Cell Therapy News Volume 20.25 | Jul 22 2019

TOP STORY

CAR-T Cells Secreting BiTEs Circumvent Antigen Escape without Detectable Toxicity

CART.bispecific T-cell engager (BiTE) cells eliminated heterogenous tumors in mouse models of glioblastoma. BiTE-EGFR was locally effective but was not detected systemically after intracranial delivery of CART.BiTE cells. [Nat Biotechnol] Abstract | Editorial

(Ranked by impact factor of the journal)

Tisagenlecleucel CAR-T Cell Therapy in Secondary CNS Lymphoma

Investigators report on an institutional experience with eight secondary central nervous system (CNS) lymphoma patients treated with commercial tisagenlecleucel. No patient experienced >grade 1 neurotoxicity and no patient required tocilizumab or steroids for CAR-T mediated toxicities. [Blood] Abstract

Pptc7 Is an Essential Phosphatase for Promoting Mammalian Mitochondrial Metabolism and Biogenesis

Scientists deleted the poorly characterized matrix phosphatase Pptc7 from mice using CRISPR-Cas9 technology. Strikingly, Pptc7^−/− mice exhibited hypoketotic hypoglycemia, elevated acylcarnitines and serum lactate, and died soon after birth. [Nat Commun] Full Article

Treatment with Humanized Selective CD19CAR-T Cells Shows Efficacy in Highly Treated B-ALL Patients Who Have Relapsed after Receiving Murine-Based CD19CAR-T Therapies

Humanized selective CAR possessed around six-fold higher affinity to CD19 vs. murine CAR. Incubation with selective domain-specific monoclonal antibodies selectively expanded CAR-transduced T cells, and led to a higher proportion of central memory T cells in the final products. [Clin Cancer Res] Abstract

Immune-Orthogonal Orthologues of AAV Capsids and of Cas9 Circumvent the Immune Response to the Administration of Gene Therapy

Scientists showed that the sequential use of immune-orthogonal orthologues of CRISPR-associated protein 9 (Cas9) and adeno-associated viruses (AAVs) evaded adaptive immune responses and enabled effective gene editing using repeated dosing. [Nat Biomed Eng] Abstract

Efficiency of Human Olfactory Ensheathing Cell Transplantation into Spinal Cysts to Improve Mobility of the Hind Limbs

Investigators demonstrated that human olfactory ensheathing cell transplantation was effective in experimental spinal cysts. [Stem Cells Dev] Abstract

Integration of Neurons Derived from Transplanted BDNF-Overexpressing Neural Stem Cells into Circuit of the Irradiated Hippocampus

Researchers investigated the differentiation and integration of brain-derived neurotrophic factor overexpressing neural stem cells. They observed that these transplanted cells migrated to the subgranular zone of irradiated rats four weeks after transplantation. [Radiat Res] Abstract

Pancreatic Ductal Cells May Have a Negative Effect on Human Islet Transplantation

Islets and islets + ductal pancreatosphere preparations were transplanted to streptozotocin-treated immunodeficient mice and glycemia, graft morphometry, and gene expression were determined. [PLoS One] Full Article

Efficacy and Safety of Autologous Stem Cell Transplantation after Induction Therapy with Lenalidomide, Bortezomib and Dexamethasone

Researchers conducted a retrospective single‐arm study to assess efficacy and safety of Lenalidomide, Bortezomib and Dexamethasone (RVD) combination in induction therapy before high‐dose chemotherapy with Melphalan followed by autologous stem cell transplantation, and RVD consolidation followed by Lenalidomide maintenance, from February 2011 to May 2016. [Eur J Haematol] Abstract

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CRISPR Craze to Transform Cardiac Biology

The authors review current applications and future potential for the use of CRISPR to study cardiac biology and disease. [Trends Mol Med] Full Article

Viral Vector: Potential Therapeutic for Glioblastoma Multiforme

Glioblastoma multiforme is a highly malignant primary brain tumour found in adults and is highlighted as the most devastating among all the other grades of glioma. Well-established standard treatment methods, such as chemotherapy, radiation and surgery, have resulted in modest improvement in the survival of patients. [Cancer Gene Ther] Abstract

Discussion on the Indication of Allogeneic Stem Cell Transplantation for Advanced Cutaneous T Cell Lymphomas

The authors discuss current evidence about allogeneic stem cell transplantation (allo-SCT) for cutaneous T cell lymphoma patients, timing, and new therapeutic options before allo-SCT, taking into account some considerations that may impact clinical outcome. [Int J Hematol] Abstract

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PharmaCyte Biotech and UTS Creating Advanced Version of Melligen Cells to Treat Diabetes

PharmaCyte Biotech, Inc. announced it has entered into a new research agreement with the University of Technology Sydney (UTS) in Australia to create a new version of Melligen cells for the treatment of diabetes with the potential to express higher levels of insulin. [PharmaCyte Biotech, Inc.] Press Release

Avalon GloboCare and GE Healthcare Announce Strategic Partnership to Accelerate Standardized Automation and Bio-Production for Cellular Medicines

Avalon GloboCare Corp. announced that they have established a strategic partnership with GE Healthcare. The partnership will accelerate Avalon’s standardization, automation and bio-production for clinical-grade CAR-T cells and other immune-effector cells for cellular immunotherapy, as well as exosomes/extracellular vesicles based regenerative therapeutics. [Avalon GloboCare Corp.] Press Release

Humanigen Secures Exclusive Worldwide License for the Prevention of GvHD through GM-CSF Neutralization from the University of Zurich

Humanigen, Inc. announced that it has secured an exclusive worldwide license agreement from the University of Zurich for technology used to prevent graft-versus-host disease (GvHD) through granulocyte macrophage-colony stimulating factor (GM-CSF) neutralization. [Humanigen, Inc.] Press Release

Adaptimmune Starts the SURPASS Clinical Trial with Its First Next-Generation SPEAR T-Cells Targeting MAGE‑A4 to Enhance Antitumor Responses

Adaptimmune Therapeutics plc has initiated SURPASS, its first clinical trial with a next-generation SPEAR T-cell targeting MAGE-A4. [Adaptimmune Therapeutics plc] Press Release

Apellis Pharmaceuticals Will Commence APL-9 Program to Control the Complement System in Host Responses to AAV Vector Administration for Gene Therapies

Apellis Pharmaceuticals Inc. announced its plans to develop APL-9 for the prevention of complement immune system activation coincident with adeno-associated virus (AAV) vector administration for gene therapies. [Apellis Pharmaceuticals Inc.] Press Release

LogicBio Therapeutics Receives Rare Pediatric Disease Designation for LB-001 for the Treatment of Methylmalonic Acidemia

LogicBio Therapeutics, Inc. announced the FDA has granted rare pediatric disease designation to LB-001, a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase gene for the treatment of methylmalonic acidemia. [LogicBio Therapeutics, Inc.] Press Release

Kite Announces Plans to Bolster Industry-Leading Cell Therapy Manufacturing Capabilities with New Viral Vector Facility

Kite announced plans for a new 67,000-square-foot facility in Oceanside, California, dedicated to the development and manufacturing of viral vectors, a critical starting material in the production of cell therapies. [Kite (Business Wire, Inc.)] Press Release

Fight for Coveted CRISPR Patents Gets Knottier, as MilliporeSigma Makes New Claims

After nearly seven years of failing to win fundamental patents on the genome-editing technology CRISPR, a unit of one of the world’s largest life sciences companies has thrown a Hail Mary: Recently, MilliporeSigma petitioned the US Patent and Trademark office to open an interference proceeding between CRISPR-Cas9 patents that it applied for way back in 2012 and patents that the University of California has applied for or been awarded. [STAT News] Editorial

The Plan to Mine the World’s Research Papers

Carl Malamud is on a crusade to liberate information locked up behind paywalls — and his campaigns have scored many victories. Now, the 60-year-old American technologist is turning his sights on a new objective: freeing paywalled scientific literature. And he thinks he has a legal way to do it. [Nature News] Editorial

NEW Cell Symposia: Hallmarks of Cancer
November 17-19, 2019
Seattle, Washington, United States

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NEW Postdoctoral Scientist – HSV Vectors as a Therapy for Nervous System Tumors (Nationwide Children’s Hospital)

Senior Research Associate – Cell & Gene Therapy (King’s College London)

Postdoctoral Position – Translational Gene Therapy (Fred Hutchinson Cancer Research Center)

Senior Scientist – Immuno-Oncology of Hematologic Malignancies (University of Kansas School of Medicine)

Research Scientist – Cellular Electrophysiology (Duke University)

Postdoctoral Scientist – Computational Biologist (Cedars-Sinai Medical Center)

Senior Scientist – Antigen Presentation (Astellas Pharma)

Postdoctoral Research Fellowships – Kidney Disease (Vanderbilt University Medical Center)

Postdoctoral Scholarship – Gene Therapy for Osteopetrosis (Lund University)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center)

Tenure Track Faculty Position – Cancer Biology & Therapeutics (Geisel School of Medicine at Dartmouth)

Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University)

Assistant/Associate or Professor – Cancer Immunotherapy/Tumor Immunology (University of California, Irvine)

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