Cell Therapy News Volume 20.05 | Feb 11 2019

TOP STORY

Phase I Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

Givosiran is an investigational RNA interference therapeutic agent that inhibits hepatic aminolevulinic acid synthase 1 synthesis. The authors conducted a Phase I trial of givosiran in patients with acute intermittent porphyria. [N Engl J Med] Abstract | Press Release

(Ranked by impact factor of the journal)

An Injectable Bone Marrow-Like Scaffold Enhances T Cell Immunity after Hematopoietic Stem Cell Transplantation

Researchers report a biomaterial-based scaffold that mimicked features of T cell lymphopoiesis in the bone marrow. The bone marrow cryogel released bone morphogenetic protein-2 to recruit stromal cells and presented the Notch ligand delta-like ligand-4 to facilitate T cell lineage specification of mouse and human hematopoietic progenitor cells. [Nat Biotechnol] Abstract | Press Release

Patterned Human Microvascular Grafts Enable Rapid Vascularization and Increase Perfusion in Infarcted Rat Hearts

Scientists report engineered perfusable microvascular constructs, wherein human embryonic stem cell-derived endothelial cells were seeded both into patterned microchannels and the surrounding collagen matrix. When implanted on infarcted rat hearts, the perfusable microvessel grafts integrated with coronary vasculature to a greater degree than non-perfusable self-assembled constructs at five days post-implantation. [Nat Commun] Full Article | Press Release

The Mechanistic Study behind Suppression of GvHD while Retaining GvL Activities by Myeloid-Derived Suppressor Cells

Using multiple murine graft-versus-host disease (GvHD)/graft-versus-leukemia (GvL) models including both MHC-mismatched and miHA-mismatched, researchers demonstrated that treatment with CD115⁺ myeloid-derived suppressor cells efficiently suppressed GvHD but did not significantly impair GvL activity, leading to 80 and 67% protection in treated mice in GvHD and GvL models, respectively. [Leukemia] Abstract

Long-Term Engraftment of ESC-Derived B-1 Progenitor Cells Supports HSC-Independent Lymphopoiesis

Investigators demonstrated that embryonic stem cell (ESC)-derived B-progenitors differentiate into B-1 and marginal zone B cells, but not B-2 cells in immunodeficient mice after transplantation. ESC-derived B-1 cells were maintained in the recipients for more than six months, secreting natural IgM antibodies in vivo. [Stem Cell Reports] Full Article | Graphical Abstract | Editorial

Evaluating CAR‐T Cell Therapy in a Hypoxic 3D Tumor Model

A microdevice platform that recapitulated a 3D tumor section with a gradient of oxygen and integrates fluidic channels surrounding the tumor for CAR‐T cell delivery was engineered. [Adv Healthc Mater] Abstract

Therapeutic Potential of Hepatocyte-Like-Cells Converted from Stem Cells from Human Exfoliated Deciduous Teeth in Fulminant Wilson’s Disease

Stem cells from human exfoliated deciduous teeth (SHED) and SHED-converted hepatocyte-like-cells (Heps) were transplanted into Wilson’s disease model and Atp7b-mutated Long-Evans Cinnamon (LEC) rats received copper overloading to induce a lethal fulminant liver failure. Due to the superior copper tolerance via ATP7B, SHED-Hep transplantation gave more prolonged life-span of fulminant LEC rats than SHED transplantation. [Sci Rep] Full Article

Liver-Specific Gene Delivery Using Engineered Virus-Like Particles of Hepatitis E Virus

Scientists investigated whether hepatitis E virus-like particles (HEV-LPs) could deliver foreign genes specifically to the liver. HEV-LPs were obtained from N-terminal truncated second open reading frame expression in Huh7 cells that were transduced with recombinant baculoviruses and purified by continuous density gradient centrifugation. [Sci Rep] Full Article

Ultrasound-Responsive Nanobubble-Mediated Gene Transfection in the Cerebroventricular Region by Intracerebroventricular Administration in Mice

Researchers aimed to characterize a transfection system with respect to transfection efficiency, spatial distribution of transgene expression, and safety. Plasmid DNA was transfected into a mouse brain by intracerebroventricular injection of ultrasound-responsive nanobubbles, followed by ultrasound irradiation to the brain. [Eur J Pharm Biopharm] Abstract | Graphical Abstract

Adipose-Derived Stem Cell Therapy Inhibits the Deterioration of Cerebral Infarction by Altering Macrophage Kinetics

Human adipose-derived stem cell administration improved neurobehavioral deficits in MCAO-treated mice and suppressed brain atrophy at the chronic phase. [Brain Res] Abstract

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Mesenchymal Stem Cell 3D Encapsulation Technologies for Biomimetic Microenvironment in Tissue Regeneration

The authors introduce several details of mesenchymal stem cell encapsulation technologies such as micromolding, electrostatic droplet extrusion, microfluidics, and bioprinting and their application for tissue regeneration. [Stem Cell Res Ther] Full Article

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Neurogene Announces Presentation of Preclinical Data for Gene Therapies to Treat AGU and CMT4J, Two Rare and Devastating Neurodegenerative Disorders

Neurogene, Inc. announced that researchers presented preliminary, unpublished “proof of concept” data in mice evaluating the safety and efficacy of adeno-associated virus gene therapy for AGU and CMT4J, two rare neurological diseases. [Press release from Neurogene, Inc. (Business Wire, Inc.) discussing research presented at the 15^th Annual WORLDSymposium™ Conference, Orlando] Press Release

Catalyst Biosciences Announces Preclinical Proof-of-Concept Data of Gene Therapy Candidate CB 2679d-GT for the Treatment of Hemophilia B

Catalyst Biosciences, Inc. presented preclinical proof-of-concept data of CB 2679d-GT Factor IX gene therapy in hemophilia B mice. [Press release from Catalyst Biosciences, Inc. discussing research presented at 12^th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), Prague] Press Release

uniQure Announces Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B

uniQure N.V. announced updated clinical data in patients treated in the company’s ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B. [Press release from uniQure N.V. discussing research presented at 12^th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), Prague] Press Release

Adverum Biotechnologies Announces Presentation of Preclinical Long-Term Safety Data of ADVM-022

Adverum Biotechnologies, Inc. announced non-human primate long-term safety data of ADVM-022, an intravitreally delivered gene therapy currently in Phase I for the treatment of wet age-related macular degeneration. [Press release from Adverum Biotechnologies, Inc. discussing research presented at the 2019 Angiogenesis, Exudation, and Degeneration Conference, Miami] Press Release

Neurotrope Announces Cooperative Research and Development Agreement for Bryostatin-1 with the National Cancer Institute

Neurotrope Inc. announced that it has entered into a Cooperative Research and Development Agreement with the National Cancer Institute for the research and clinical development of Bryostatin-1. [Neurotrope Inc.] Press Release

Cellect Begins Collaboration with Washington University in Preparation for First U.S. Clinical Trial Using Cellect’s ApoGraft™ for Bone Marrow Transplantations

Cellect Biotechnology Ltd. announced a collaboration with Washington University aimed at determining the safety and tolerability in a U.S. Phase I/II study using ApoGraft™ for bone marrow transplantations. [Cellect Biotechnology Ltd. (PR Newswire Association LLC)] Press Release

Capricor Resumes Dosing of Enrolled Patients in HOPE-2 Clinical Trial for Duchenne Muscular Dystrophy

Capricor Therapeutics announced that it has resumed per protocol dosing of patients already enrolled in its HOPE-2 clinical trial of CAP-1002, the company’s novel cell therapy candidate to treat Duchenne muscular dystrophy. [Capricor Therapeutics] Press Release

New Cell-Based Technology by Cytonus Aims to Revolutionize the Fight against Cancer and Inflammatory Diseases

Cytonus Therapeutics Inc. announced that it has developed Cargocytes™ a first-of-its-kind, controllable, cell-based platform technology for delivery of biologics. [Cytonus Therapeutics Inc. (Business Wire, Inc.)] Press Release

Moderna Announces Dosing of the First Monoclonal Antibody Encoded by mRNA in a Clinical Trial

Moderna, Inc. announced the dosing of the first subject in a Phase I clinical trial evaluating the safety and tolerability of escalating doses of mRNA-1944 via intravenous infusion in healthy adults. [Moderna, Inc.] Press Release

AVROBIO, Inc. Announces Updated Clinical Data from Ongoing Phase I and Phase II Studies for AVR-RD-01 Gene Therapy for Fabry Disease

AVROBIO, Inc. announced clinical trial updates from the ongoing investigator-sponsored Phase I clinical study and the AVROBIO-sponsored Phase II clinical trial of AVR-RD-01. [AVROBIO, Inc.] Press Release

Actinium Provides Patent Portfolio Update for Its Iomab-ACT Next-Generation CAR-T Lymphodepletion Program

Actinium Pharmaceuticals, Inc. announced that it has further expanded the intellectual property portfolio for the technology underpinning its Iomab-ACT program. The Iomab-ACT program is being developed for lymphodepletion, which is also referred to as conditioning, prior to administration of CAR-T based therapies. [Actinium Pharmaceuticals, Inc.] Press Release

Fate Therapeutics Announces FDA Clearance of IND Application for World’s First Cell Therapy Derived from an Engineered Pluripotent Stem Cell

Fate Therapeutics, Inc. announced that the FDA has allowed its Investigational New Drug (IND) application for FT516, the company’s off-the-shelf natural killer cell product candidate derived from a clonal master induced pluripotent stem cell line engineered to express a novel CD16 Fc receptor. [Fate Therapeutics, Inc.] Press Release

Solid Biosciences Announces Preliminary SGT-001 Data and Intention to Dose Escalate in IGNITE DMD Clinical Trial for Duchenne Muscular Dystrophy

Solid Biosciences announced preliminary findings from IGNITE DMD, the company’s Phase I/II dose-ascending clinical trial evaluating the safety and efficacy of SGT-001 microdystrophin gene transfer for the treatment of Duchenne muscular dystrophy (DMD). [Solid Biosciences] Press Release

Precigen Announces Clearance of IND to Initiate Phase I Study for First-in-Class PRGN-3005 UltraCAR-T™ to Treat Advanced Stage Platinum Resistant Ovarian Cancer Patients

Precigen, Inc. announced that the FDA has cleared the Investigational New Drug (IND) application for Precigen’s PRGN-3005, a first-in-class investigational therapy using autologous chimeric antigen receptor T (CAR-T) cells to treat advanced-stage platinum-resistant ovarian cancer patients. [Precigen, Inc. (PR Newswire Association LLC.)] Press Release

The Parker Institute for Cancer Immunotherapy Expands Its Network to Include More Rising Stars in Cancer Immunotherapy

The Parker Institute for Cancer Immunotherapy recently welcomed seven talented early career researchers in cancer immunotherapy to its network as part of the Parker Scholars, Parker Bridge Scholars and Parker Fellows programs. These researchers will receive a total of up to $3.1 million in funding and the opportunity to train with top scientists in the field, to support them as they embark on their research to move the field forward. [The Parker Institute for Cancer Immunotherapy] Press Release

New Patent Win for University of California Upends CRISPR Legal Battle

The University of California has received good news on a patent for the invention of the genome editor known as CRISPR—and it likely moves a fierce legal war over who owns the valuable intellectual property for this powerful tool closer to a peace treaty. [ScienceInsider] Editorial

Europe’s Next €1-Billion Science Projects: Six Teams Make It to Final Round

The European Commission has selected six research projects — in areas from health and energy to artificial intelligence and cultural heritage — to compete to become one of its next billion-euro ‘flagship’ science initiatives, Nature has learned. [Nature News] Editorial

France Set to Get First National Strategy for Research

France is preparing to implement a national, multi-year research plan for the first time — a move warmly welcomed by the heads of the country’s major research agencies. [Nature News] Editorial

Ukraine’s Science Revolution Stumbles Five Years On

Ukraine’s science system is in a precarious state, despite promised improvements in the wake of a revolution five years ago that aligned the country with the European Union. [Nature News] Editorial

FDA

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting (FR Doc. No:2019-00769) Notice

NEW 2^nd Annual Meeting of the American Society for Gene & Cell Therapy (ASGCT)
April 29-May 2, 2019
Washington, DC, United States

Visit our events page to see a complete list of events in the community.

NEW Research Technologist – Immunology (STEMCELL Technologies Inc.)

NEW Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma)

Research Associate – Bioengineering (STEMCELL Technologies Inc.)

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

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

Cell Manufacturing Scientist (CSL Behring)

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

Postdoctoral Fellow – Inherited Retinal Degenerations (Ocular Genomics Institute)

Postdoctoral Position – Pluripotent Stem Cells and Rare Diseases (I-STEM)

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

Assistant Project Scientist – Hematology and Oncology (University of California, Davis)

Faculty Positions – Hematology and Oncology (University of California, Davis)

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