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Pulmonary Cell News| Cell Therapy News 20.16 May 6, 2019 | |
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TOP STORYAnti-BCMA CAR T-Cell Therapy bb2121 in Relapsed or Refractory Multiple Myeloma In this Phase I study involving patients with relapsed or refractory multiple myeloma, the authors administered bb2121 as a single infusion at doses of 50×106, 150×106, 450×106, or 800×106 CAR-positive (CAR+) T cells in the dose-escalation phase and 150×106 to 450×106 CAR+ T cells in the expansion phase. [N Engl J Med] Abstract | Press Release | |
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PUBLICATIONS(Ranked by impact factor of the journal)Subretinal injection of adeno-associated vector serotype-8-soluble CX3CL1 significantly prolonged cone survival in three strains of retinitis pigmentosa mice. Rescue of cones was accompanied by improvements in visual function. [Proc Natl Acad Sci USA] Abstract Investigators studied adult patients with acute myeloid leukemia harboring deletion 5q or monosomy 5 (-5/5q-) having their first allogeneic transplantation between 2000 and 2015. Five hundred and one patients with -5/5q- have been analyzed. Three hundred thirty-eight patients were in first remission and 142 had an active disease at time of allogeneic transplantation. [Haematologica] Abstract Transplantation of immature human embryonic stem cell-derived cardiomyocytes resulted in substantial myocardial implants within the infarct scar that matured over time, formed vascular networks with the host, and evoked minimal cellular rejection. [Stem Cell Reports] Full Article In a rat model for central nervous system demyelination, aging suppressed the capability of systemically transplanted mesenchymal stem cells to boost oligodendrocyte progenitor cell differentiation during remyelination. [Glia] Full Article Researchers investigated the therapeutic potential of clinical-grade human neural stem cells that have been successfully used in a recently concluded Phase I clinical trial for amyotrophic lateral sclerosis patients. [Cell Death Dis] Full Article Scientists isolated Müller glia from retinal organoids formed by human induced pluripotent stem cells in vitro and showed their ability to partially restore visual function in rats depleted of retinal ganglion cells by NMDA. [Stem Cells Transl Med] Full Article Investigators evaluated the effect of a porous silk fibroin scaffold seeded with bone mesenchymal stem cells on nerve regeneration, myelination and functional recovery after spinal cord injury. [Biochem Biophys Res Commun] Abstract Scientists determined whether defects in lymphocyte differentiation and function in DOCK8-deficient patients were restored following hematopoietic stem cell transplantation. DOCK8-deficient T and B lymphocytes exhibited aberrant activation and effector function in vivo and in vitro. [JCI Insight] Abstract | Full Article Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSScaffold‐Based Delivery of Nucleic Acid Therapeutics for Enhanced Bone and Cartilage Repair The authors provide an overview of the current state‐of‐the‐art in the field of gene‐activated scaffolds and their use within orthopaedic tissue engineering applications. [J Orthop Res] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSAveXis announced interim data from ongoing trials of the investigational product Zolgensma® that showed positive results across a broad spectrum of patients with SMA. [Press release from AveXis, Inc. discussing research presented at 2019 American Academy of Neurology (AAN) Annual Meeting, Philadelphia] Press Release Neurocrine Biosciences, Inc. and Voyager Therapeutics, Inc. announced Phase I trial results for VY-AADC from eight patients with Parkinson’s disease who participated in the open-label trial to evaluate the safety and efficacy of VY-AADC and to further assess the posterior surgical delivery approach. [Press release from Neurocrine Biosciences, Inc. discussing research presented at 2019 American Academy of Neurology (AAN) Annual Meeting, Philadelphia] Press Release Tessa Therapeutics announced ‘proof-of-concept’ data from the preclinical study of TT16, a first-of-its-kind combination immunotherapy that integrates Chimeric Antigen Receptor T cell therapy and oncolytic adenovirus expressing immunomodulatory molecules for the treatment of human epidermal growth factor receptor 2 (HER2)-positive solid tumors. [Press release from Tessa Therapeutics discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Homology Medicines, Inc. announced the presentation of data highlighting its gene therapy and gene editing platform and manufacturing capabilities. [Press release from Homology Medicines, Inc. discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Adaptimmune Therapeutics plc new and compelling data during an oral presentation about its off-the-shelf SPEAR T-cell program. [Press release from Adaptimmune Therapeutics plc discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Adverum Biotechnologies, Inc. announced new long-term safety and expression data out to 30 months following a single intravitreal injection of ADVM-022 in a preclinical study in wet age-related macular degeneration. [Press release from Adverum Biotechnologies, Inc. discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Audentes Therapeutics, Inc. announced new positive data from ASPIRO, the Phase I/II clinical trial of AT132 for the treatment of X-linked myotubular myopathy. [Press release from Audentes Therapeutics, Inc. (PR Newswire Association LLC.) discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Rocket Pharmaceuticals Presents Preclinical Data of RP-A501 for Danon Disease Rocket Pharmaceuticals, Inc. presented preclinical data of RP-A501. [Press release from Rocket Pharmaceuticals, Inc. discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release MaxCyte announced that Robert Keefe, Director of Technical Operations, provided an oral presentation on April 29, titled “Novel mRNA-based Autologous CAR Therapies in Oncology,”. [Press release from MaxCyte, Inc. discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Abeona Therapeutics Inc. reported new preclinical data demonstrating that ABO-401, the company’s novel gene therapy for cystic fibrosis (CF), efficiently delivered a highly-expressed, functional copy of human mini-CFTR to the lung of CF mice and restored CFTR function in human CF patient nasal and bronchial epithelial cells. [Press release from Abeona Therapeutics Inc. discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Amicus Therapeutics Presents Positive Preclinical Pompe Disease Gene Therapy Data Amicus Therapeutics announced initial preclinical data from its investigational adeno-associated viral gene therapy program for Pompe disease in mice. [Press release from Amicus Therapeutics discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Cell Medica announced that its collaborators from Baylor College of Medicine and Texas Children’s Hospital presented the latest positive progress in the GINAKIT2 trial for children with relapsed or refractory high risk neuroblastoma. [Press release from Cell Medica discussing research presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release Abeona Therapeutics Inc. announced that intravitreal administration of the company’s novel AIM™ AAV204 capsid to non-human primates led to robust transgene expression in the inner and outer retina. [Press release from Abeona Therapeutics Inc. discussing research presented at the 2019 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Vancouver] Press Release Homology Medicines, Inc. announced the presentation of preclinical data demonstrating that, with a single dose, the company’s suite of novel adeno-associated virus vectors targeted cell types frequently associated with inherited retinal diseases. [Press release from Homology Medicines, Inc. discussing research presented at the 2019 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Vancouver] Press Release Adverum Biotechnologies, Inc. presented for the first time preclinical data on sequentially dosing a contralateral eye with ADVM-022 gene therapy. [Press release from Adverum Biotechnologies, Inc. discussing research presented at the 2019 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Vancouver] Press Release | |
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INDUSTRY NEWSTmunity Therapeutics, Inc. announced it has entered into an exclusive licensing and research collaboration with the University of California (UC) San Francisco aimed at advancing novel engineered T cell receptor therapies for rare and currently incurable pediatric conditions with high mortality rates. [Tmunity Therapeutics, Inc.] Press Release First-in-Human Trial Shows Promise in Treating Neuroblastoma Neuroblastoma is the most common extracranial solid cancer in children, and patients with high-risk disease are challenging to treat. In a promising development, researchers at Baylor College of Medicine released initial findings from the first two patients treated in a Phase I, first-in-human clinical trial of a new form of immunotherapy to treat neuroblastoma using genetically engineered natural killer T cells. [Baylor College of Medicine] Press Release Orgenesis Inc. announced it has received Institutional Review Board approval to collect liver biopsies from patients at Rambam Medical Center located in Haifa, Israel for a planned study to confirm the suitability of liver cells for personalized cell replacement therapy for patients with insulin-dependent diabetes resulting from total or partial pancreatectomy. [Orgenesis Inc.] Press Release First US Use of CRISPR to Directly Target Cancer Will Seek Go-Ahead from Regulators If all goes as planned, the first clinical trial in the United States testing CRISPR against cancer by altering the DNA of tumor cells inside patients could begin recruiting participants next year, the scientist leading the effort told STAT. [STAT News] Press Release Dicerna™ Pharmaceuticals, Inc. announced the submission of an updated investigational new drug (IND) application to the FDA for DCR-PHXC, the company’s lead GalXC™ product candidate for the treatment of all forms of PH. [Dicerna™ Pharmaceuticals, Inc.] Press Release Inovio Pharmaceuticals, Inc. announced that its lead Phase III product candidate, VGX-3100, has been granted an Advanced Therapy Medicinal Product Certificate for quality and non-clinical data. [Inovio Pharmaceuticals, Inc.] Press Release BioTime, Inc. announced the issuance of a Notice of Allowance for a patent from the United States Patent and Trademark Office for United States Patent Application No. 15/156,316 for a method of reducing spinal cord injury-induced parenchymal cavitation in patients that have suffered an acute spinal cord injury. [BioTime, Inc.] Press Release PharmaCyte Biotech Advances Manufacturing Process for Clinical Trial in Pancreatic Cancer PharmaCyte Biotech, Inc. announced that advances have been completed in the manufacturing process for the clinical trial product that will be used in PharmaCyte’s planned clinical trial in locally advanced, inoperable pancreatic cancer. [PharmaCyte Biotech, Inc.] Press Release Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have been awarded a $4.6 million grant from the California Institute for Regenerative Medicine – also known as CIRM – to support a Phase I clinical trial of a novel treatment for advanced sarcomas and other cancers with a specific tumor marker called NY-ESO-1. [University of California] Press Release Researchers Ready B Cells for Novel Cell Therapy Scientists at Seattle Children’s Research Institute are paving the way to use gene-edited B cells – a type of white blood cell in the immune system – to treat a wide range of potential diseases that affect children, including hemophilia and other protein deficiency disorders, autoimmune diseases, and infectious diseases. [Seattle Children’s Research Institute] Press Release | |
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POLICY NEWSNIH Imposes Term Limits for Lab Chiefs The National Institutes of Health’s intramural research program will begin imposing 12-year term limits on laboratory leaders, Science reports. The policy, which goes into effect next year, aims to expand the diversity of those in management positions who oversee research groups, most of which are currently held by white men. [The Scientist] Editorial German Research Promised a Decade of Budget Increases German research organizations cheered a decision announced by state and federal ministers to increase research budgets by 3% a year for the next decade—a total boost of €17 billion over that time. [ScienceInsider] Editorial
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EVENTSNEW ISCT North America 2019 Regional Meeting Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Postdoctoral Position – Biomedical Engineering (US Food and Drug Administration) Stem Cell Podcast Co-Host (Stem Cell Podcast) Postdoctoral Research Fellow – Translational Gene Therapy (Fred Hutchinson Cancer Research Center) Postdoctoral Associate – Cancer Immunology (Winship Cancer Institute) DPhil Scholarship – Retinal Gene Therapy (University of Oxford) 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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