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Pulmonary Cell News| Cell Therapy News 20.13 April 15, 2019 | |
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TOP STORYGene Correction for SCID-X1 in Long-Term Hematopoietic Stem Cells Researchers achieved high levels of targeting frequencies in CD34+ HSPCs from six severe combined immunodeficiency (SCID)-X1 patients and demonstrated rescue of lymphopoietic defect in a patient derived HSPC population in vitro and in vivo. [Nat Commun] Full Article | |
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PUBLICATIONS(Ranked by impact factor of the journal)Therapeutic Potential of Patient iPSC-Derived iMelanocytes in Autologous Transplantation A modified hair follicle reconstitution assay in vivo showed that MITF+PAX3+TYRP1+ human iPSC-derived melanocytes were localized in the mouse hair bulb and epidermis and produced melanin up to seven weeks after transplantation, whereas MITF+PAX3+TYRP1− hiMelanocyte stem cells integrated into the bulge-subbulge regions. [Cell Rep] Full Article | Graphical Abstract TALEN® gene editing of BCMA CAR T was used to confer lymphodepletion resistance and reduced GvHD potential. The safety profile of allogeneic BCMA CAR T was further enhanced by incorporating a CD20 mimotope-based intra-CAR off-switch enabling effective CAR T elimination in the presence of rituximab. [Mol Ther] Abstract Impact of Pre-Existing Immunity on the Non-Clinical Pharmacodynamics of AAV5-Based Gene Therapy To better define the relationship between various forms of pre-existing adeno-associated virus (AAV) immunity and gene transfer, researchers studied Valoctocogene roxaparvovec in cynomolgus monkeys with varying pre-dose levels of neutralizing anti-AAV antibodies and non-antibody transduction inhibitors. [Mol Ther Methods Clin Dev] Abstract With the capacity to propagate regulatory compliant human corneal endothelial cells (CEnCs), scientists evaluated the functionality of propagated CEnCs delivered via tissue-engineered endothelial keratoplasty or corneal endothelial cell injection within a rabbit model of bullous keratopathy. [Sci Rep] Full Article Investigators report the therapeutic effects of a single injection of human umbilical cord mesenchymal stem cells on acute and chronic colitis. [Sci Rep] Full Article Researchers analyzed correlations between visual acuity prognosis and baseline characteristics of Leber’s hereditary optic neuropathy after rAAV2‐ND4 gene therapy. [Clin Exp Ophthalmol] Abstract The authors assessed the therapeutic effect of intravenously injected bone morphogenetic protein receptor type 2‐augmented rat bone marrow‐derived endothelial‐like progenitor cells on pulmonary arterial hypertension in the rat monocrotaline model. [Respirology] Abstract Scientists investigated whether local injection of stromal vascular fraction (SVF) can reduce established fibrosis in a rat model of chronic phase of Peyronie’s disease (PD). Eighteen-male 12-wk-old Sprague-Dawley rats were divided in three equal groups: sham, PD without treatment (PD) and PD treated with SVF. [Int J Impot Res] Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSThe Landscape of Cellular and Gene Therapy Products: Cost, Approvals, and Discontinuations The authors examine the approval characteristics, discontinuations and cost of all cellular and gene therapy products approved worldwide. [Hum Gene Ther Clin Dev] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSAbeona Therapeutics to Present Data for ABO-401 in Cystic Fibrosis and Retinal Disorders Abeona Therapeutics Inc. announced that new data demonstrating the capabilities of the AIM™ capsid library in cystic fibrosis and retinal disorders will be presented. [Press release from Abeona Therapeutics Inc. discussing research to be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting, Washington DC] Press Release Homology Medicines, Inc. announced upcoming presentations highlighting its gene therapy and gene editing platform, including its phenylketonuria and metachromatic leukodystrophy programs, as well as its manufacturing capabilities. [Press release from Homology Medicines, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting, Washington DC] Press Release uniQure N.V. announced that six data presentations, of which three are oral presentations, will be delivered. [Press release from uniQure N.V. discussing research to be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting, Washington DC] Press Release Amicus Therapeutics to Present Preclinical Pompe Disease Gene Therapy Data Amicus Therapeutics announced the company will present initial preclinical data from its investigational adeno-associated viral gene therapy program for Pompe disease in a poster. [Press release from Amicus Therapeutics discussing research to be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting, Washington DC] Press Release Voyager Therapeutics Announces Upcoming Data Presentations Voyager Therapeutics, Inc., a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, announced multiple data presentations [Press release from Voyager Therapeutics, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting, Washington DC] Press Release Orchard Therapeutics announced the achievement of clinical proof-of-concept based on data from the trial of its ex vivo, autologous, hematopoietic stem cell gene therapy, OTL-300, for the treatment of transfusion-dependent beta-thalassemia. [Press release from Orchard Therapeutic discussing research to be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting, Washington DC] Press Release Rocket Pharmaceuticals Announces Upcoming Presentations Rocket Pharmaceuticals, Inc. announced data presentations. Presentations at this year’s meeting include four oral presentations and three poster presentations related to Rocket’s four leading pipeline programs. [Press release from Rocket Pharmaceuticals, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting, Washington DC] Press Release Promethera Biosciences SA announced clinical data from the ongoing Phase IIa study of its lead product candidate HepaStemJ® in patients with Acute-on-Chronic Liver Failure (ACLF) or acute decompensation at high risk of developing ACLF. [Press release from Promethera Biosciences SA. discussing research presented at the The International Liver Congress™ (ILC) 2019, Vienna] Press Release | |
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INDUSTRY NEWSFibrocell Science, Inc. announced the completion of a collaboration agreement with Castle Creek Pharmaceuticals to develop and commercialize Fibrocell’s lead gene therapy candidate, FCX-007, for the treatment of recessive dystrophic epidermolysis bullosa, a rare, life-threatening genetic disorder diagnosed at infancy with no cure or treatment approved by the FDA. [Fibrocell Science, Inc.] Press Release Orgenesis Inc. announced that it has entered into a Collaboration and Licensing Agreement with ExcellaBio Ltd. for certain technologies developed by Sabina Glozman, Ph.D., including an exosome-like membrane nanostructure and related processes for the production of exosomes/extracellular vesicles. [Orgenesis Inc.] Press Release Paragon Bioservices announced that it will be exploring the formation of a wide-ranging partnership in gene therapy manufacturing with Sarepta Therapeutics, Inc. [Paragon Bioservices] Press Release Mallinckrodt plc announced that Stratatech has completed patient enrollment in its ongoing pivotal Phase III clinical trial of StrataGraft®, an investigational regenerative skin tissue. [Mallinckrodt plc] Press Release MaaT Pharma announced that the independent Data and Safety Monitoring Board (DSMB) recommended the continuation, without amendment, of the ongoing Phase II HERACLES study. [MaaT Pharma] Press Release Hemostemix Inc. announced that it has reached an enrollment milestone in its continuing Phase II Clinical Trial for critical limb ischemia. [Hemostemix Inc. (GlobeNewswire, Inc.)] Press Release Catalent to Acquire Gene Therapy Leader Paragon Bioservices, Inc. for $1.2 Billion Catalent, Inc. and Paragon Bioservices, Inc. announced they have entered into a definitive agreement under which Catalent will acquire Paragon for $1.2 billion. [Catalent, Inc.] Press Release Adverum Biotechnologies Provides Clinical Program Update on ADVM-022 Gene Therapy for Wet AMD Adverum Biotechnologies, Inc. provided a program update on ADVM-022, a novel, single-administration gene therapy being evaluated in the OPTIC Phase I clinical trial in patients with wet age-related macular degeneration (wet AMD). [Adverum Biotechnologies, Inc.] Press Release Celularity, Inc. announced the company has entered into a long-term lease agreement to develop a 145,000 square-foot advanced cell manufacturing and research facility in Florham Park, New Jersey. [Celularity, Inc. (Business Wire, Inc.)] Press Release The SanBio Group announced that SB623, a regenerative cell therapy that the Group is developing globally for the treatment of chronic motor deficit resulting from traumatic brain injury, has received the Sakigake Designation for innovative medical products from the MHLW of Japan. [The SanBio Group (Business Wire, Inc.)] Press Release | |
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POLICY NEWSIndonesian Mega-Science Agency in the Balance as Election Begins Indonesians will vote in a national election that pits two old rivals against each other. The stakes are high for science. President Joko Widodo says that if he is re-elected, he will overhaul how much of the country’s research is organized and funded. The main opposition candidate has been silent on science so far. [Nature News] Editorial African Research Projects Are Failing Because Funding Agencies Can’t Match Donor Money Although African countries appreciate research grants from donor countries, they often chafe at the condition that they bring in their own money in order to be eligible. Some research projects fall by the wayside because African granting agencies simply have no way to provide their share of the money, sometimes called counterfunding, the heads of 15 national science councils in Africa said at a meeting on 4 and 5 April. [ScienceInsider] Editorial
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EVENTSNEW FEBS: From Molecules to Living Systems Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW DPhil Scholarship – Retinal Gene Therapy (University of Oxford) Research Technologist – Immunology (STEMCELL Technologies Inc.) Gene Editing Specialist – Rare Genetic Disorders (Novo Nordisk) Staff Scientist – Development of Mechanism-Based Tumor Therapies (Frankfurt Cancer Institute) Cell Biologist – Regenerative Medicine (Hepatx Corporation) Stem Cell Podcast Co-Host (Stem Cell Podcast) Associate Scientist – Immuno-Oncology (HMH 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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