetal lungs. Pulmonary epithelial cells are predominantly targeted in this approach, with alveolar type 1, alveolar type 2, and airway secretory cells exhibiting high and persistent gene editing. [Sci Transl Med] Full Article | Press Release Researchers performed a non-randomized, open-label, Phase I/II clinical study in pediatric patients with severe Wiskott-Aldrich syndrome, defined by either WAS gene mutation or absent Wiskott-Aldrich syndrome protein expression or a Zhu clinical score of 3 or higher. [Lancet Haematol] Full Article The authors treated mucopolysaccharidosis type-I mice with an artificial immunization protocol to mimic the enzyme replacement therapy effect in patients and they demonstrated that alpha-L-iduronidase (IDUA)-corrected hematopoietic stem cell engraftment was impaired in pre-immunized animals by IDUA-specific CD8+ T cells spared by pre-transplant irradiation. [Mol Ther] Abstract Non-human-primates with pre-existing αAAV-Abs were used to investigate the impact of αAAV9-Abs levels on transduction efficiency of rAAV9 via systemic delivery. No significant differences were observed in vector genome biodistribution in animals with ≤1:400 total serum αAAV9-IgG compared to αAAV9-Ab-negative animals, following an IV rAAV9-hNAGLUop injection. [Mol Ther Methods Clin Dev] Full Article Anti-CD166/4-1BB Chimeric Antigen Receptor T Cell Therapy for the Treatment of Osteosarcoma The ability of CD166.BBζ CAR-T cells to kill CD166+ osteosarcoma cells was evaluated in vitro and in vivo. Intravenous injection of CD166.BBζ CAR-T cells into mice resulted in the regression of the tumor with no obvious toxicity. [J Exp Clin Cancer Res] Full Article Investigators demonstrated that neural stem cell-small extracellular vesicles can significantly reduce the extent of spinal cord injury, improve functional recovery, and reduce neuronal apoptosis, microglia activation, and neuroinflammation in rats. [Cell Death Dis] Full Article DNA-Encoded Bispecific T Cell Engagers and Antibodies Present Long-Term Antitumor Activity Scientists describe the development of a designed synthetic DNA plasmid, which they optimized to permit high expression of an anti-HER2 antibody (HER2dMAb) and delivered it into animals through adaptive electroporation. HER2dMAb was efficiently expressed in vitro and in vivo, reaching levels of 50 μg/ml in mouse sera. [JCI Insight] Full Article Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSThe authors discuss the factors that affect the outcome of cell therapy in Parkinson’s disease, with focus on the cell types used and the functional effects of the host immune response on graft survival and differentiation. [Gene Ther] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSDystrogen Therapeutics’ Gene Therapy Shows Promise for Huntington’s DYST 201, a gene therapy targeting the mutated forms of the gene responsible for Huntington’s disease, has shown promising results, Dystrogen Therapeutics announced. [Press release from Dystrogen Therapeutics (BioNews Services, LLC) discussing research to be presented at the 44th Federation of European Biochemical Societies (FEBS) Congress and the 24th Annual Meeting of the RNA Society, Krakow] Press Release Celyad announced that clinical data from the SHRINK and alloSHRINK Phase I trials, evaluating the safety of NKG2D-based autologous and allogeneic CAR-T candidates, CYAD-01 and CYAD-101, respectively, will be presented. [Press release from Celyad discussing research to be presented at the European Society for Medical Oncology (ESMO) 21st World Congress on Gastrointestinal Cancer (WCGIC), Barcelona] Press Release Cell Medica Collaborator, Baylor College of Medicine, Showcasing Promise of CAR-NKT Therapies Cell Medica announced that its collaborators from Baylor College of Medicine and Texas Children’s Hospital will be presenting the latest progress related to its innovative CAR therapy utilizing natural killer T cells (CAR-NKT). [Press release from Cell Medica discussing research to be presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Axovant Gene Therapies Ltd. announced two upcoming oral presentations and one poster presentation. [Press release from Axovant Gene Therapies Ltd. discussing research to be presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release BioTime to Present Data from OpRegen® Phase I/IIa Clinical Study BioTime, Inc. announced that updated results from a Phase I/IIa clinical study of its lead product candidate, OpRegen®, a retinal pigment epithelium cell transplant therapy currently in development for the treatment of dry age-related macular degeneration with geographic atrophy, will be presented. [Press release from BioTime, Inc. discussing research to be presented at the 2019 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Vancouver] Press Release | |
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INDUSTRY NEWSVineti Inc. announced a new partnership to advance and scale Marker Therapeutics’ T cell therapy pipeline. Marker Therapeutics is a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications. [Vineti Inc. (GlobeNewswire, Inc.)] Press Release CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced that the FDA has granted Fast Track Designation for CTX001 for the treatment of transfusion-dependent beta thalassemia. [CRISPR Therapeutics] Press Release Precision BioSciences announced it has dosed the first patient in the Phase I/IIa clinical trial of PBCAR0191, its first gene-edited allogeneic anti-CD19 CAR T cell product candidate. [Precision BioSciences] Press Release Two Patients Treated with CRISPRed Cells in Immunotherapy Trial Doctors have infused cells edited using CRISPR-Cas9 into two patients in a trial conducted at the University of Pennsylvania. A university spokesperson confirmed in an emailed statement to The Scientist that the trial is underway and that two patients, one with multiple myeloma and one with sarcoma, have been treated so far. [The Scientist] Editorial US Patent & Trademark Office Issues Patent on Anixa Biosciences CAR-T Cancer Treatment Technology Anixa Biosciences, Inc. announced that the United States Patent and Trademark Office has issued US Patent No. 10,259,855. This is the first patent covering Anixa’s CAR-T cancer treatment technology. [Anixa Biosciences, Inc.] Press Release Mesoblast Limited announced that the FDA has agreed that Mesoblast can submit on a rolling basis a Biologics License Application for its allogeneic cellular medicine remestemcel-L in children with steroid-refractory acute graft versus host disease. [Mesoblast Limited] Press Release PPMD Awards $100,000 Grant to Nationwide Children’s Hospital to Further Explore GALGT2 Gene Therapy Parent Project Muscular Dystrophy (PPMD) announced that Drs. Paul Martin and Kevin Flanigan from the Research Institute at Nationwide Children’s Hospital have completed work under a $100,000 grant exploring GALGT2 gene therapy technology as a potential treatment for Duchenne. [Parent Project Muscular Dystrophy] Press Release Sernova Corp. announced the first Cell Pouch™ transplant of insulin-producing cells in its US Phase I/II clinical trial evaluating the safety and efficacy of the cell pouch for clinical islet transplantation in type 1 diabetes. [Sernova Corp.] Press Release | |
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POLICY NEWSMajor U.S. Cancer Center Ousts ‘Asian’ Researchers after NIH Flags Their Foreign Ties The MD Anderson Cancer Center has ousted three senior researchers after the U.S. National Institutes of Health (NIH) in Bethesda, Maryland, informed it that the scientists had committed potentially “serious” violations of agency rules involving confidentiality of peer review and the disclosure of foreign ties. [ScienceInsider] Editorial How US-China Political Tensions Are Affecting Science In the latest twist, several US universities are expected this month to announce the actions they have taken against foreign scientists caught breaking rules concerning National Institutes of Health funding, according to comments made by agency director Francis Collins to the Senate Appropriations Committee. [Nature News] Editorial
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EVENTSNEW European CanCer Organisation (ECCO) 2019: European Cancer Summit Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Postdoctoral Associate – Cancer Immunology (Winship Cancer Institute) Research Technologist – Immunology (STEMCELL Technologies Inc.) Stem Cell Podcast Co-Host (Stem Cell Podcast) DPhil Scholarship – Retinal Gene Therapy (University of Oxford) Gene Editing Specialist – Rare Genetic Disorders (Novo Nordisk) Cell Biologist – Regenerative Medicine (Hepatx Corporation) Associate Scientist – Immuno-Oncology (Hackensack Meridian Health Center for Discovery & Innovation) Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma) Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center) Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University) Faculty Positions – Hematology and Oncology (University of California, Davis) 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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