Cell Therapy News Volume 20.09 | Mar 18 2019

TOP STORY

With Single Gene Insertion, Blind Mice Regain Sight

It was surprisingly simple. Scientists inserted a gene for a green-light receptor into the eyes of blind mice and, a month later, they were navigating around obstacles as easily as mice with no vision problems. [Press release from UC Berkeley discussing online prepublication in Nature Communications] Press Release | Abstract

(Ranked by impact factor of the journal)

Organoid-Derived C-Kit⁺/SSEA4⁻ Human Retinal Progenitor Cells Promote a Protective Retinal Microenvironment during Transplantation in Rodents

Scientists used cell surface markers to effectively eliminate tumorigenic embryonic cells and enrich retinal progenitor cells from human embryonic stem cell-derived retinal organoids, which, following subretinal transplantation into retinal degeneration models of rats and mice, significantly improved vision and preserved the retinal structure. [Nat Commun] Full Article

Genetically Engineered CAR NK Cells Display Selective Cytotoxicity against FLT3‐Positive B‐ALL and Inhibit In Vivo Leukemia Growth

In a SEM B cell acute lymphoblastic leukemia (B‐ALL) xenograft model in NOD‐SCID IL2R γ^null mice, treatment with chimeric antigen receptor (CAR) natural killer (NK)‐92 but not parental NK‐92 cells markedly inhibited disease progression, demonstrating high anti‐leukemic activity in vivo. [Int J Cancer] Abstract

Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy

Researchers describe the sustained delivery of therapeutic human α-galactosidase protein in vivo via nanoparticle-formulated mRNA in mouse and non-human primate, with a demonstration of efficacy through clinically relevant biomarker reduction in a mouse Fabry disease model. [Mol Ther] Full Article | Press Release

AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

Adeno-associated viral vector serotype 5 expressing a microRNA targeting human huntingtin (AAV5-miHTT) caused a dose-dependent and sustained huntingtin protein reduction with subsequent suppression of mutant huntingtin aggregate formation in the striatum and cortex. [Mol Ther Methods Clin Dev] Abstract | Full Article

Adipose‐Derived Stem Cell Therapy Ameliorates Ionizing Irradiation Fibrosis (RIF) via Hepatocyte Growth Factor Mediated TGF‐β Down Regulation and Recruitment of Bone Marrow Cells

A single adipose‐derived stem cell injection on day 28 at the irradiated site decreased by day 40: epithelial thickness, collagen deposition, and significantly improved limb excursion compared to irradiated controls. [Stem Cells] Abstract

Therapeutic Targeting of Stat3 Using Lipopolyplex Nanoparticle-Formulated siRNA in a Syngeneic Orthotopic Mouse Glioma Model

In a therapeutic setting, intracranial application of the siRNA-containing lipopolyplexes led to knockdown of signal transducer and activator of transcription 3 (STAT3) target gene expression, decreased tumor growth and significantly prolonged survival in Tu2449 glioma-bearing mice compared to negative control-treated animals. [Cancers] Full Article

Antitumor Efficacy of Chimeric Antigen Receptor T Cells against EGFRvIII-Expressing Glioblastoma in C57BL/6 Mice

A low dose of 806-28Z chimeric antigen receptor (CAR) T cells suppressed GL261/epidermal growth factor receptor variant III (EGFRvIII) tumor growth, whereas a high dose of 806-28Z CAR T cells completely eradicated xenograft tumors. [Biomed Pharmacother] Full Article | Graphical Abstract

Mesenchymal Stromal Cells Modulate Tissue Repair Responses within the Injured Vocal Fold

Investigators determined whether local injection of human mesenchymal stromal cells could modulate the early inflammatory response within injured vocal folds to promote wound‐healing processes. [Laryngoscope] Abstract

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CRISPR/Cas9 – An Evolving Biological Tool Kit for Cancer Biology and Oncology

This review discusses the diverse applications of CRISPR-based gene-editing tools in oncology and potential future cancer therapies. [npj Precis Oncol] Full Article

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

Orchard Therapeutics Announces Acceptance of OTL-200 Abstract for MLD

Orchard Therapeutics announced that new clinical data from the registrational trial of OTL-200 in metachromatic leukodystrophy (MLD) will be featured in an oral presentation. [Press release from Orchard Therapeutics discussing research to be presented at the 45^th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT), Frankfurt] Press Release

Amphera Recruits First Patients to Pivotal Phase II/III Study of MesoPher to Treat Pleural Mesothelioma

Amphera B.V. announced that it has recruited the first patients to its pivotal Phase II/III study of MesoPher to treat pleural mesothelioma, a rare cancer of the lining of the lungs. [Amphera B.V.] Press Release

BrainStorm Announces First Patient Enrolled in Phase II Clinical Trial of Progressive MS

BrainStorm Cell Therapeutics Inc. announced that the first patient has been enrolled in its Phase II open-label, multicenter study of repeated intrathecal administration of autologous MSC-NTF cells in participants with progressive Multiple Sclerosis (MS). [BrainStorm Cell Therapeutics Inc.] Press Release

Pluristem Therapeutics Announces Completion of 2^nd Cohort and DSMB Approval to Enroll Final Cohort in Phase I Hematological Study of PLX-R18

Pluristem Therapeutics Inc. announced that the company has fully enrolled the second cohort of six patients in its ongoing Phase I clinical study evaluating PLX-R18 for the treatment of incomplete hematopoietic recovery following hematopoietic cell transplantation, and has received Data and Safety Monitoring Board (DSMB) approval to continue to the final cohort of the study. [Pluristem Therapeutics Inc.] Press Release

Rocket Pharmaceuticals Announces First Patient Dosed in Phase I Clinical Trial of RP-L102 in the U.S.

Rocket Pharmaceuticals, Inc. announces that the first patient has been dosed in the open-label, Phase I clinical trial of RP-L102, the company’s lentiviral vector-based gene therapy for the treatment of Fanconi Anemia. [Rocket Pharmaceuticals, Inc.] Press Release

Arrowhead Pharmaceuticals Files IND for Pivotal Phase II/III Study of ARO-AAT for Treatment of Alpha-1 Liver Disease

Arrowhead Pharmaceuticals Inc. announced that it has submitted an Investigational New Drug (IND) application to the FDA for an adaptive Phase II/III trial with the potential to serve as a pivotal registrational study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency. [Arrowhead Pharmaceuticals Inc.] Press Release

Cynata Receives Favourable Advice from UK MHRA on CLI Clinical Trial

Cynata Therapeutics Limited announced that it has received favourable advice from the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom (UK), regarding its planned Phase II clinical trial of CYP-002 in patients with critical limb ischemia (CLI). [Cynata Therapeutics Limited (GlobeNewswire, Inc.)] Press Release

Rubius Therapeutics Announces FDA Clearance of Investigational New Drug Application for First-Ever Red Cell Therapeutic, RTX-134, for Treatment of Phenylketonuria

Rubius Therapeutics, Inc. announced that the FDA has cleared the company’s Investigational New Drug application for RTX-134, an allogeneic, off-the-shelf cellular therapy for the potential treatment of patients with phenylketonuria. [Rubius Therapeutics, Inc.] Press Release

NEOVIVA Project Supporting the Development of myvac™ Awarded a €5.2 Million Grant from Bpifrance’s “Investments for the Future” Program

Transgene announced that the NEOVIVA project was selected by the “Investments for the Future” Program operated by Bpifrance for the development of an industrial sector focused on Transgene’s individualized immunotherapy platform myvac™. [Transgene SA] Press Release

Leading Scientists, Backed by NIH, Call for a Global Moratorium on Creating ‘CRISPR Babies’

In an effort to prevent another He, 18 scientists from seven countries have called for “a global moratorium on all clinical uses of human germline editing” — that is, changing DNA in sperm, eggs, or early embryos to make genetically altered children, alterations that would be passed on to future generations. [STAT News] Editorial

Japan Poised to Allow ‘Reprogrammed’ Stem-Cell Therapy for Damaged Corneas

A Japanese committee has provisionally approved the use of reprogrammed stem cells to treat diseased or damaged corneas. Researchers are now waiting for final approval from the health ministry to test the treatment in people with corneal blindness, which affects millions of people around the world. [Nature News] Editorial

FDA

Cancer Clinical Trial Eligibility Criteria: Patients with Human Immunodeficiency Virus, Hepatitis B Virus, or Hepatitis C Virus Infections; Draft Guidance for Industry; Availability (FR Doc. No:2019-04572) Notice

Considerations for the Inclusion of Adolescent Patients in Adult Oncology Clinical Trials; Guidance for Industry; Availability (FR Doc. No:2019-04582) Notice

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting (FR Doc. No:2019-04708) Notice

NEW 2019 International Society for Cellular Therapy (ISCT)
May 29-June 1, 2019
Melbourne, Australia

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

NEW Postdoctoral Research Associate – Retinal Stem Cell Biology and Regeneration (University of Nebraska Medical Center)

Research Technologist – Immunology (STEMCELL Technologies Inc.)

Research Associate – Bioengineering (STEMCELL Technologies Inc.)

Cell Biologist – Regenerative Medicine (Hepatx Corporation)

Stem Cell Podcast Co-Host (Stem Cell Podcast)

Associate Scientist – Immuno-Oncology (HMH Center for Discovery & Innovation)

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

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)

Cell Manufacturing Scientist (CSL Behring)

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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