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Pulmonary Cell News| Cell Therapy News 20.06 February 25, 2019 | |
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TOP STORYEndothelial cells, smooth muscle cells, and cardiomyocytes derived from hypoimmunogenic mouse or human induced pluripotent stem cells reliably evaded immune rejection in fully MHC-mismatched allogeneic recipients and survived long-term without the use of immunosuppression. [Nat Biotechnol] Abstract | Press Release | |
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PUBLICATIONS(Ranked by impact factor of the journal)Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy Researchers showed that genome editing and dystrophin protein restoration was sustained in the mdx mouse model of Duchenne muscular dystrophy for one year after a single intravenous administration of an adeno-associated virus that encodes CRISPR (AAV-CRISPR). [Nat Med] Abstract | Press Release Single-Dose CRISPR-Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome Scientists showed that reduction of lamin A/progerin by a single-dose systemic administration of adeno-associated virus-delivered CRISPR-Cas9 components suppressed Hutchinson-Gilford progeria syndrome in a mouse model. [Nat Med] Abstract | Press Release Development of a CRISPR/Cas9-Based Therapy for Hutchinson-Gilford Progeria Syndrome The authors explored the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in Hutchinson-Gilford progeria syndrome cells and mice by introducing frameshift mutations in the LMNA gene. [Nat Med] Abstract Lung Regeneration by Multipotent Stem Cells Residing at the Bronchioalveolar-Duct Junction Investigators generated a genetic lineage-tracing system that uses dual recombinases to specifically track bronchioalveolar stem cells (BASCs) in vivo. Fate-mapping and clonal analysis showed that BASCs became activated and responded distinctly to different lung injuries, and differentiated into multiple cell lineages including club cells, ciliated cells, and alveolar type 1 and type 2 cells for lung regeneration. [Nat Genet] Abstract Nonviral Ultrasound-Mediated Gene Delivery in Small and Large Animal Models This protocol describes how to prepare, conduct and optimize ultrasound-mediated gene delivery in both a murine and a porcine animal model. The protocol includes the preparation of a microbubble-DNA mix and in vivo sonoporation under ultrasound imaging. [Nat Protoc] Abstract Dual AAV-Mediated Gene Therapy Restores Hearing in a DFNB9 Mouse Model Researchers focused on the otoferlin gene underlying DFNB9, one of the most frequent genetic forms of congenital deafness. They adopted a dual adeno-associated virus (AAV) approach using two different recombinant vectors, one containing the 5′ and the other the 3′ portions of otoferlin cDNA, which exceeded the packaging capacity of the AAV when combined. [Proc Natl Acad Sci USA] Full Article | Press Release Scientists demonstrated that adoptive transfer of mesenchymal stem cells could prevent fetal loss in a lipopolysaccharide-induced abortion model and immune response-mediated spontaneous abortion model. [Cell Mol Immunol] Abstract Adipocytes: A Novel Target for IL-15/IL-15Rα Cancer Gene Therapy Investigators used a recombinant adeno-associated viral system to deliver an IL-15/IL-15Rα complex to the abdominal fat by intraperitoneal injection. Adipose IL-15/IL-15Rα complex gene transfer led to the expansion of natural killer cells in the adipose tissue and spleen in normal mice without notable side effects. [Mol Ther] Abstract | Graphical Abstract The authors developed an ex vivo regional gene therapy strategy using BMP-2-transduced allogeneic umbilical cord blood-MSCs to promote bone repair. [Hum Gene Ther] Abstract Scientists report a combination therapy in which conventional surgical treatment for osteoarthritis of the knee was followed by autologous chondrocyte sheet transplantation for cartilage repair. [npj Regen Med] Full Article Young Bone Marrow Transplantation Preserves Learning and Memory in Old Mice Investigators report that transplantation of young bone marrow to rejuvenate the hematopoietic system preserved cognitive function in old recipient mice, despite irradiation-induced suppression of neurogenesis, and without reducing β2-microglobulin. [Commun Biol] Full Article | Press Release Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSDelivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery The authors discuss challenges for clinical translation of mRNA-based therapeutics with an emphasis on recent advances in biomaterials and delivery strategies, and present an overview of the applications of mRNA-based delivery for protein therapy, gene editing and vaccination. [Mol Ther] 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 Presents Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD Orchard Therapeutics presented additional clinical proof-of-concept data evaluating OTL-102, an ex vivo, autologous, hematopoietic stem cell based gene therapy for the treatment of X-linked chronic granulomatous disease (X-CGD). [Press release from Orchard Therapeutics plc. discussing research presented at the 2019 Transplantation and Cellular Therapy Meetings of ASBMT and CIBMTR, Houston] Press Release Orchard Therapeutics will present two-year follow-up data in 20 patients from the registrational trial evaluating OTL-101, an autologous, ex vivo, hematopoietic stem cell gene therapy for the treatment of severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). [Press release from Orchard Therapeutics plc. discussing research presented at the 2019 Transplantation and Cellular Therapy Meetings of ASBMT and CIBMTR, Houston] Press Release Magenta Therapeutics Presents Clinical and Preclinical Data on MGTA-456 Cell Therapy Magenta Therapeutics announced that the company presented Phase II clinical data and preclinical research on its MGTA-456 program. [Press release from Magenta Therapeutics discussing research presented at the 2019 Transplantation and Cellular Therapy Meetings of ASBMT and CIBMTR, Houston] Press Release Gamida Cell Ltd. announced that new data from its NAM-NK and NiCord® programs was presented. [Press release from Gamida Cell Ltd.. discussing research presented at the 2019 Transplantation and Cellular Therapy Meetings of ASBMT and CIBMTR, Houston] Press Release | |
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INDUSTRY NEWSSpark Therapeutics Enters into Definitive Merger Agreement with Roche Spark Therapeutics announced that it has entered into a definitive merger agreement for Roche to fully acquire Spark Therapeutics at a price of $114.50 per share in an all-cash transaction. [Spark Therapeutics, Inc.] Press Release AbbVie and Voyager Therapeutics, Inc. announced an exclusive, global strategic collaboration and option agreement to develop and commercialize vectorized antibodies directed at pathological species of alpha-synuclein for the potential treatment of Parkinson’s disease and other diseases (synucleinopathies) characterized by the abnormal accumulation of misfolded alpha-synuclein protein. [AbbVie Inc.] Press Release CRISPR Therapeutics and StrideBio Expand Exclusive Development and Option Agreement CRISPR Therapeutics and StrideBio, Inc. announced that a strategic collaboration, previously initiated in April 2017 to generate engineered AAV capsids with improved properties for in vivo gene editing programs, has now been expanded to include additional undisclosed applications. [CRISPR Therapeutics] Press Release Mustang Bio, Inc. and Nationwide Children’s Hospital announced that they have partnered and entered into an exclusive worldwide license agreement to develop an oncolytic virus for the treatment of glioblastoma multiforme. [Mustang Bio, Inc.] Press Release Pluristem Enters into Collaboration with NASA to Study PLX Therapeutic Benefits in Space Missions Pluristem Therapeutics Inc. announced a collaboration between the company and NASA’s Ames Research Center to evaluate the potential of Pluristem’s PLX cell therapies in preventing and treating medical conditions caused during space missions. [Pluristem Therapeutics Inc.] Press Release Torque Announces Clinical Trial Collaboration with Merck Torque announced that it has entered into a clinical trial collaboration agreement with Merck. [Torque Therapeutics] Press Release Morphocell Technologies Inc. has negotiated a worldwide exclusive license agreement with Univalor for the commercialization of the stem cell-derived liver tissue technology developed by Dr. Massimiliano Paganelli at the Centre Hospitalier Universitaire Sainte-Justine. [Morphocell Technologies Inc.] Press Release Final Patient Dosed in Mesoblast Phase III Trial of Revascor Cell Therapy for Advanced Heart Failure Mesoblast Limited announced that the last patient has now been dosed in the Phase III events-driven trial of its allogeneic cell therapy product candidate Revascor for advanced chronic heart failure. [Mesoblast Limited] Press Release Lysogene and Sarepta Therapeutics, Inc. announced that the first patient has been dosed in AAVance, a global Phase II-III clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA). [Sarepta Therapeutics, Inc.] Press Release CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated reviewed recent progress in the clinical development programs for CTX001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe hemoglobinopathies. [CRISPR Therapeutics] Press Release Neurotech Pharmaceuticals, Inc. announced that the FDA has granted Fast Track designation for the company’s development candidate, NT-501 or Renexus® for the treatment of macular telangiectasia type 2. [Neurotech Pharmaceuticals, Inc.] Press Release Ocugen, Inc. announced the FDA granted orphan drug designation for OCU0400, Ocugen’s novel gene therapy, for the treatment of NR2E3 mutation-associated retinal degenerative disease. [Ocugen, Inc.] Press Release Sarepta Therapeutics, Inc. announced the FDA, Division of Neurology, has accepted its New Drug Application seeking accelerated approval for golodirsen and provided a regulatory action date of August 19, 2019. [Sarepta Therapeutics, Inc.] Press Release BrainStorm Announces First Contracted U.S. Clinical Site for Phase II Progressive MS Study BrainStorm Cell Therapeutics Inc. announced Cleveland Clinic as the first U.S. clinical site contracted for a planned Phase II open-label, multicenter study of repeated intrathecal administration of autologous MSC-NTF cells in participants with progressive multiple sclerosis (MS). [BrainStorm Cell Therapeutics Inc.] Press Release | |
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POLICY NEWS‘Reprogrammed’ Stem Cells to Treat Spinal-Cord Injuries for the First Time Scientists in Japan now have permission to inject ‘reprogrammed’ stem cells into people with spinal-cord injuries. An upcoming trial will mark the first time that induced pluripotent stem cells have been used to treat spinal-cord injuries, after a committee at Japan’s health ministry approved the study on 18 February. [Nature News] Editorial CRISPR Babies Trial May Have Been Government Funded Before news broke of a trial to genetically alter human embryos using CRISPR, the leader of the project, He Jiankui, claimed in various documents that the experiment was backed by government funding, STAT reports. The papers contradict both the assertion of an investigation by the government of Guangdong Province that He raised funds on his own and He’s own statement in a late-November talk that he had bankrolled the trial from his own savings and startup funds from his university. [STAT News] Editorial Open-Access Pioneer Randy Schekman on Plan S and Disrupting Scientific Publishing Nobel laureate Randy Schekman shook up the publishing industry when he launched the open-access journal eLife in 2012. Armed with millions in funding from three of the world’s largest private biomedical charities — the Wellcome Trust, the Max Planck Society and the Howard Hughes Medical Institute — Schekman designed the journal to compete with publishing powerhouses such as Nature, Science and Cell. [Nature News] Editorial
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REGULATORYFDAEnhancing the Incorporation of Patient Perspectives on Clinical Trials; Public Workshop; Request for Comments (FR Doc. No:2019-01826) Notice NIHProspective Grant of an Exclusive Patent License: “Treatment of Acute Lymphoblastic Leukemia, T-Cell Lymphoma, and Non-Small Cell Lung Cancer Using the 4A10 Antibody and Fragments Thereof” (FR Doc. No:2019-02442) Notice
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EVENTSNEW ISCT 2019 Annual Scientific Meeting Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESResearch Technologist – Immunology (STEMCELL Technologies Inc.) Research Associate – Bioengineering (STEMCELL Technologies Inc.) Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma) Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center) Cell Manufacturing Scientist (CSL Behring) Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University) Postdoctoral Fellow – Inherited Retinal Degenerations (Ocular Genomics Institute) Postdoctoral Fellow – Genome and Metabolic Engineering (Columbia University Medical Center) Postdoctoral Research Associate – Gene Therapy (University of Massachusetts) Research Fellow – Stem Cell Biology (Brigham & Women’s Hospital – Harvard Medical School) Assistant Project Scientist – Hematology and Oncology (University of California, Davis) 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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