Cell Therapy News Volume 19.00 | Jan 8 2018

TOP STORY

CRISPR Treatment Prevents Hearing Loss in Mice

Using molecular scissors wrapped in a greasy delivery package, researchers disrupted a gene variant that leads to deafness in mice. A single treatment involving injection of a genome editing cocktail prevented progressive hearing loss in young animals that would have otherwise gone deaf, David Liu and colleagues report. [Press release from Howard Hughes Medical Institute discussing online prepublication in Nature] Press Release | Abstract | Editorial

(Ranked by impact factor of the journal)

YAP/TAZ-Dependent Reprogramming of Colonic Epithelium Links ECM Remodeling to Tissue Regeneration

In a defined cell culture system recapitulating the extracellular matrix (ECM) remodeling observed in vivo, the authors showed that a collagen 3D matrix supplemented with Wnt ligands is sufficient to sustain endogenous YAP/TAZ and induce conversion of cell fate. [Cell Stem Cell] Full Article | Graphical Abstract

Reconstruction of the Human Colon Epithelium In Vivo

Investigators established an orthotopic xenograft system for normal human colon organoids, enabling stable reconstruction of the human colon epithelium in vivo. Xenografted organoids formed crypt structures distinctively different from surrounding mouse crypts, reflecting their human origin. [Cell Stem Cell] Abstract | Graphical Abstract

ERBB3 and NGFR Mark a Distinct Skeletal Muscle Progenitor Cell in Human Development and hPSCs

Researchers observed that human pluripotent stem cell-skeletal muscle progenitor cells (SMPCs) derived by directed differentiation are less functional in vitro and in vivo compared to human satellite cells. Their enrichment and maturation strategy restored dystrophin in hundreds of dystrophin-deficient myofibres after engraftment of CRISPR–Cas9-corrected Duchenne muscular dystrophy human induced pluripotent stem cell-SMPCs. [Nat Cell Biol] Abstract | Press Release

Enhanced Human Hematopoietic Stem and Progenitor Cell Engraftment by Blocking Donor T Cell-Mediated TNFα Signaling

To mimic clinical hematopoietic stem cell transplantation, the authors injected human umbilical cord blood (UCB) cells at different doses and compositions into immunodeficient NOD/SCID/IL-2Rgc-null mice. Surprisingly, higher UCB cell doses inversely correlated with stem and progenitor cell engraftment. [Sci Transl Med] Abstract | Press Release

Human ESC-Derived Retinal Epithelial Cell Sheets Potentiate Rescue of Photoreceptor Cell Loss in Rats with Retinal Degeneration

Scientists showed that transplantation of the human embryonic stem cell (hESC)-retinal pigment epithelial (RPE) cell sheets grown on a human amniotic membrane scaffold resulted in rescue of photoreceptor cell death and improved visual acuity in rats with retinal degeneration compared to hESC-RPE cells injected as a cell suspension. [Sci Transl Med] Abstract

Transplanted Donor- or Stem Cell-Derived Cone Photoreceptors Can Both Integrate and Undergo Material Transfer in an Environment-Dependent Manner

Researchers determined whether stem cell- and donor-derived cones undergo integration and/or material transfer. They found that material transfer accounts for a significant proportion of rescued cells following cone transplantation into non-degenerative hosts. [Stem Cell Reports] Full Article | Graphical Abstract

A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies

The authors established a nonhuman primate (NHP) transplantation model to evaluate hematopoietic stem cell-based gene editing strategies aimed at reactivating fetal hemoglobin (HbF). After validating function of the HbF repressor BCL11A in NHPs, they transplanted a pigtailed macaque with CD34⁺ cells electroporated with TALE nuclease mRNA targeting the BCL11A coding sequence. [Mol Ther Methods Clin Dev] Full Article

Long-Term Persistence and Function of Hematopoietic Stem Cell-Derived Chimeric Antigen Receptor T Cells in a Nonhuman Primate Model of HIV/AIDS

Investigators report the use of a protective CD4 chimeric antigen receptor to redirect hematopoietic stem/progenitor cell-derived T-cells against simian/human immunodeficiency virus infection in pigtail macaques. CAR-containing cells persisted for more than two years without any measurable toxicity and were capable of multilineage engraftment. [PLoS Pathog] Full Article | Press Release

Transplantation of Gene-Edited Hepatocyte-Like Cells Modestly Improves Survival of Arginase-1-Deficient Mice

Scientists report transcription activator-like effector nuclease-mediated reincorporation of Arg1 exons 7 and 8 in induced pluripotent stem cells derived from arginase-1-deficient mice possessing Arg1Δ alleles lacking these terminal exons. The edited cells could be induced to differentiate into hepatocyte-like cells in vitro and were subsequently used for transplantation into a tamoxifen-inducible Arg1-Cre arginase-1-deficient mouse model. [Mol Ther Nucleic Acids] Full Article

Generation of Transplantable Retinal Photoreceptors from a Current Good Manufacture Practice-Manufactured Human Induced Pluripotent Stem Cell Line

Researchers used a human induced pluripotent stem cell (iPSC) line (NCL-1) derived under current Good Manufacture Practice-compliant conditions from CD34⁺ cord blood cells. They showed that retinal cells including photoreceptors, retinal pigmented epithelial cells and optic cup-like retinal organoids can be generated from the NCL-1 iPSC line. [Stem Cells Transl Med] Full Article

Generation of iPSC-Derived Limb Progenitor-Like Cells for Stimulating Phalange Regeneration in the Adult Mouse

Investigators established a simple and efficient protocol for deriving limb progenitor-like cells from mouse induced pluripotent stem cells (iPSCs). They report that both embryonic and iPSC-derived limb progenitor-like cells promoted adult mouse P2 regeneration. [Cell Discov] Full Article

Concise Review: Human Pluripotent Stem Cells to Produce Cell-Based Cancer Immunotherapy

Human pluripotent stem cells (PSCs) provide a promising resource to produce immune cells for adoptive cellular immunotherapy to better treat and potentially cure otherwise lethal cancers. Cytotoxic T cells and natural killer cells can now be routinely produced from human PSCs. [Stem Cells] Full Article

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

Juno Therapeutics Highlights Data and Presentations Supporting Best-in-Class Strategy for JCAR017

Juno Therapeutics, Inc., a biopharmaceutical company developing innovative cellular immunotherapies for the treatment of cancer, highlighted presentations and data. [Press release from Juno Therapeutics, Inc. discussing research presented at the 59^th American Society of Hematology (ASH) Annual Meeting, Atlanta] Press Release

Janssen Enters Worldwide Collaboration and License Agreement with Chinese Company Legend Biotech to Develop Investigational CAR-T Anti-Cancer Therapy

Janssen Biotech, Inc. announced that it has entered into a worldwide collaboration and license agreement with Legend Biotech USA Inc. and Legend Biotech Ireland Limited, subsidiaries of Genscript Biotech Corporation, to develop, manufacture and commercialize a CAR T-cell drug candidate, LCAR-B38M, which specifically targets the B-cell maturation antigen. [Janssen Global Services, LLC] Press Release

Vertex and CRISPR Therapeutics to Co-Develop and Co-Commercialize CTX001 as CRISPR/Cas9 Gene Edited Treatment for Sickle Cell Disease and β-Thalassemia

Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics AG announced that the companies will co-develop and co-commercialize CTX001, an investigational gene editing treatment, as part of the companies’ previously announced collaboration aimed at the discovery and development of new gene editing treatments that use the CRISPR/Cas9 technology. [Vertex Pharmaceuticals Incorporated] Press Release

FDA Approves Novel Gene Therapy to Treat Patients with a Rare Form of Inherited Vision Loss

The U.S. Food and Drug Administration approved Luxturna, a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene. [The U.S. Food and Drug Administration] Press Release

Singapore’s IP Office to Grant MilliporeSigma’s Patent Application for CRISPR Technology

MilliporeSigma announced that the Intellectual Property (IP) Office of Singapore has issued a “Notice of Eligibility for Grant” for MilliporeSigma’s patent application covering the company’s CRISPR technology used in a genomic-integration method for eukaryotic cells. [EMD Millipore Corporation] Press Release

ViaCyte Announces First Patients Implanted in Cohort 2 with Potentially Efficacious Dose of PEC-Direct Islet Cell Replacement Therapy

ViaCyte, Inc. announced that the first patients have been implanted in Cohort 2 with a potentially efficacious dose of the company’s PEC-Direct islet cell replacement therapy. This novel cell replacement therapy is being developed as a potential functional cure for patients with type 1 diabetes who are at high risk for acute life-threatening complications. [ViaCyte, Inc.] Press Release

Swedish Cell Therapy Candidate CellProtect Has Received Orphan Drug Designation for the Treatment of Multiple Myeloma

CellProtect Nordic Pharmaceuticals AB has been notified by the European Commission that its cell therapy candidate CellProtect has received orphan drug designation for the treatment of multiple myeloma. [CellProtect Nordic Pharmaceuticals AB (PR Newswire Association LLC.)] Press Release

Clinical Trial Utilizing BioAtla’s Conditionally Active Biologics in CAR-T Candidates for Solid Tumors to Be Initiated in China

BioAtla, LLC announced that Shanghai Sinobioway Sunterra Biotechnology has received ethics committee approval of a clinical trial for two novel, conditionally active CAR-T product candidates targeting Axl and Ror2 for the treatment of metastatic renal cell carcinoma. [BioAtla, LLC (PR Newswire Association LLC.)] Press Release

BioCardia Submits Clinical Trial to FDA for New Indication of Chronic Myocardial Ischemia for CardiAMP Cell Therapy

BioCardia^®, Inc. reported filing a second Investigational Device Exemption with the FDA for the CardiAMP Chronic Myocardial Ischemia Trial to treat patients with refractory angina. [BioCardia^®, Inc.] Press Release

HSS Researchers Receive Grant for Clinical Trial to Improve Outcomes for Rotator Cuff Tears

Hospital for Special Surgery (HSS) has been awarded the Orthopaedic Research and Education Foundation Clinical Research Grant in Cellular Therapy in Collaboration with National Stem Cell Foundation. The $800,000 grant will fund a clinical trial to determine if the use of stem cell therapy can improve outcomes for patients who suffer rotator cuff tears. [Hospital for Special Surgery] Press Release

CIRM Grants ViaCyte $1.4M to Create Immune-Evasive Pluripotent Stem Cell Lines

ViaCyte, Inc. announced that the California Institute for Regenerative Medicine (CIRM) approved a grant of $1.4 million to support the initial development of immune-evasive pluripotent stem cell lines. The focus of the project will be to genetically engineer the Company’s CyT49 pluripotent stem cell line. [ViaCyte, Inc.] Press Release

Indonesian Scientists Embrace Preprint Server

A preprint server that focuses exclusively on Indonesian research passed a milestone when the number of papers posted on it reached 1,500. [Nature News] Editorial

Germany vs Elsevier: Universities Win Temporary Journal Access after Refusing to Pay Fees

The Dutch publishing giant Elsevier has granted uninterrupted access to its paywalled journals for researchers at around 200 German universities and research institutes that had refused to renew their individual subscriptions at the end of 2017. [Nature News] Editorial

FDA

Best Practices for Communication between Investigational New Drug Application Sponsors and the Food and Drug Administration; Guidance for Industry and Review Staff; Availability (FR Doc. No:2017-28139) Notice

NEW World Advanced Therapies and Regenerative Medicine Congress 2018
May 16-18, 2018
London, United Kingdom

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

NEW Postdoctoral Research Fellows – Bioengineering and Nanotechnology (University of Queensland)

NEW National Operations Manager – Cellular and Molecular Therapies (NHS Blood & Transplant)

NEW Research Associate – Immunology and Gene Therapy (Fred Hutchinson Cancer Research Center)

NEW Postdoctoral Associate – Gene Therapy (University of Massachusetts)

Scientist – Gene Therapy (Medicines and Healthcare Products Regulatory Agency)

Postdoctoral Positions – Immunotherapy (University of Houston)

Assistant Scientist – Cancer Therapy Research (University of Miami)

Postgraduate Positions – Translational Medicine (Qingdao University)

Scientist – Immuno-Oncology (Regeneron Pharmaceuticals, Inc.)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

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