Cell Therapy News 20.11 April 1, 2019 | |
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TOP STORYAmong patients with advanced heart failure, intramyocardial injections of mesenchymal precursor cells, compared with injections of a cryoprotective medium as sham treatment, did not improve successful temporary weaning from left ventricular assist device support at six months. [JAMA] Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)Photothermal Therapy Promotes Tumor Infiltration and Antitumor Activity of CAR T Cells Investigators found that chondroitin sulfate proteoglycan‐4‐specific CAR T cells infused in Nod scid gamma mice engrafted with the human melanoma WM115 cell line had superior antitumor activity after photothermal ablation of the tumor. [Adv Mater] Abstract | Press Release The authors developed an investigational bioengineered human acellular vessel, which is currently being studied as a hemodialysis conduit in patients with end-stage renal disease. [Sci Transl Med] Abstract | Press Release | Editorial Gene Replacement Therapy in a Model of Charcot-Marie-Tooth 4C Neuropathy Scientists developed a gene replacement therapy for treating Charcot-Marie-Tooth disease type 4C to rescue the phenotype of the Sh3tc2−/− mouse model. They generated a lentiviral vector LV-Mpz.SH3TC2.myc to drive expression of the human SH3TC2 cDNA under the control of the Mpz promoter specifically in myelinating Schwann cells. [Brain] Full Article CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters Researchers describe a novel approach based on CRISPR/dCas9 transcriptional activation to achieve robust transgene expression from transgene cassettes containing tissue or cell type-specific promoters after infection with AAV vectors in cell-based systems. [Mol Ther Methods Clin Dev] Abstract | Full Article A moderately severe cervical clip compression/contusion injury was induced at C7‐T1 in adult female rats, followed by an intravenous tail vein infusion one‐hour post‐spinal cord injury of term‐birth human umbilical cord perivascular cells, first‐trimester human umbilical cord perivascular cells, adult bone marrow mesenchymal stem cells, or vehicle control. [Stem Cells Transl Med] Full Article Combination of Cabazitaxel and p53 Gene Therapy Abolishes Prostate Carcinoma Tumor Growth In a xenograft mouse model of in situ gene therapy, AdRGD-PGp53 treatment, when combined with cabazitaxel, drastically reduced tumor progression and increased survival rates to 100%. [Gene Ther] Abstract Safe and Neuroprotective Vectors for Long-Term Traumatic Brain Injury Gene Therapy The authors evaluated in the controlled cortical impact model of traumatic brain injury, the safety of integrative-deficient lentiviral vectors or the non-viral HNRK recombinant modular protein/DNA nanovector. [Gene Ther] Abstract Safety and Efficacy of Oncolytic HSV-1 G207 Inoculated into the Cerebellum of Mice Scientists demonstrated that cerebellar inoculation of G207 was systemically non-toxic in an immunocompetent, herpes simplex virus (HSV)-1 sensitive mouse strain. Mice had neither abnormal brain/organ pathology nor evidence of G207 replication by immunohistochemistry at days 7 and 30 after cerebellar G207 inoculation. [Cancer Gene Ther] Abstract Combination of Stem Cell and Gene Therapy Ameliorates Symptoms in Huntington’s Disease Mice Researchers demonstrated the efficacy of the combination of stem cell and gene therapy in a transgenic Huntington’s disease mouse model using rhesus monkey neural progenitor cells. [npj Regen Med] Full Article Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSCAR T Cell Toxicity – Current Management and Future Directions The authors offer a practical guide for the recognition and management of the most important toxicities related to the use of current commercial CAR T cells, and also highlight strategies to diminish these side effects in the future. [HemaSphere] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSOrchard Therapeutics presented the full registrational dataset from a 20 patient trial evaluating the efficacy and safety of OTL-200, an ex vivo autologous hematopoietic stem cell-based gene therapy for the treatment of metachromatic leukodystrophy (MLD). [Press release from Orchard Therapeutics discussing research presented at the 45th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Frankfurt] Press Release Two BrainStorm Scientific Abstracts Accepted for Presentation BrainStorm Cell Therapeutics Inc. announced that two scientific abstracts have been accepted for presentation. [Press release from BrainStorm Cell Therapeutics Inc. discussing research presented at the 71st American Academy of Neurology (AAN) Annual Meeting, Philadelphia] Press Release Dicerna™ Pharmaceuticals, Inc. announced the presentation of updated data from its ongoing PHYOX™1 Phase I clinical trial evaluating DCR-PHXC, the company’s lead GalXC™ product candidate. [Press release from Dicerna™ Pharmaceuticals, Inc. discussing research presented at the German Society of Pediatric Nephrology 50th Annual Meeting, Cologne] Press Release Sensei Biotherapeutics, Inc. announced new data from two programs targeting a novel tumor specific embryonic antigen, human aspartate β-hydroxylase (ASPH), including preclinical data on SNS-723, a first-in-class CAR-T cell therapy demonstrating effective killing of tumor cells by a series of ASPH-specific CAR-Ts as well as additional data from its Phase I study on the long-term effects of SNS-301, a first-in-class cancer immunotherapy demonstrating that all patients experienced dose-dependent and durable ASPH-specific immune responses. [Press release from Sensei Biotherapeutics, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release Celularity, Inc. announced results from two Phase I studies of PNK-007, an investigational allogeneic off-the-shelf natural killer (NK) cell therapy, in patients with acute myeloid leukemia and in patients with multiple myeloma. [Press release from Celularity, Inc. (Business Wire, Inc.) discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release F1 Oncology, Inc. will present four abstracts that support novel technologies to treat solid tumor malignancies. [Press release from F1 Oncology, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release Oncorus, Inc. unveiled preclinical data yesterday supporting the clinical advancement of its lead oncolytic virus candidate, ONCR-177, during an oral presentation. [Press release from Oncorus, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release IO Biotech Presents Data on an Immunomodulatory CCL22-Based Vaccine IO Biotech presented preclinical data on the therapeutic potential of an immunomodulatory CCL22-based vaccine. [Press release from IO Biotech (PR Newswire Association LLC) discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release Torque Presents New Preclinical Data for Its Deep-Primed Cellular Immunotherapy Programs Torque announced two preclinical data presentations for its cellular immunotherapy programs. [Press release from Torque discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release Fate Therapeutics, Inc. presented late-breaking preclinical data highlighting the company’s unmatched ability to mass produce uniformly engineered chimeric antigen receptor (CAR) T cells for off-the-shelf cancer immunotherapy. [Press release from Fate Therapeutics, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release Allogene Therapeutics, Inc. announced results from a preclinical study of its AlloCAR T program targeting CD70, a cancer target that is expressed on both hematologic and solid tumor cells. [Press release from Allogene Therapeutics, Inc. (GlobeNewswire, Inc.) discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release | |
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INDUSTRY NEWSIchor® Medical Systems, Inc. announced that it has entered into a collaboration and research license agreement with AstraZeneca for the development and clinical assessment of plasmid DNA constructs. [Ichor® Medical Systems, Inc. (Business Wire, Inc.)] Press Release TCR2Therapeutics Inc. announced it has selected the advanced therapy medicinal products ecosystem in the United Kingdom to establish a site for global manufacturing systems and capabilities. [TCR2Therapeutics Inc.] Press Release StrideBio, Inc announced the signing of a collaboration and license agreement with Takeda Pharmaceutical Company Limited to develop in vivo adeno-associated viral based therapies for Friedreich’s Ataxia and two additional undisclosed targets. [StrideBio, Inc] Press Release Phio Pharmaceuticals Corp. announced that it has entered into a research collaboration with Glycostem Therapeutics BV to explore the potential synergies of using Phio’s self-delivering RNAi technology in combination with Glycostem’s proprietary Natural Killer-cell generation technology to develop cellular immunotherapies for cancer treatment with enhanced efficacy and/or safety. [Phio Pharmaceuticals Corp.] Press Release Harvard Researchers’ Immune-Silent Stem Cell Technology Licensed to Sana Biotechnology Harvard’s Office of Technology Development has executed a license agreement with Sana Biotechnology, a recently launched company co-founded by Harvard faculty that is focused on creating and delivering engineered cells as medicines for patients. Harvard’s foundational technology consists of methods for producing hypoimmunogenic stem cells that can be differentiated into any cell type and then transplanted into a patient without triggering immune rejection. [Harvard Office of Technology Development] Press Release Orgenesis Inc. announced that it has entered into a Master Service and Joint Venture Agreement with TheraCell Advanced Biotechnology, for the clinical development and commercialization of cell and gene therapies. [Orgenesis Inc.] Press Release Oxford Biomedica plc noted an announcement by Novartis that Japan’s Ministry of Health, Labor and Welfare has approved Kymriah® for the treatment of two distinct indications – CD19-positive relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia and CD19-positive r/r diffuse large B-cell lymphoma. [Oxford Biomedica plc] Press Release Fibrocell Science, Inc. announced the completion of a Type B end-of-Phase II meeting with the FDA to discuss the design of a Phase III clinical trial for FCX-007, the company’s gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa, to support a Biologics License Application filing. [Fibrocell Science, Inc.] Press Release Herantis Pharma Plc announced the expansion of its Phase II AdeLE clinical trial in Sweden to include the Karolinska University Hospital in Stockholm, and the Uppsala University Hospital. [Herantis Pharma Plc] Press Release bluebird bio, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion recommending conditional marketing authorization for ZYNTEGLO™, a gene therapy for patients 12 years and older with TDT who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available. [bluebird bio, Inc.] Press Release Incysus Therapeutics, Inc. announced that the FDA has approved the Investigational New Drug (IND) application for the clinical study of a genetically modified γδ T cell in combination with chemotherapy for the treatment of patients with newly-diagnosed glioblastoma. [Incysus Therapeutics, Inc. (GlobeNewswire, Inc.)] Press Release NantKwest announced that the company has been issued a new patent that further expands the company’s foundational natural killer cell-therapeutics platform intellectual property estate. [NantKwest] Press Release | |
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POLICY NEWSFirst Gene Therapy to Treat Rare Blood Disease Nears European Approval The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use, the drug-reviewing arm of the European Medicines Agency. [STAT News] Editorial Canadian academic and industry stakeholders are concerned about the inclusion of “virus-like particles or sub-viral particles” in the definition of New Substances Notification Regulations for Organisms (NSNR(O)) which impacts clinical cell and gene therapy and commercialization. [Front Med] Article What Thailand’s Long-Awaited Election Could Mean for Science Thailand is bracing for the results of its first election since a military junta took charge in a 2014 coup. Scientists, like many other people in Thailand, want a more democratic country. But whether a pro-democracy party or a military-backed one ends up in charge, science is likely to prosper as a result of ongoing reforms put in place by the junta that are likely to continue under either government. [Nature News] Editorial National Academy of Sciences Will Vote on Ejecting Sexual Harassers The U.S. National Academy of Sciences in Washington, D.C., will ask its members this month to change the organization’s bylaws to allow proven sexual harassers and those guilty of other misconduct to be ejected from their ranks. That’s a first for the prestigious organization that advises the U.S. government on scientific issues: Its members, who are voted in by other members, have always been elected for life. [ScienceInsider] Editorial
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REGULATORYFDAAdvisory Committee; Cellular, Tissue and Gene Therapies Advisory Committee, Renewal (FR Doc. No:2019-05985) Notice
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EVENTSNEW 24th Congress of the European Hematology Association (EHA) Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Staff Scientist – Development of Mechanism-Based Tumor Therapies (Frankfurt Cancer Institute) Research Technologist – Immunology (STEMCELL Technologies Inc.) Research Associate – Bioengineering (STEMCELL Technologies Inc.) Postdoctoral Position – Immunotherapy (McGill University) 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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