Cell Therapy News Volume 20.18 | May 27 2019

TOP STORY

Intein-Mediated Protein Trans-Splicing Expands Adeno-Associated Virus Transfer Capacity in the Retina

Researchers showed that delivery of multiple adeno-associated viral vectors each encoding one of the fragments of target proteins flanked by short split inteins resulted in protein trans-splicing and full-length protein reconstitution in the retina of mice and pigs and in human retinal organoids. [Sci Transl Med] Abstract

(Ranked by impact factor of the journal)

Phagocytosis-Shielded Lentiviral Vectors Improve Liver Gene Therapy in Nonhuman Primates

Exploiting intravital microscopy and lentiviral vector (LV) surface engineering, scientists report a major role of the human phagocytosis inhibitor CD47, incorporated into LV cell membrane, in protecting LVs from uptake by professional phagocytes and innate immune sensing, thus favoring biodistribution to hepatocytes after systemic administration. [Sci Transl Med] Full Article

CTL-Mediated Immunotherapy Can Suppress SHIV Rebound in ART-Free Macaques

The authors evaluated the use of adoptively transferred HIV vaccine-induced subtype C envelope-specific cytotoxic T lymphocytes in a macaque subtype B simian-human immunodeficiency virus (SHIV) model to determine whether plasma viremia can be controlled after antiretroviral therapy interruption. [Nat Commun] Full Article

CD22 CAR T-Cell Therapy in Refractory or Relapsed B Acute Lymphoblastic Leukemia

Investigators conducted a CD22 CAR T-cell therapy in 34 relapsed or refractory B-ALL pediatric and adult patients who failed from previous CD19 CAR T-cell therapy. [Leukemia] Abstract

Multipotent Fetal-Derived Cdx2 Cells from Placenta Regenerate the Heart

Using a lineage-tracing strategy, scientists specifically labeled fetal-derived caudal-type homeobox-2 (Cdx2) cells with enhanced green fluorescent protein (eGFP). Cdx2-eGFP cells from end-gestation placenta were assayed for cardiac differentiation in vitro and in vivo using a mouse model of myocardial infarction. [Proc Natl Acad Sci USA] Full Article | Press Release

BCMA-Targeted CAR T-Cell Therapy plus Radiation Therapy for the Treatment of Refractory Myeloma Reveals Potential Synergy

Researchers present a case of a patient with multiply-relapsed, refractory myeloma whose clinical course showed evidence of a synergistic abscopal-like response to CAR T-cell therapy and localized radiation therapy (XRT). Shortly after receiving B-cell maturation antigen (BCMA)-targeted CAR T-cell therapy, the patient required urgent high-dose steroids and XRT for spinal cord compression. [Cancer Immunol Res] Abstract

Alpha-1 Antitrypsin-Expressing Mesenchymal Stromal Cells Confer a Long-Term Survival Benefit in a Mouse Model of Lethal GvHD

To assess the in vivo efficacy of apceth-201, it was tested in two different mouse models of acute graft-versus-host disease (GvHD). Control animals in a humanized model succumbed quickly to disease, whereas median survival was doubled in apceth-201-treated animals. [Mol Ther] Abstract | Graphical Abstract

Life-Long AAV-Mediated CRISPR Genome Editing in Dystrophic Heart Improves Cardiomyopathy without Causing Serious Lesions in mdx Mice

The authors found that AAVrh.74-mediated life-long CRISPR genome editing in mdx mice restored dystrophin expression and improved cardiac function without inducing serious adverse effects. [Mol Ther] Abstract

TNF-α/IL-1β-Licensed Mesenchymal Stromal Cells Promote Corneal Allograft Survival via Myeloid Cell-Mediated Induction of Foxp3⁺ Regulatory T Cells in the Lung

In vivo, when administered post-transplantation, nonlicensed syngeneic mesenchymal stromal cells (MSCs) improved graft survival from 0 to 50% and MSCs licensed with TNF-α/IL-1β, in an NO-dependent manner, improved survival to 70%. [FASEB J] Abstract

Review of the Long-Term Effects of Autologous Bone-Marrow Mononuclear Cell Implantation on Clinical Outcomes in Patients with Critical Limb Ischemia

Scientists investigated the outcomes of intramuscular injection of autologous bone-marrow mononuclear cells in a long-term follow-up period in atherosclerotic peripheral arterial disease patients who have no optional therapy. [Sci Rep] Full Article

Amido-Bridged Nucleic Acid (AmNA)-Modified Antisense Oligonucleotides Targeting α-Synuclein as a Novel Therapy for Parkinson’s Disease

AmNA-modified antisense oligonucleotide (ASO) efficiently downregulated α-synuclein (SNCA) at both the mRNA and protein level in vitro and in vivo. Administration of AmNA-ASO ameliorated neurological defects in Parkinson’s disease model mice expressing human wild type SNCA. [Sci Rep] Full Article

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Circulatory Support and Stem Cell Therapy in the Management of Advanced Heart Failure: A Concise Review of Available Evidence

The authors describe a rationale for concurrent bone marrow mononuclear cell and left ventricular assist device therapy in selected heart failure patients. This combination therapy has demonstrated improved myocardial perfusion and cardiac function in patients with advanced ischemic cardiomyopathy. [Regen Med] Abstract

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

bluebird bio to Present New Data from Clinical Studies of LentiGlobin™ Gene Therapy for Transfusion-Dependent β-thalassemia (TDT) and LentiGlobin Gene Therapy for Sickle Cell Disease (SCD)

bluebird bio, Inc. announced that new data from its investigational gene therapy programs for TDT and SCD will be presented. [Press release from bluebird bio, Inc. discussing research to be presented at the 24^th European Hematology Association (EHA) Congress, Amsterdam] Press Release

Acute Myocardial Infarction Patients Treated Early with Cell Therapy Delivered with BioCardia’s Helix Endocardial Delivery System Improved in Ejection Fraction and NYHA Class

BioCardia^®, Inc. announced positive results from a study of the company’s Helix™ Biotherapeutic Delivery System for cell therapy used to treat patients early following acute myocardial infarction to prevent long term heart failure. [Press release from BioCardia^®, Inc. discussing research presented at EuroPCR, Paris] Press Release

AveXis Receives FDA Approval for Zolgensma^®, the First and Only Gene Therapy for Pediatric Patients with Spinal Muscular Atrophy (SMA)

AveXis announced the FDA has approved Zolgensma^® for the treatment of pediatric patients less than two years of age with SMA with bi-allelic mutations in the survival motor neuron 1 gene. [Novartis AG] Press Release | Editorial

Adaptimmune and Alpine Immune Sciences Announce Collaboration and License Agreement to Develop Next-Generation SPEAR T-Cell Products

Adaptimmune Therapeutics plc and Alpine Immune Sciences, Inc. announced a collaboration and license agreement to develop next-generation SPEAR T-cell products which incorporate Alpine’s secreted and transmembrane immunomodulatory protein technology. [Adaptimmune Therapeutics plc] Press Release

Santhera Starts Collaboration in Gene Therapy Research for Congenital Muscular Dystrophy with the Biozentrum, University of Basel, Co-Financed by Innosuisse

Santhera Pharmaceuticals announced its collaboration with the Biozentrum of the University of Basel to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy. [Santhera Pharmaceuticals] Press Release

Avalon GloboCare Partners with MIT to Develop Innovative AI-Enhanced Protein Design Technology in Cellular Therapy

Avalon GloboCare Corp. announced it has entered into a sponsored research and licensing agreement with Massachusetts Institute of Technology (MIT) to generate novel therapeutic targets for cellular therapy using artificial intelligence (AI) enhanced protein design “QTY Code” technology. [Avalon GloboCare Corp.] Press Release

Sensorion Announces the Signature of a Partnership Framework Agreement with Institut Pasteur on Gene Therapy Programs Targeting Hearing Loss

Sensorion announced the signature of a research partnership framework agreement with Institut Pasteur granting to Sensorion an option to obtain exclusive licenses to develop and market drug candidates in gene therapy coming from collaborative projects, for the restoration, treatment and prevention of hearing disorders. [Sensorion] Press Release

Be The Match BioTherapies^® Announces Collaboration with Poseida Therapeutics to Support Development of Life-Saving Cellular Therapies

Be The Match BioTherapies^® announced a collaboration with Poseida Therapeutics to provide end-to-end services to ensure timely collection, transport and delivery of high-quality cellular starting material to meet the specifications and needs of Poseida throughout the development of their autologous CAR-T programs. [Be The Match BioTherapies^®] Press Release

Be The Match BioTherapies^® Announces Strategic Collaboration with Kiadis Pharma to Accelerate Patient Access to Haploidentical Stem Cell Therapies

Be The Match BioTherapies^® and Kiadis Pharma N.V. announced a strategic collaboration to support clinical evaluation of an innovative cell therapy product with potential to make haploidentical hematopoietic stem cell transplantations safer and more effective for patients. [Be The Match BioTherapies^®] Press Release

MeiraGTx Announces Positive Six-Month Data from Phase I/II Trial of Investigational Gene Therapy AAV-RPE65 for RPE65-Deficiency

MeiraGTx Holdings plc announced positive data from a Phase I/II dose escalation trial of AAV-RPE65, the company’s investigational gene therapy for the treatment of RPE65-deficiency, a condition that causes blindness. [MeiraGTx Holdings plc] Press Release

Abeona Therapeutics Treats First Patient in Second Cohort of Phase I/II Clinical Trial for ABO-101 in Sanfilippo Syndrome Type B (MPS IIIB)

Abeona Therapeutics Inc. announced that the company has completed dosing cohort 1 and treated the first patient in cohort 2 in an ongoing Phase I/II clinical trial evaluating ABO-101, its novel one-time gene therapy for MPS IIIB. [Abeona Therapeutics Inc.] Press Release

Adverum Biotechnologies Provides Update on OPTIC Phase I Trial for ADVM-022 in Wet AMD

Adverum Biotechnologies, Inc. announced that the FDA has lifted the clinical hold on ADVM-022 for the second cohort in the company’s OPTIC Phase I trial for wet age-related macular degeneration (wet AMD), allowing dose escalation to 2 x 10¹² vg/eye. [Adverum Biotechnologies, Inc.] Press Release

Homology Medicines Announces FDA Fast Track Designation for HMI-102 Gene Therapy Development Candidate for Adults with PKU

Homology Medicines, Inc. announced that the FDA has granted Fast Track designation for HMI-102, Homology’s one-time gene therapy development candidate for the treatment of adults with phenylketonuria (PKU). [Homology Medicines, Inc.] Press Release

Abeona Therapeutics Announces FDA Clearance of Investigational New Drug Application for ABO-202 Gene Therapy in CLN1 Disease

Abeona Therapeutics Inc. announced that the company is cleared to begin a Phase I/II clinical trial evaluating its novel, one-time gene therapy ABO-202 for the treatment of CLN1 disease, following acceptance of its Investigational New Drug application by the FDA. [Abeona Therapeutics Inc.] Press Release

Axovant Announces Dosing of First Patient in Clinical Program for AXO-AAV-GM1, a Novel Gene Therapy for GM1 Gangliosidosis

Axovant Gene Therapies Ltd. announced dosing of the first patient in a clinical study of AXO-AAV-GM1, an investigational gene therapy for the treatment of GM1 gangliosidosis. [Axovant Gene Therapies Ltd.] Press Release

Athersys Announces Fast Track Designation from FDA for MultiStem^® Program for Acute Respiratory Distress Syndrome

Athersys, Inc. announced that its clinical program evaluating MultiStem^® cell therapy for the treatment of acute respiratory distress syndrome has received Fast Track designation from the FDA. [Athersys, Inc.] Press Release

uniQure Strengthens Intellectual Property Portfolio with Granted Patent Claims Covering AMT-130 for Huntington’s Disease

uniQure N.V. announced the issuance of two new patents covering AMT-130, the company’s gene therapy candidate for the treatment of Huntington’s disease. AMT-130 comprises a recombinant AAV5 vector carrying a DNA cassette, encoding a microRNA that non-selectively lowers or knocks-down human huntingtin protein in Huntington’s disease patients. [uniQure N.V.] Press Release

House Spending Panel Drops US Ban on Gene-Edited Babies

A Democrat-led spending panel in the US House of Representatives has dropped a provision that banned embryo editing with the intention of creating a baby. The draft bill is still moving through the legislative process, however, and Republicans will likely push to restore the language. [ScienceInsider] Editorial

Taiwan Considers Double-Blind Peer Review for Grants

Taiwan’s science ministry is thinking of introducing double-blind peer review to assess research-grant proposals, a trend being adopted by some journals to eliminate bias. [Nature News] Editorial

NIH

Proposed Collection; 60-Day Comment Request: Request for Human Embryonic Stem Cell Line to Be Approved for Use in NIH Funded Research (Office of the Director) (FR Doc. No:2019-10154) Notice

Prospective Grant of an Exclusive Patent License: RP2 AAV Vectors for Treating X-Linked Retinitis Pigmentosa (FR Doc. No:2019-10285) Notice

NEW 8^th InterAmerican Oncology Conference
October 17-18, 2019
Buenos Aires, Argentina

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

NEW Postdoctoral Scholarship – Gene Therapy for Osteopetrosis (Lund University)

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

Stem Cell Podcast Co-Host (Stem Cell Podcast)

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)

Postdoctoral Associate – Cancer Immunology (Winship Cancer Institute)

DPhil Scholarship – Retinal Gene Therapy (University of Oxford)

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

Associate Scientist – Immuno-Oncology (Hackensack Meridian Health Center for Discovery & Innovation)

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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