Cell Therapy News 20.04 February 4, 2019 | |
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TOP STORYThe authors showed that peptides developed by modular design approaches could spontaneously form compact and monodisperse nanoparticles with poloxamines and nucleic acids via self-assembly. Both messenger RNA and plasmid DNA expression mediated by peptide-poloxamine nanoparticles were greatly boosted in vitro and in the lungs of cystic fibrosis mice with negligible toxicity. [Nat Nanotechnol] Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)By transplantation of lentiviral transduced hematopoietic cells in the Mpl-deficient mouse model scientists addressed whether known or predicted thrombopoietin target genes were able to rescue the Mpl-deficient phenotype of the mice. [Blood] Abstract | Press Release Next-Generation Muscle-Directed Gene Therapy by In Silico Vector Design Researchers identified potent muscle-specific transcriptional cis-regulatory modules (CRMs), containing clusters of transcription factor binding sites, using a genome-wide data-mining strategy. These novel muscle-specific CRMs resulted in a substantial increase in muscle-specific gene transcription when delivered using adeno-associated viral vectors in mice. [Nat Commun] Full Article Investigators used a previously established approach of islet transplant in the anterior chamber of the eye in allogeneic recipient mouse models and a baboon model of diabetes, which were treated transiently with anti-CD154/CD40L blocking antibody in the peri-transplant period. [Diabetologia] Abstract | Press Release Researchers evaluated the efficacy of allogeneic transplantation of adipose-derived multi-lineage progenitor cells in a micro-mini pig periodontal defect model. [Sci Rep] Full Article The authors sought to determine whether co-transplantation of endothelial colony-forming cells and placenta-derived mesenchymal stromal cells could achieve long-term engraftment and Factor VIII expression. [Stem Cell Res Ther] Full Article Measles Vector as a Multiple Genes Delivery Platform Facilitating iPSC Reprogramming Scientists present a highly efficient multi-transgene delivery system based on a vaccine strain of measles virus, a non-integrating RNA virus that has a long-standing safety record in humans. [Gene Ther] Abstract The authors evaluated the efficacy of the LDL receptor (LDLR)-TF chimeric protein in CHOLDLA7, a cell line lacking LDLR expression, restoring the ability to uptake LDL. Subsequently, they administered intravenously 1 × 10E13 vp/kg of this vector in LDLR-deficient mice and observed amelioration of lipid profile and reduction of aortic atherosclerosis. [Gene Ther] Abstract Investigators evaluated the effect of pretreating bone marrow-derived mesenchymal stem cells with melatonin on the prosurvival and renoprotective of transplanted cells into the irreversible model of unilateral ureteral obstruction. [J Mol Histol] Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSAdeno-Associated Virus Vector as a Platform for Gene Therapy Delivery Preclinical and clinical successes in adeno-associated virus (AAV)-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States. [Nat Rev Drug Discov] Abstract Exosomes — Beyond Stem Cells for Restorative Therapy in Stroke and Neurological Injury The authors discuss the current knowledge of exosomes and advances in our knowledge of their effects on endogenous neurovascular remodeling events. They also consider the opportunities for exosome-based approaches to therapeutic amplification of brain repair and improvement of recovery after stroke, traumatic brain injury and other diseases in which neurorestoration could be a viable treatment strategy. [Nat Rev Neurol] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSSpark Therapeutics Announces Presentation of Preclinical Data in Pompe Disease and CLN2 Disease Spark Therapeutics announced the presentation of preclinical data for SPK-3006 for Pompe disease and SPK-1001 for CLN2 disease, a form of Batten disease. [Press release from Spark Therapeutics discussing research to be presented at the 15th Annual WORLDSymposium™, Orlando] Press Release Abeona Therapeutics to Present New Supportive Data for Novel Gene Therapies Abeona Therapeutics Inc. announced that data from ongoing clinical trials for lysosomal storage diseases and novel AIM™ chimeric AAV-based vector gene therapy programs will be presented. [Press release from Abeona Therapeutics Inc. discussing research to be presented at the 15th Annual WORLDSymposium™, Orlando] Press Release | |
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INDUSTRY NEWSNeurocrine Biosciences, Inc. and Voyager Therapeutics, Inc. announced the formation of a strategic collaboration focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. [Neurocrine Biosciences, Inc.] Press Release The Janssen Pharmaceutical Companies of Johnson & Johnson announced a worldwide collaboration and license agreement with MeiraGTx Holdings plc to develop, manufacture and commercialize its clinical stage inherited retinal disease portfolio, including leading product candidates for achromatopsia caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa. [Janssen Global Services, LLC] Press Release GenSight Biologics announced results from the first scheduled readout, at week 48, of the RESCUE Phase III clinical trial evaluating the safety and efficacy of a single intravitreal injection of GS010 in 39 subjects whose visual loss due to 11778-ND4 LHON occurred up to six months prior to study treatment. [GenSight Biologics] Press Release uniQure N.V. announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. [uniQure N.V.] Press Release Audentes Therapeutics, Inc. provided an update following the receipt of minutes from its Regenerative Medicine and Advanced Therapy (RMAT)/Type B meeting with the FDA held in December 2018, regarding the company’s lead gene therapy candidate, AT132, for the treatment of X-linked myotubular myopathy (XLMTM). [Audentes Therapeutics, Inc.] Press Release REGENXBIO Inc. announced the FDA granted Rare Pediatric Disease Designation to RGX-181. RGX-181 is a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 gene. [REGENXBIO Inc.] Press Release Hemostemix Announces Submission of Orphan Drug Designation Application to the FDA Hemostemix Inc. announced that it has submitted an application with the FDA to receive Orphan Drug Designation status for its lead product ACP-01 for the treatment of critical limb ischemia. [Hemostemix Inc.] Press Release CARsgen Therapeutics Receives IND Clearance for GPC3-CAR-T Cells from the NMPA CARsgen Therapeutics announced that one of its leading drug candidates, humanized GPC3-CAR-T cell for the treatment of patients suffering from GPC3-positive solid tumors, has received Investigational New Drug (IND) clearance from the National Medical Products Administration (NMPA). [CARsgen Therapeutics (PR Newswire Association LLC.)] Press Release Hitachi Chemical Advanced Therapeutics Solutions, LLC announced an agreement by which apceth Biopharma GmbH will join Hitachi Chemical’s PCT global services platform. [Hitachi Chemical Advanced Therapeutics Solutions, LLC (Business Wire, Inc.)] Press Release First US Patient in Novel Stem Cell Trial for Stroke Disability Enrolled at UTHealth The first U.S. patient to participate in a global study of a stem cell therapy injected directly into the brain to treat stroke disability was enrolled in the clinical trial this week at The University of Texas Health Science Center at Houston (UTHealth). [University of Texas Health Science Center at Houston] Press Release | |
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POLICY NEWSAfter Shutdown, NSF Catching Up on Review Panels, Funding Requests When the US government was partially shut down for 35 days starting December 22, work at several government agencies ground to a halt, with a total of 800,000 federal employees furloughed. The National Science Foundation (NSF), one of the sidelined science agencies, is now addressing all that went undone during that time, NSF officials told reporters. [The Scientist] Editorial Rookies Lead the Way on House Science Panel A major perk of being the majority party in the U.S. Congress is getting to fill the leadership slots on every committee. For several new Democratic legislators, however, having their party regain control of the House of Representatives also creates an unprecedented opportunity to shape U.S. science policy. [ScienceInsider] Editorial Indian Government to Boost Stipend for Early-Career Scientists The Indian government announced that it will increase the stipend for PhD students who have qualified for a research fellowship by up to 25%. [Nature News] Editorial
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