Cell Therapy News 19.35 October 22, 2018 | |
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TOP STORYBeneficial Effects on Vision in Patients undergoing Retinal Gene Therapy for Choroideremia Scientists designed an adeno-associated viral vector to express Rab-escort protein 1 and assessed it in a gene therapy clinical trial by subretinal injection in 14 patients with choroideremia. [Nat Med] Abstract | Press Release 1 | Press Release 2 | |
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PUBLICATIONS(Ranked by impact factor of the journal)Researchers estimated the long-term survival and value of tisagenlecleucel for children and young adults with B-cell acute lymphoblastic leukemia. [JAMA Pediatr] Abstract The authors developed two gene therapy approaches for the delivery of the therapeutic anti-HER2 monoclonal antibody, trastuzumab, to the brain. They utilized the helper dependent adenovirus vector, containing trastuzumab light and heavy chains coding sequences. [J Control Release] Abstract | Graphical Abstract Alpha 1 Antitrypsin Gene Therapy Extends the Life-Span of Lupus-Prone Mice Scientists examined the effects of human alpha 1 antitrypsin (hAAT) on the functions of dendritic cells (DCs) from lupus-prone mice and showed that hAAT treatment efficiently inhibited CpG induced activation of bone marrow-derived conventional and plasmacytoid DCs as well as the production of pro-inflammatory cytokines. [Mol Ther Methods Clin Dev] Abstract | Full Article A pro-inflammatory environment, mainly mediated by monocyte activation that leads to the activation of both T-cells and natural killer (NK) cells, was generated when oncolytic adenovirus (OAdv)-loaded menstrual blood-derived mesenchymal stem cells (MenSCs) were co-cultured with allogeneic peripheral blood mononuclear cells (PBMCs). This combination of allogeneic PBMCs and OAdv-loaded MenSCs enhanced antitumor efficacy both in vitro and in vivo, an effect partially mediated monocytes and NK cells. [Mol Cancer Ther] Abstract Scientists investigated the biodistribution, therapeutic efficacy, and mode of action of mesenchymal stem cell-extracellular vesicles (MSC-EVs) in a rat stroke model. MSC-EVs successfully stimulated neurogenesis and angiogenesis in vivo. [Transl Stroke Res] Abstract Transplanted rats, which were injected with exosomes from indoleamine 2,3-dioxygenase (IDO)-bone marrow derived mesenchymal stem cells (BMSCs), had reduced allograft-targeting immune responses and improved cardiac allograft function. Exosomes secreted by IDO-BMSCs exhibited significant upregulations of the immunoregulatory protein FHL-1, miR-540-3p, and a downregulation of miR-338-5p. [Cell Transplant] Abstract Mesenchymal Stromal Cells in the Treatment of Perianal Fistulas in Crohn’s Disease A Phase III trial demonstrated that in patients with Crohn’s disease and refractory complex perianal fistulas, a single injection of 120 × 106 cells was superior to local injection of placebo associated with the same surgical procedure, in obtaining closure of the fistula tracts together with absence of abscesses >2 cm, 24 weeks after the injection, a stringent combined primary end point. [Immunotherapy] Abstract In an in vivo mouse model, intratumoral administration of cationic niosomes-carrying anti-luciferase short interfering RNA (siRNA) led to significant suppression of luciferase expression compared with naked siRNA. [J Liposome Res] Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSPromise of Gene Therapy to Treat Sickle Cell Disease The authors focus on techniques to treat sickle cell anemia by ex vivo genetic manipulation of hematopoietic stem/progenitor cells, emphasizing replacement gene therapy and gene editing. [Expert Opin Biol Ther] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSIntellia Therapeutics Presents New Data in In Vivo and Ex Vivo Programs Intellia Therapeutics, Inc. presented new data from three of its programs, including the company’s first data on complex edits. [Press release from Intellia Therapeutics, Inc. discussing research presented at the 26th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), Lausanne] Press Release Rocket Pharmaceuticals, Inc. presented updated long-term clinical data from its ongoing Phase I/II clinical trial of RP-L102 for Fanconi Anemia. [Press release from Rocket Pharmaceuticals, Inc. discussing research presented at the 26th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), Lausanne] Press Release uniQure N.V. presented non-clinical data demonstrating that a next-generation synthetic promoter developed for liver-directed gene therapy is able to drive gene expression at increased levels and with very high specificity, allowing uniQure to tailor expression levels required for a specific therapeutic transgene. [Press release from uniQure N.V. discussing research presented at the 26th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), Lausanne] Press Release | |
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INDUSTRY NEWSSQZ Biotechnologies announced the expansion of its collaboration with Roche in cellular therapy. The expanded partnership furthers the synergistic combination of SQZ’s innovation and expertise in cell therapy with Roche’s cancer immunotherapy expertise. [SQZ Biotechnologies] Press Release RepliCel Announces Federal Grant Funding for Collaboration with the University of Victoria RepliCel Life Sciences Inc. announced it has signed a collaborative research project agreement with the University of Victoria. The project will be co-funded through a grant from the National Science and Engineering Research Council of Canada (NSERC) under the NSERC Collaborative Research and Development program. [RepliCel Life Sciences Inc. (PR Newswire Association LLC.)] Press Release Anixa Biosciences, Inc. announced that it, along with its partner Moffitt Cancer Center, completed a pre-Investigational New Drug (IND) meeting with the FDA. [Anixa Biosciences, Inc.] Press Release Aglaris Talks Automated and Closed Cell Therapy Platform Aglaris has developed an autonomous, closed production system for the manufacture of cell therapies such as T-cells and mesenchymal stem cells. According to new CEO Steven Docksey, the platform overcomes many issues associated with this new product class. [Aglaris (BioProcess International)] Press Release Scientists at the University of California Los Angeles Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and the Stein Eye Institute have been awarded a $5.1 million grant from the California Institute for Regenerative Medicine to advance the development of a novel therapy for blinding retinal conditions. [University of California Los Angeles] Press Release Krystal Biotech, Inc. announced positive interim results from its ongoing placebo-controlled Phase I/II clinical trial of KB103. [Krystal Biotech, Inc.] Press Release Transgene announced that the first patient of a Phase I/II clinical trial evaluating TG6002 in patients with advanced gastrointestinal tumors, such as colon cancers, has been dosed at Centre Léon Bérard. This multicenter trial has already been approved in France, Belgium and Spain and will enroll up to 59 patients. [Transgene] Press Release Immusoft Receives Rare Pediatric Disease Designation for Treatment of MPS I Immusoft Corporation announced that the FDA has granted it Rare Pediatric Disease Designation for Iduronicrin genleukocel-T, Immusoft’s Sleeping Beauty transposon-engineered autologous plasmablasts for the expression and delivery of alpha-L-iduronidase to treat mucopolysaccharidosis type I (MPS I). [Immusoft Corporation (Business Wire, Inc.)] Press Release Homology Medicines, Inc. announced long-term efficacy data of a single dose of its lead gene therapy candidate for the treatment of phenylketonuria, an inborn error of metabolism. [Homology Medicines, Inc.] Press Release | |
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POLICY NEWSFeds Crack Down on Stem Cell Clinics that Touted Autism Treatments, Blindness Cures For the first time, the Federal Trade Commission has cracked down on stem cell clinics for overzealous marketing claims, filing a complaint against two California clinics that promoted their treatments for everything from autism to Parkinson’s despite a lack of evidence. [STAT News] Editorial British Universities Set Up European Outposts as Brexit Looms Some of Britain’s leading research institutions are establishing alliances with counterparts in other European countries — a move that might allow them to keep drawing on the EU’s science funds even in the case of a ‘no-deal’ divorce from the bloc, a more extreme form that Brexit could take. [Nature News] Editorial UK’s Science Reputation ‘at Risk if Academic Visa Issues Not Resolved’ The Wellcome Trust, which grants more than £1bn for research each year, said the immigration system was “not up to scratch” after another scientific summit in the UK was marred by the visa barriers delegates and speakers faced. [The Guardian] Editorial Cancer Research to Cut Funding for Scientists Who Bully Colleagues Britain’s largest cancer charity has announced new rules to crack down on bullying and harassment. Cancer Research UK said scientists who bullied or harassed colleagues would face sanctions that could include being prevented from supervising PhD students, losing funding from the charity or being barred from applying for future grants. [The Guardian] Editorial
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JOB OPPORTUNITIESNEW Faculty Positions – Hematology and Oncology (University of California Davis) PhD and Posdoctoral Research Positions – Innate Immunity (Universitatsklinikum Tubingen) Lab Technologist – Cell Therapy (University of Maryland Medical Center) Quality Control Scientists/Analysts – Cell and Gene Therapy (GlaxoSmithKline) Postdoctoral Fellow – Stem Cell Biology/Stroke, and Alzheimer’s Disease Research (Tulane University) Open Rank Professor – Biopharmaceutical Discovery (University of Delaware) Assistant Professor – Biology (University of Delaware) Research Associate – Immunotherapy (Baylor College of Medicine) Director – Clinical Development (California Institute for Biomedical Research) Faculty Position – Adoptive T Cell Therapy (Dana-Farber Cancer Institute) Professorship – Somatic Gene Therapy (University of Zurich) Associate Professor – Cancer Research (University of Maryland) Senior Scientist – CAR-T Cell Research (CELLECTIS Inc.) 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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