Cell Therapy News 19.02 January 22, 2018 | |
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TOP STORYResearchers report a global AAV9-based gene therapy protocol to deliver therapeutic galactosylceramidase (GALC), a lysosomal enzyme that is deficient in Krabbe’s disease. [Mol Ther] Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)Scientists showed that a single systemic injection of wild type/native human glucose-6-phosphatase (G6PC) mRNA results in significant improvements in fasting blood glucose levels for up to seven days post-dose. These changes were associated with significant reductions in liver mass, hepatic G6P, glycogen, and triglycerides. [Mol Ther] Abstract Investigators tested whether a synthetic extracellular matrix mimic, a hydrogel containing heparin, could ameliorate mesenchymal stromal cell (MSC) engraftment and binding/release of soluble paracrine factors, thus improving MSC therapy efficacy. [Acta Biomater] Abstract Using an in vitro model of axonal growth, the dorsal root ganglia explants, the authors demonstrated that olfactory ensheathing cells induce neurite outgrowth mainly through cell-cell interactions, while adipose tissue-derived stem cells’ effects are strongly mediated by the release of paracrine factors. [Stem Cells] Abstract A Humanized Mouse Model of Liver Fibrosis following Expansion of Transplanted Hepatic Stellate Cells To investigate whether hepatic stellate cells (HSCs) could engraft and repopulate the liver, scientists transplanted GFP-transduced immortalized human HSCs (hHSCs) into immunodeficient NOD/SCID mice. Biodistribution analysis with radiolabeled hHSCs showed that after intrasplenic injection, the majority of transplanted cells rapidly translocated to the liver. [Lab Invest] Abstract Mesenchymal stem cells were manufactured, cryopreserved, transported to our facility, thawed, and infused into nine recipients with moderate bronchiolitis obliterans syndrome who were refractory to standard therapy and not candidates for retransplant. [Stem Cells Transl Med] Abstract In Vivo Ovarian Cancer Gene Therapy Using CRISPR-Cas9 System Investigators use a folate receptor-targeted liposome to deliver a CRISPR plasmid DNA co-expressing Cas9 and single guide RNA targeting the DNA methyltransferase 1 gene of ovarian cancer. [Hum Gene Ther] Abstract Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies The authors demonstrated high-performance liquid chromatography-based measurements of globin expression after differentiation of the commonly applied HUDEP-2 cell line, in erythroid progeny of CD34+ cells for the analysis of CRISPR/Cas9-mediated disruption of the globin regulator BCL11A and of transgenic mice holding the human β-globin locus. [Hum Gene Ther] Abstract Scientists report on the first ever in vivo use of self-assembling peptide amphiphile carrying a tenascin-C signal for the re-direction of endogenous neuroblasts in the rodent brain. [J Tissue Eng Regen Med] Abstract | |
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REVIEWSCell Therapy for Spinal Cord Injury with Olfactory Ensheathing Glia Cells (OECs) Researchers highlight the properties of OECs that make them suitable to achieve neuroplasticity/neuroregeneration in spinal cord injury. OECs can interact with the glial scar, stimulate angiogenesis, axon outgrowth and remyelination, improving functional outcomes following lesion. [Glia] Abstract Combining Cell and Gene Therapy to Advance Cardiac Regeneration Allogeneic cardiac stem cell therapy, through its paracrine stimulation of the endogenous resident reparative/regenerative process, produces functional meaningful myocardial regeneration in pre-clinical porcine myocardial infarction models and is currently being tested in the first-in-man human trial. [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 NEWSRXi Pharmaceuticals to Present at Immuno-Oncology Frontiers World Conference RXi Pharmaceuticals Corporation announced that it will give an oral presentation highlighting the company’s proprietary therapeutic platform at the Immuno-Oncology Frontiers World conference. [RXi Pharmaceuticals] Press Release | |
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INDUSTRY NEWSCGT Catapult and CombiGene announced that they will be collaborating on a project to develop manufacturing processes for CombiGenes drug candidate CG01, a novel gene therapy for treatment of epilepsy. The collaboration aims to develop a complete and final manufacturing process which will allow CombiGene to progress to commercial GMP production and thereafter clinical trials. [CGT Catapult] Press Release The Cell and Gene Therapy Catapult (CGT Catapult) announced a partnership with Freeline Therapeutics to further develop the company’s proprietary manufacturing platform at the CGT Catapult’s large scale GMP manufacturing centre in Stevenage, Hertfordshire, UK. [CGT Catapult] Press Release Astellas Pharma Inc. and Vical Incorporated announced that ASP0113, an investigational DNA vaccine being developed for cytomegalovirus-seropositive hematopoietic stem cell transplant recipients, did not meet its primary or secondary endpoints in the Phase III HELIOS clinical trial. [Astellas Pharma Inc.] Press Release CytRx Corporation highlighted that aldoxorubicin licensee NantCell, Inc. has dosed the first patient in the Phase Ib portion of a Phase Ib/II clinical trial for patients with metastatic pancreatic cancer. [CytRx Corporation] Press Release First Duchenne Muscular Dystrophy Patient Dosed in Systemic Microdystrophin Gene Therapy Parent Project Muscular Dystrophy announced that the first Duchenne patient has been dosed with microdystrophin gene therapy by Dr. Jerry Mendell, Dr. Louise Rodino-Klapac, and their team at Nationwide Children’s Hospital. [Parent Project Muscular Dystrophy (PR Newswire Association LLC.)] Press Release Benitec Biopharma Limited announced that the FDA has granted Orphan Drug Designation to BB-301 for the treatment of oculopharyngeal muscular dystrophy. [Benitec Biopharma Limited (PR Newswire Association LLC.)] Press Release CBMG Accelerates Cell Therapy Manufacturing with GE Healthcare’s New Start-to-Finish Solution Cellular Biomedicine Group Inc. announced its plan to configure part of its facility in Shanghai with GE Healthcare’s FlexFactory™ platform, which is expected to be designed to speed up manufacturing timelines for its cell therapy clinical trials and commercial launch. [Cellular Biomedicine Group Inc.] Press Release Cytori Therapeutics, Inc. announced completion of patient enrollment in SCLERADEC II, a randomized, double-blind, placebo-controlled, parallel group, multi-center clinical trial intended to study the safety and efficacy of Habeo™ Cell Therapy in up to 40 subjects with impaired hand function due to scleroderma. [Cytori Therapeutics, Inc.] Press Release Celgene Corporation and Juno Therapeutics, Inc. announced the signing of a definitive merger agreement in which Celgene has agreed to acquire Juno. [Celgene Corporation] Press Release AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy AveXis, Inc. provided an overview of the expanded clinical development program for the company’s initial gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy. [AveXis, Inc.] Press Release Medeor Therapeutics Awarded $18.8 Million from the California Institute for Regenerative Medicine Medeor Therapeutics, Inc. announced that the California Institute for Regenerative Medicine has approved an award to the company for $18.8 million under its Clinical Trial Funding Initiative. [Medeor Therapeutics, Inc. (PR Newswire Association LLC.)] Press Release TxCell Obtains Government Funding to Advance its Lead CAR-Treg Program TxCell SA announced it has obtained an interest-free loan from Bpifrance for the preclinical development of its most advanced CAR-Treg program. [TxCell SA] Press Release U.S. Doctors Plan to Treat Cancer Patients Using CRISPR The first human test in the U.S. involving the gene-editing tool CRISPR could begin at any time and will employ the DNA cutting technique in a bid to battle deadly cancers. Doctors at the University of Pennsylvania say they will use CRISPR to modify human immune cells so that they become expert cancer killers, according to plans posted to a directory of ongoing clinical trials. [MIT Technology Review] Press Release | |
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POLICY NEWSBroad Institute Takes a Hit in European CRISPR Patent Struggle A decision from the European Patent Office (EPO) has put the Broad Institute in Cambridge, Massachusetts, on shaky ground with its intellectual property claims to the gene-editing tool CRISPR. EPO yesterday revoked a patent granted to the Broad for fundamental aspects of the technology, one of several of its patents facing opposition in Europe. [ScienceInsider] Editorial U.S. Scientists Breathe a Sigh of Relief as Government Shutdown to End It’s over—at least for the moment. The U.S. Senate voted 81–18 to advance legislation funding government activities, essentially ending a partial government shutdown that had moved into its third day. The House of Representatives is expected to follow suit, sending the bill to President Donald Trump for his signature. [ScienceInsider] Editorial Online Tool Calculates Reproducibility Scores of PubMed Papers A new online tool measures the reproducibility of published scientific papers by analyzing data about articles that cite them. The software comes at a time when scientific societies and journals are alarmed by evidence that findings in many published articles are not reproducible and are struggling to find reliable methods to evaluate whether they are. [ScienceInsider] Editorial
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EVENTSNEW The 2nd Annual European Neoantigen Summit NEW Stem Cell Community Day: Bioprocessing Technologies in Stem Cell Research Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Postdoctoral Fellowships – Cancer Research (University of Miami) NEW Postdoctoral Scientist – Systems Oncology (Cancer Research UK Manchester Institute) Tenure-Track Group Leader Position – Pancreatic Cancer Biology (University of Copenhagen) Postdoctoral Research Fellows – Bioengineering and Nanotechnology (University of Queensland) Research Associate – Immunology and Gene Therapy (Fred Hutchinson Cancer Research Center) Postdoctoral Associate – Gene Therapy (University of Massachusetts) Postgraduate Positions – Translational Medicine (Qingdao University) 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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