Cell Therapy News Volume 20.24 | Jul 15 2019

TOP STORY

Enhanced CAR-T Cell Activity against Solid Tumors by Vaccine Boosting through the Chimeric Receptor

Scientists demonstrated an approach to enhancing CAR-T function in solid tumors by directly vaccine-boosting donor cells through their chimeric receptor in vivo. [Science] Abstract | Press Release

(Ranked by impact factor of the journal)

CD19 CAR T Cells following Autologous Transplantation in Poor Risk Relapsed and Refractory B Cell Non-Hodgkin Lymphoma

The authors investigated whether CD19-specific chimeric antigen receptor T cells administered following high-dose chemotherapy followed by autologous stem cell transplantation may enhance progression-free survival. [Blood] Abstract

Therapeutic Efficacy of Different Routes of Mesenchymal Stem Cell Administration in Corneal Injury

Researchers evaluated the efficacies of different modes of mesenchymal stem cell delivery to promote tissue repair and restore corneal transparency in a murine model of corneal injury. [Ocul Surf] Abstract

Effective Hematopoietic Stem Cell-Based Gene Therapy in a Murine Model of Hereditary Pulmonary Alveolar Proteinosis

Using Csf2rb^-/- mice, a model closely reflecting the human herPAP disease phenotype, the authors demonstrated robust pulmonary engraftment of an alveolar macrophage population following intravenous transplantation of lentivirally corrected hematopoietic stem and progenitor cells. [Haematologica] Abstract

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

Researchers developed a scAAV9 vector expressing the human CLN6 gene under the control of a chicken β-actin (CB) hybrid promoter. Intrathecal delivery of scAAV9.CB.hCLN6 into the cerebrospinal fluid of the lumbar spinal cord of four-year old non-human primates was safe, well tolerated, and led to efficient targeting throughout the brain and spinal cord. [Mol Ther] Abstract | Graphical Abstract | Full Article

Intranasal Mesenchymal Stem Cell Secretome Administration Markedly Inhibits Alcohol and Nicotine Self-Administration and Blocks Relapse-Intake: Mechanism and Translational Options

Investigators aimed to inhibit chronic ethanol and nicotine self-administration and relapse by the non-invasive intranasal administration of antioxidant and anti-inflammatory secretome generated by adipose tissue-derived activated mesenchymal stem cells. [Stem Cell Res Ther] Full Article

Transplantation of Mesenchymal Stem Cells Overexpressing FGF21 Facilitates Cognitive Recovery and Enhances Neurogenesis in a Mouse Model of Traumatic Brain Injury

Mesenchymal stem cells (MSCs) were genetically engineered to overexpress fibroblast growth factor 21 (FGF21) in order to improve their efficacy in traumatic brain injury (TBI). MSCs overexpressing FGF21 were transplanted to mouse brain by intracerebroventricular injection 24 hours after TBI was induced by controlled cortical impact. [J Neurotrauma] Abstract

Muscle Spindle Reinnervation Using Transplanted Embryonic Dorsal Root Ganglion Cells after Peripheral Nerve Transection in Rats

The authors demonstrated that transplantation of embryonic dorsal root ganglion cells induced regeneration of sensory nerve fiber and reinnervation of muscle spindles in skeletal muscle. [Cell Prolif] Full Article

Reconstruction of the Damaged Dorsal Root Entry Zone by Transplantation of Olfactory Ensheathing Cells

Investigators compared short- and long-term olfactory ensheathing cell (OEC) survival and their interaction with the surrounding dorsal root tissue. They reveal how transplanted OECs orient toward the spinal cord and allow newly formed axons to travel across into the dorsal horn of the spinal cord. [Cell Transplant] Full Article

Neural Stem Cell Transplantation Improves Locomotor Function in Spinal Cord Transection Rats Associated with Nerve Regeneration and IGF-1 R Expression

Scientists showed that neural stem cells (NSCs) could survive and differentiate into neurons in vitro and in vivo. Spinal cord transection-induced deficits were reduced by NSC transplantation, including increase in NeuN-positive cells and decrease in apoptotic cells. [Cell Transplant] Full Article

TCR α⁺β⁺/CD19⁺ Cell-Depleted Hematopoietic Stem Cell Transplantation for Pediatric Patients

Researchers report 20 pediatric patients who underwent TCR α⁺β⁺/CD19⁺ cell-depleted HSCT in four Australian centers. Conditioning regimen was dependent on HSCT indication, which included immunodeficiency, Fanconi anemia, and acute leukemia. [Pediatr Transplant] Abstract

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Gene Therapy of Hematological Disorders: Current Challenges

The author discusses tools used in gene therapy for hematological disorders, choices of target cells, and delivery vehicles with emphasis on current hurdles and attempts to solve them, and presents examples of successful clinical trials to give a glimpse of current progress. [Gene Ther] Abstract

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Be The Match BioTherapies^® and Tmunity Announce Collaboration to Support Clinical Trials of Next-Generation Immunotherapies

Be The Match BioTherapies^® and Tmunity Therapeutics, Inc. announced a partnership to accelerate patient access to life-saving cell therapies. [Be The Match BioTherapies^®] Press Release

ProtoKinetix Reaches Mid-Point of 3^rd Stage of Retinal Cell Replacement Therapy Testing at UBC

ProtoKinetix, Incorporated has reached the mid-point of 3^rd stage of testing in retinal cell replacement therapy at the University of British Columbia (UBC). [ProtoKinetix, Incorporated] Press Release

AVROBIO Announces 87% Substrate Reduction in First Kidney Biopsy and Additional Positive Data from Clinical Trials of AVR-RD-01 Investigational Gene Therapy in Fabry Disease

AVROBIO, Inc. announced the first kidney biopsy result and additional positive data from two ongoing clinical trials of its AVR‑RD‑01 investigational gene therapy in Fabry disease. [AVROBIO, Inc.] Press Release

Avalon GloboCare Provides Updates on Its Lead Clinical Programs in CAR-T Therapy and Exosome-Based Regenerative Therapeutics

Avalon GloboCare Corp. announced an update on its four clinical programs in cellular therapy, including AVA-001 and AVA-101, that leverage individualized CAR T-cell therapy for immuno-oncology, as well as AVA-201 and AVA-202 exploring novel development with stem cell derived exosomes. [Avalon GloboCare Corp.] Press Release

Caladrius Biosciences Receives Advanced Therapy Medicinal Product Classification for CLBS12, Its CD34⁺ Cell Therapy for Critical Limb Ischemia

Caladrius Biosciences, Inc. announced that the European Medicines Agency has granted Advanced Therapy Medicinal Product classification to the company’s CD34⁺ cell therapy product, CLBS12, for the treatment of critical limb ischemia. [Caladrius Biosciences, Inc.] Press Release

Dicerna Receives Breakthrough Therapy Designation for DCR-PHXC for Treatment of Primary Hyperoxaluria Type 1 (PH1)

Dicerna™Pharmaceuticals, Inc. announced that the FDA has granted a Breakthrough Therapy Designation to DCR-PHXC for the treatment of patients with PH1. [Dicerna™Pharmaceuticals, Inc.] Press Release

Gene Therapy: £2m Boost for a Core Facility at the UK DRI

Adeno-associated virus is one of the most actively investigated gene therapy vehicles. Now, under the leadership of Prof Chris Shaw, Associate Director of the UK Dementia Research Institute (UK DRI) at Kings, the institute will be at the forefront of realizing this potential. This program has been awarded the first in a series of Directors’ Strategic Initiatives from the UK DRI. [UK Dementia Research Institute] Press Release

Children’s Hospital Los Angeles Has Treated 14 Patients with Retinal Degeneration Using Gene Therapy

More than a year after becoming one of the first medical institutions nationally to complete a revolutionary gene replacement surgery to restore vision in patients with retinal degeneration, surgeons at the of The Vision Center at Children’s Hospital Los Angeles have successfully completed the procedure on an additional 13 patients. [Children’s Hospital Los Angeles] Press Release

Passage Bio Announces New Dedicated Gene Therapy Manufacturing Suite

Passage Bio announced that it has entered into a collaboration agreement with Paragon Gene Therapy for the development of a dedicated manufacturing suite at their facility near Baltimore, Maryland. [Passage Bio (GlobeNewswire, Inc.)] Press Release

A Brief Guide to the Current CRISPR Landscape

Hundreds of CRISPR patents have been granted around the world, and the number of applications continues to grow at a rapid pace. [The Scientist] Editorial

University of California Loses Access to New Journal Articles Published by Elsevier after Research Access Fight

Researchers in the University of California system lost subscription access to the major publisher Elsevier, the result of a closely watched fight between the two parties over how academic research should get read and paid for. [STAT News] Editorial

NIH

Prospective Grant of an Exclusive Patent License: Autologus Therapy Using Bicistronic Chimeric Antigen Receptors Targeting CD19 and CD20 (FR Doc. No:2019-14823) Notice

Prospective Grant of an Exclusive Patent License: Allogeneic Therapy Using Bicistronic Chimeric Antigen Receptors Targeting CD19 and CD20 (FR Doc. No:2019-14822) Notice

NEW Biotech Week Boston
September 9-12, 2019
Boston, Massachusetts, United States

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

Stem Cell Podcast Co-Host (Stem Cell Podcast)

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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