Cell Therapy News Volume 20.20 | Jun 10 2019

TOP STORY

Gene Therapy for Catecholaminergic Polymorphic Ventricular Tachycardia by Inhibition of Ca²⁺/Calmodulin-Dependent Kinase II

The authors developed AAV9-GFP-AIP, an adeno-associated viral vector in which a potent CaMKII inhibitory peptide was fused to GFP and expressed from a cardiomyocyte selective promoter. The vector was delivered systemically. Arrhythmia burden was evaluated using invasive electrophysiology testing in adult mice. [Circulation] Abstract

(Ranked by impact factor of the journal)

Mesenchymal Stem Cell Therapy Induces FLT3L and CD1c⁺ Dendritic Cells in Systemic Lupus Erythematosus Patients

Transplantation of allogeneic umbilical cord-derived mesenchymal stem cells significantly up-regulated peripheral blood CD1c⁺ dendritic cells and serum FLT3L. [Nat Commun] Full Article

Enhancement of Adeno-Associated Viral (AAV) Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues

Scientists treated mouse embryonic fibroblasts, primate retinal pigment epithelial cells and human retinal explants with hydroxychloroquine one hour prior to transduction with an AAV2 vector encoding GFP driven by a ubiquitous CAG promoter. [Mol Ther Methods Clin Dev] Full Article

Delayed Autologous Stem Cell Transplantation following Cardiac Transplantation Experience in Patients with Cardiac Amyloidosis

The authors retrospectively investigated the outcomes of patients with light‐chain amyloidosis with advanced cardiac involvement who were treated with a strategy of heart transplantation followed by delayed autologous stem cell transplantation at one‐year post‐transplant. [Am J Transplant] Abstract

Dose-Dependent Improvement of Cardiac Function in a Swine Model of Acute Myocardial Infarction after Intracoronary Administration of Allogeneic Heart-Derived Cells

Researchers performed a comprehensive preclinical feasibility and safety evaluation of porcine cardiac-derived progenitor cells in the infarcted porcine model, analyzing biodistribution and mid-term efficacy, as well as safety in healthy non-infarcted swine. [Stem Cell Res Ther] Full Article

Cardiac Regeneration by Statin‐Polymer Nanoparticle‐Loaded Adipose‐Derived Stem Cell Therapy in Myocardial Infarction

A small number of intravenously administered simvastatin‐conjugated nanoparticle‐loaded adipose‐derived stem cells improved cardiac function following myocardial infarction, inducing endogenous cardiac regeneration in the infarcted myocardium. [Stem Cells Transl Med] Full Article

Nanofiber-Expanded Human CD34⁺ Cells Heal Cutaneous Wounds in Streptozotocin-Induced Diabetic Mice

Scientists offer an aminated polyethersulfone nanofiber-expanded human umbilical cord blood-derived CD34⁺ cell effective therapy, tested in cutaneous wounds developed in streptozotocin-induced diabetic NOD/SCID mice. [Sci Rep] Full Article

HITM-SIR: Phase Ib Trial of Intraarterial Chimeric Antigen Receptor T-Cell Therapy and Selective Internal Radiation Therapy for CEA⁺ Liver Metastases

Investigators previously demonstrated the safety of CAR-T hepatic artery infusions (HAI). They subsequently conducted a Phase Ib HITM-SIR trial, in which six patients with CEA⁺ liver metastases received anti-CEA CAR-T HAIs and selective internal radiation therapy. [Cancer Gene Ther] Abstract

Endothelial Progenitor Cell Transplantation Attenuates Lipopolysaccharide-Induced Acute Lung Injury via Regulating miR-10a/b-5p

Endothelial progenitor cells promoted lipopolysaccharide-induced mouse pulmonary microvascular endothelial cells proliferation and exerted the therapeutic effect of acute lung injury via regulating the miR-10a/b-5p/ADAM15 axis. [Lipids Health Dis] Full Article

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Mesenchymal Stem Cells for Spinal Cord Injury: Current Options, Limitations, and Future of Cell Therapy

The therapeutic role of mesenchymal stem cells is discussed, together with their properties, application, limitations, and future perspectives. [Int J Mol Sci] Full Article

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Vertex Expands into New Disease Areas and Enhances Gene Editing Capabilities through Expanded Collaboration with CRISPR Therapeutics and Acquisition of Exonics Therapeutics

Vertex Pharmaceuticals Incorporated announced that the company is enhancing its gene editing capabilities to develop novel therapies for Duchenne muscular dystrophy and myotonic dystrophy type 1 by expanding its collaboration with CRISPR Therapeutics and acquiring Exonics Therapeutics. [Vertex Pharmaceuticals Incorporated] Press Release

Eloxx Pharmaceuticals’ Phase II Study of ELX-02 in Cystinosis Receives Authorization of Clinical Trial Application (CTA) by Health Canada and Funding from Genome Quebec and Genome Canada

Eloxx Pharmaceuticals, Inc. announced that it has received authorization of its CTA from Health Canada for a Phase II clinical trial of ELX-02 in cystinosis. [Eloxx Pharmaceuticals, Inc.] Press Release

Homology Medicines Initiates Phase I/II Study for HMI-102 Gene Therapy Candidate for Adults with PKU and Expects Initial Clinical Results by Year End 2019

Homology Medicines, Inc. announced that it has commenced enrollment of the Phase I/II pheNIX trial for HMI-102, a one-time gene therapy development candidate for the treatment of adults with phenylketonuria (PKU). [Homology Medicines, Inc.] Press Release

Adverum Biotechnologies Doses First Patient in Second Cohort of OPTIC Phase I Clinical Trial of ADVM-022 Gene Therapy for Wet AMD

Adverum Biotechnologies, Inc. announced that the first patient was dosed in the second cohort of the ongoing OPTIC Phase I clinical trial for ADVM-022 for the treatment of wet age-related macular degeneration (AMD). [Adverum Biotechnologies, Inc.] Press Release

Enlivex Therapeutics Provides Clinical Update on Positive Safety Profile of Its Off-the-Shelf Cell Therapy Product in First Three Severe Sepsis Patients

Enlivex Therapeutics Ltd. provided a clinical update on the positive safety profile of its off-the-shelf cell therapy, Allocetra™, in the first three severe sepsis patients, as part of its ongoing Phase Ib clinical trial. [Enlivex Therapeutics Ltd. (GlobeNewswire, Inc.)] Press Release

Allogene Therapeutics Announces FDA Clearance of the IND for ALLO-715, a BCMA Allogeneic CAR T (AlloCAR T™) Therapy

Allogene Therapeutics, Inc. announced that the FDA has cleared Allogene’s Investigational New Drug (IND) application for ALLO-715 in patients with relapsed/refractory multiple myeloma. [Allogene Therapeutics, Inc.] Press Release

Altimmune Announces Successful Pre-IND Meeting with FDA

Altimmune, Inc. announced that it successfully completed a pre-Investigational New Drug (IND) meeting with the FDA regarding its Phase II trial design and manufacturing plans for HepTcell. [Altimmune, Inc.] Press Release

Rubius Therapeutics Announces Issuance of Multiple US Patents for Its Red Cell Therapeutic Programs

Rubius Therapeutics, Inc. announced the issuance of three patents from the US Patent and Trademark Office related to its rare disease and oncology programs and platforms. [Rubius Therapeutics, Inc.] Press Release

Parent Project Muscular Dystrophy Awards $175,000 Grant to UCLA to Explore Optimization of CRISPR Technology in Duchenne Muscular Dystrophy

Parent Project Muscular Dystrophy announced that Drs. Melissa Spencer and April Pyle from the David Geffen School of Medicine at UCLA and the Center for Duchenne Muscular Dystrophy at University of California, Los Angeles (UCLA) is the recipient of a $175,000 grant to continue their work optimizing a CRISPR/Cas9 platform to treat people living with Duchenne. [Parent Project Muscular Dystrophy] Press Release

Kiadis Pharma Completes Acquisition of CytoSen Therapeutics, Inc.

Kiadis Pharma N.V. announced that it has closed the previously announced acquisition of CytoSen Therapeutics, Inc. [Kiadis Pharma N.V.] Press Release

Judge Says FDA Can Stop Clinic from Selling Stem Cell Treatments

The decision may facilitate the agency cracking down more effectively on the unproven interventions these companies sell. [The Scientist] Editorial

China Urged to Abandon Plan to Sell Unproven Cell Therapies

An international group of stem-cell researchers is urging China to cancel draft regulations that would permit some hospitals to sell therapies developed from patients’ own cells, without approval from the nation’s drug regulator. [Nature News] Editorial

The Trump Administration Will End Fetal Tissue Research by Government Scientists

Government scientists will no longer conduct research using fetal tissue, the Trump administration said, granting the wishes of anti-abortion groups and overruling the concerns of scientists. [STAT News] Editorial

Russian Biologist Plans More CRISPR-Edited Babies

A Russian scientist says he is planning to produce gene-edited babies, an act that would make him only the second person known to have done this. It would also fly in the face of the scientific consensus that such experiments should be banned until an international ethical framework has agreed on the circumstances and safety measures that would justify them. [Nature News] Editorial

FDA

Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability (FR Doc. No:2019-11978) Notice

Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes; Public Workshop (FR Doc. No:2019-12113) Notice

NEW 6^th Annual European Congress on Clinical and Translational Medicine
October 18-20, 2019
Vienna, Austria

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

NEW Senior Scientist – Antigen Presentation (Astellas Pharma)

Stem Cell Podcast Co-Host (Stem Cell Podcast)

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

Postdoctoral Scholarship – Gene Therapy for Osteopetrosis (Lund University)

Postdoctoral Fellow – Hematopoietic Stem Cells and Gene Therapy (UC San Diego)

Postdoctoral Position – Tregs & Cell Therapy (INSERM)

Postdoctoral Position – Biomedical Engineering (US Food and Drug Administration)

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

Director of Research – Division of Translational Medicine & Human Genetics (University of Pennsylvania)

Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma)

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)

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

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