Cell Therapy News 18.26 July 31, 2017 | |
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TOP STORYLong-Term Microdystrophin Gene Therapy Is Effective in a Canine Model of Duchenne Muscular Dystrophy Investigators showed that locoregional and systemic delivery of a rAAV2/8 vector expressing a canine microdystrophin was effective in restoring dystrophin expression and stabilizing clinical symptoms in studies performed on a total of 12 treated golden retriever muscular dystrophy dogs. [Nat Commun] Full Article | Press Release | |
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PUBLICATIONS(Ranked by impact factor of the journal)PUMA Gene Delivery to Synoviocytes Reduces Inflammation and Degeneration of Arthritic Joints Intra-articular injection in an adjuvant-induced rat model of rheumatoid arthritis induces apoptosis of fibroblast-like synoviocytes, leading to a significant decrease in joint inflammation, joint damage, and bone loss with improvement in joint function and mobility. [Nat Commun] Full Article Benchmarked against leading commercial small interfering RNA (siRNA) nanocarrier in vivo jetPEI, siRNA conjugated to a diacyl lipid moiety achieved 19-fold greater tumor accumulation and 46-fold increase in per-tumor-cell uptake in a mouse orthotopic model of human triple-negative breast cancer. [Proc Natl Acad Sci USA] Abstract Gene-Based Therapy in a Mouse Model of Blue Cone Monochromacy Scientists showed that recombinant middle-wavelength (M)-opsin delivered by AAV5 vectors rescued M-cone function in Opn1mw−/− mice. They also showed that AAV delivered M-opsin localized in the dorsal cone outer segments, and co-localized with short-wavelength sensitive-opsin in the ventral retina. [Sci Rep] Full Article Researchers examined whether increasing the reactivity of donor T cells by gene modification could enhance the therapeutic efficacy of donor lymphocyte infusion in a murine model of acute lymphocytic leukemia. [Exp Mol Med] Full Article Scientists demonstrated that combination of non-viral gene delivery and CRISPR/Cas9-mediated knockin via homology-directed repair can replace the use of viral vectors for the generation of genetically modified therapeutic cells. [Mol Ther Nucleic Acids] Full Article As part of a Phase II trial, scientists used a consolidation therapy regimen which combined a busulfan/melphalan conditioning schema, autologous hematopoietic cell transplant (AHCT), and experimental immunotherapy with hu14.18K322A, GM-CSF, and IL-2, with or without the adoptive transfer of haploidentical natural killer cells (NKs). They report on 30 patients that went undergone AHCT with this experimental immunotherapy regimen, 21 of which received haploidentical NKs. [Biol Blood Marrow Transplant] Abstract In vivo toxicology studies were conducted to evaluate potential toxicity of Lenti/βAS3-FB LV in the context of a murine bone marrow transplant. [Cytotherapy] Abstract The authors administered AAV2/9-hNDUFS4 by intravenous (IV) and/or intracerebroventricular (ICV) routes to either newborn or young Ndufs4−/− mice. They found that IV administration alone was only able to correct the complex I deficiency in peripheral organs, whereas ICV administration partially corrected the deficiency in the brain. [Gene Ther] Full Article | |
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REVIEWSStem Cell Therapies for Reversing Vision Loss Researchers discuss current bioengineering approaches and biomaterial usage in combination with stem cell therapy for macular degeneration disease treatment. [Trends Biotechnol] Abstract Cholangiocytes: Cell Transplantation The authors discuss the advantages and pitfalls of the different cell sources tested so far in clinical trials and the rationale underlying the potential benefits of transplantation of human biliary tree stem cells. [Biochim Biophys Acta] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSOncolytics Biotech® to Present REOLYSIN® Safety Data in Combination with Chemotherapy Oncolytics Biotech® Inc. announced that two abstracts describing both pooled safety and tolerability data and the mechanism of REOLYSIN® have been selected for poster presentation. [Press release from Oncolytics Biotech® Inc. discussing research presented at the European Society for Medical Oncology (ESMO) 2017 Congress, Madrid] Press Release | |
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INDUSTRY NEWSNeon Therapeutics and Apexigen Announce Immuno-Oncology Clinical Trial Collaboration Neon Therapeutics, Inc. and Apexigen, Inc. announced an immuno-oncology clinical trial collaboration. Under this agreement, the companies plan to evaluate Neon Therapeutics’ proprietary personalized neoantigen vaccine, NEO-PV-01, in combination with and Apexigen’s CD40 agonist antibody, APX005M, for the treatment of patients with metastatic melanoma. [Apexigen, Inc.] Press Release Gamida Cell Announces $3.5 Million Grant from the Israeli Government Gamida Cell announced that it has been awarded a grant of $3.5 million from the Israel Innovation Authority of the Israeli Ministry of Economy and Industry. [Gamida Cell] Press Release CIRM Approves $5.8 Million Grant for CAR-T Therapy that Targets Cancer Stem Cells The Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine (CIRM) unanimously approved a $5.8 million award to University of California San Diego School of Medicine researchers to develop a new immunotherapy in which patients’ cells would be equipped with a special receptor that recognizes and targets cancer stem cells, whose survival abilities often render standard therapies ineffective or short-term. [UC San Diego Health] Press Release Quark Pharmaceuticals, Inc. announced successful completion of a randomized, double-blinded, placebo-controlled multicenter Phase II trial of QPI-1002, a synthetic chemically modified siRNA acting to temporarily reduce p53 expression, for the prophylaxis of AKI following cardiac surgery. [Quark Pharmaceuticals, Inc.] Press Release Transgene announced that the first patient has been treated in a Phase I/II clinical trial evaluating the combination of Pexa-Vec with Opdivo® as a first-line treatment of advanced hepatocellular carcinoma, which accounts for approximately 75% of liver cancers. [Transgene] Press Release Kite Pharma, Inc. announced that it has submitted a Marketing Authorization Application to the European Medicines Agency for axicabtagene ciloleucel as a treatment for patients with relapsed/refractory diffuse large B-cell lymphoma, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma who are ineligible for autologous stem cell transplant. [Kite Pharma, Inc.] Press Release Capricor Announces Results of FDA Meeting on Intravenous CAP-1002 for Duchenne Muscular Dystrophy Capricor Therapeutics, Inc. announced that it has received official minutes of the meeting held recently between the FDA and Capricor to discuss the development of intravenous CAP-1002 for the treatment of Duchenne muscular dystrophy. [Capricor Therapeutics, Inc.] Press Release Researchers have opened a Phase I clinical trial, PLAT-04, for children and young adults with relapsed or refractory CD22-positive acute lymphoblastic leukemia. They will examine the safety, feasibility and efficacy of administering cancer-fighting CAR T cells that have been reprogrammed to target the CD22 protein expressed by some leukemia cells. [Seattle Children’s Hospital] Press Release New SU2C-Lustgarten Foundation Team Aims to Apply CAR T-Cell Therapy to Pancreatic Cancer Stand Up To Cancer is supporting a new translational research team to explore how a type of immunotherapy that has been very successful in blood cancers can be applied to pancreatic cancer, the American Association for Cancer Research, SU2C’s Scientific Partner, announced. [American Association for Cancer Research] Press Release | |
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POLICY NEWSChina Cracks Down after Investigation Finds Massive Peer-Review Fraud A massive peer-review fraud has triggered a tough response from the Chinese government. Officials last week announced that more than 400 researchers listed as authors on some 100 now-retracted papers will face disciplinary action because their misconduct has seriously damaged China’s scientific reputation. [ScienceInsider] Editorial At Harvard, Extraordinary Court Battle between Ph.D. Student and Prominent Researcher Grinds On The conflict between Gustavo German, a Harvard doctoral student in biomedicine, and Lee Rubin, a prominent stem cell researcher, has continued to evolve. Amid continued legal jousting, the situation has remained difficult for all of the parties involved in this tumultuous episode. [ScienceInsider] Editorial First Human Embryos Edited in U.S. The first known attempt at creating genetically modified human embryos in the United States has been carried out by a team of researchers in Portland, Oregon. The effort, led by Shoukhrat Mitalipov of Oregon Health and Science University, involved changing the DNA of a large number of one-cell embryos with the gene-editing technique CRISPR, according to people familiar with the scientific results. [MIT Technology Review] Editorial Budding UK Innovation Agency Gains Cash — and Clout UK scientists fearful for their research funds ahead of Brexit were cheered when the government announced it would plough an extra £4.7 billion (US$6.1 billion) into research and development by 2020–21. But the biggest winner from the largely industry-focused cash may be a government innovation agency that is rapidly gaining clout. [Nature News] Editorial
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REGULATORYFDADeveloping a Framework for Regulatory Use of Real-World Evidence; Public Workshop (FR Doc. No:2017-16021) Notice Institutional Review Board Waiver or Alteration of Informed Consent for Clinical Investigations Involving No More than Minimal Risk to Human Subjects; Guidance for Sponsors, Investigators, and Institutional Review Boards; Availability (FR Doc. No:2017-15539) Notice
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EVENTSNEW VII InterAmerican Oncology Conference Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Research Group Leaders (Centro de Investigación PrÃncipe Felipe) Postdoctoral Research Associate – Gene Therapy (King’s College London) Research Associate – Cell Engineering (Caribou Biosciences, Inc.) Scientist – Analytical Development (KBI Biopharma) Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic) Postdoctoral Fellow – Various Projects (University of Oklahoma) 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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