Cell Therapy News Volume 18.16 | May 8 2017

TOP STORY

CRISPR/Cas9 Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo

Scientists established a CRISPR/Cas9 gene editing system in human CD34⁺ hematopoietic stem/progenitor cells and achieved efficient CCR5 ablation evaluated in long-term reconstituted NOD/Prkdc^scid/IL-2Rγ^null mice. [Mol Ther] Abstract | Full Article

(Ranked by impact factor of the journal)

Tbet Is a Critical Modulator of FoxP3 Expression in Autoimmune Graft versus Host Disease

Using an established murine model that isolates the autoimmune component of graft-versus-host disease, the authors hypothesized that Th1 cells would restrict FoxP3-driven regulatory T cells. Upon transfer into immune-deficient syngeneic hosts, alloreactive Tbx21^-/- CD4⁺ T cells led to marked increases in FoxP3⁺ cells and reduced clinical evidence of autoimmunity. [Haematologica] Abstract

Partial Break in Tolerance of NKG2A⁻/LIR-1⁻ Single KIR⁺ NK Cells Early in the Course of HLA-Matched, KIR-Mismatched Hematopoietic Cell Transplantation

Natural killer (NK) cell subpopulations from eight HLA-matched but killer cell immunoglobulin-like receptor (KIR)/HLA-ligand-mismatched patient–donor pairs were analyzed in the course of allogeneic hematopoietic stem cell transplantation. [Bone Marrow Transplant] Abstract

Potential Mechanisms of MicroRNA-141-3p to Alleviate Chronic Inflammatory Pain by Down-Regulation of Downstream Target Gene HMGB1: In Vitro and In Vivo Studies

The authors investigated the potential role of microRNA-141-3p in chronic inflammatory pain by targeting HMGB1 gene. [Gene Ther] Abstract

Allogeneic pASC Transplantation in Humanized Pigs Attenuates Cardiac Remodeling Post-Myocardial Infarction

Researchers tested whether increasing doses of porcine adipose-tissue derived mesenchymal stem cells (pASCs) increase cardiac tissue perfusion in pigs post-myocardial infarction receiving angiotensin-converting-enzyme inhibitor and Beta-blockers similarly to patients. [PLoS One] Full Article

Transplantation of HGF Gene-Engineered Skeletal Myoblasts Improve Infarction Recovery in a Rat Myocardial Ischemia Model

Scientists evaluated the therapeutic effects of hepatocyte growth factor (HGF) gene-engineered skeletal myoblasts on tissue regeneration and restoration of heart function in a rat myocardial infarction model. [PLoS One] Full Article

In-Vivo Evaluation of Subcutaneously Implanted Cell-Loaded Apatite Microcarriers for Osteogenic Potency

The authors evaluated the osteogenic potency of human foetal mesenchymal stem cells-loaded apatite microcarriers when implanted subcutaneously in a mouse model. [J Mater Sci Mater Med] Abstract

Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery

To reduce peripheral transgene expression, researchers explored the safety and efficacy of adeno-associated virus serotype 9 (AAV9)/hMECP2 injected into the cisterna magna. AAV9/hMECP2 extended Mecp2^−/y survival but aggravated hindlimb clasping and abnormal gait phenotypes. [Mol Ther Methods Clin Dev] Full Article | Graphical Abstract

Platelets Subvert T Cell Immunity against Cancer via GARP-TGFβ Axis

Investigators showed that genetic targeting of platelets enhanced adoptive T cell therapy of cancer. An unbiased biochemical and structural biology approach established transforming growth factor β (TGFβ) and lactate as major platelet-derived soluble factors to obliterate CD4⁺ and CD8⁺ T cell functions. [Sci Immunol] Abstract

Current Application of CRISPR/Cas9 Gene-Editing Technique to Eradication of HIV/AIDS

The authors summarize the most recent progress in the application of the CRISPR/Cas9 gene-editing technique to HIV/AIDS therapy and elimination. Future directions and trends of such applications are also discussed. [Gene Ther] Abstract

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

Presidential Symposium at the American Society of Gene and Cell Therapy (ASGCT) 20^th Annual Meeting Will Feature Presentation on Asterias’ AST-OPC1 for Spinal Cord Injury

Asterias Biotherapeutics, Inc. announced that data from its AST-OPC1 clinical program for severe cervical spinal cord injury will be presented. [Press release from Asterias Biotherapeutics, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 20^th Annual Meeting, Washington D.C.] Press Release

GenSight Biologics to Present Data on GS010 and GS030

GenSight Biologics announced that five abstracts were accepted for one oral and four poster presentations. [Press release from GenSight Biologics (Business Wire, Inc.) discussing research to be presented at the 2017 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Baltimore] Press Release

Be The Match BioTherapies Announces Collaboration Agreement with Magenta Therapeutics

Be The Match BioTherapies^SM announced that it has entered into a strategic partnership with Magenta Therapeutics. The collaboration is intended to support efforts to improve transplant outcomes and expand the application of stem cell transplantation into disease indications that include autoimmunity, serious inherited immune and metabolic disorders, blood defects and blood cancers. [Be The Match BioTherapies^SM] Press Release

TxCell and Inserm Collaborate to Develop New CAR-Tregs in Transplantation and Multiple Sclerosis

TxCell SA and Inserm Transfert, on behalf of Inserm and the Nantes University, announced the signature of a R&D collaboration agreement. This collaboration agreement complements the December 2016 exclusive worldwide licensing agreement pertaining to a new subset of Treg cells originated in one of the Inserm laboratories. [TxCell SA] Press Release

First Patient Enrolled in Mallinckrodt Phase II Trial of StrataGraft^® Regenerative Skin Tissue

Mallinckrodt plc confirmed enrollment of the first patient in the company’s Phase II study assessing the safety, tolerability and efficacy of Stratagraft^® regenerative skin tissue as an alternative to autografting full-thickness complex skin defects. [Mallinckrodt plc] Press Release

European Commission Approves KEYTRUDA^® (Pembrolizumab) for Patients with Relapsed or Refractory Classical Hodgkin Lymphoma Who Failed Autologous Stem Cell Transplant and Brentuximab Vedotin (BV), or Who Are Transplant-Ineligible and Have Failed BV

Merck announced that the European Commission has approved KEYTRUDA^®, the company’s anti-PD-1 therapy, for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma who have failed autologous stem cell transplant and brentuximab vedotin, or who are transplant-ineligible and have failed BV. [Merck Sharp & Dohme Corp.] Press Release

University of Miami Health System Starts Clinical Trial to Test Regenerative Treatment for Stroke

The University of Miami Health System is starting a clinical trial to further evaluate the safety and efficacy of an investigational cell therapy for the treatment of chronic motor deficit following an ischemic stroke. [The University of Miami Health System (PR Newswire Association LLC.)] Press Release

Celyad Grants to Novartis a Non-Exclusive License for its Allogeneic TCR-Deficient CAR-T Cells Patents

Celyad announced a non-exclusive license agreement with Novartis for Celyad’s US patents for the production of allogeneic chimeric antigen receptor (CAR)-T cells. [Celyad] Press Release

bluebird bio Enters Lentiviral Vector Patent License Agreement with GlaxoSmithKline for Commercialization of Gene Therapies

bluebird bio, Inc. announced that it has entered into a worldwide license agreement around its proprietary lentiviral vector platform with GlaxoSmithKline Intellectual Property Development Limited. [bluebird bio, Inc.] Press Release

bluebird bio Licenses Lentiviral Vector Patent Rights for Development and Commercialization of Cell Therapies

bluebird bio, Inc. announced that it has entered into a worldwide license agreement around its proprietary lentiviral vector platform with Novartis Pharma AG. [bluebird bio, Inc.] Press Release

CARsgen Therapeutics Opens CAR T-Cell Manufacturing Facility in Shanghai

CARsgen Therapeutics announced the opening of its new manufacturing facility in Shanghai. The facility features state-of-art capabilities to manufacture vectors and chimeric antigen receptor (CAR)-T cells to support CARsgen clinical programs. [CARsgen Therapeutics, Ltd] Press Release

Scientists Relieved by Emmanuel Macron’s French Election Victory

French scientists say they’re relieved and happy that their country’s next president will be Emmanuel Macron, a 39-year-old former civil servant and economy minister who swept to victory in elections. Macron intends to make cuts to public spending, but has said he will ring-fence the budgets for research and higher education, areas that he wants to make the central plank of a program to boost innovation and cut unemployment. [Nature News] Editorial

Money Still Missing as the Plan to Synthesize a Human Genome Takes Another Step Forward

More than 200 biologists, businesspeople, and ethicists will converge upon the New York Genome Center in Manhattan to jumpstart what they hope will be biology’s next blockbuster: Genome Project-write, a still-unfunded sequel to the Human Genome Project where instead of reading a human genome, scientists create one from scratch and incorporate it into cells for various research and medical purposes. [ScienceInsider] Editorial

French Plan to Create €5-Billion Science ‘Super-Campus’ in Disarray

French ambitions to create a €5-billion (US$5.5-billion) science ‘super-campus’ near Paris by 2020 seem to be in falling further apart, after a compromise scheme to save the troubled project was rejected by one of its creators. [Nature News] Editorial

NIH Grant Limits Rile Biomedical Research Community

A decision by the US National Institutes of Health (NIH) to limit its grant support for individual researchers has sparked concerns that the policy could discourage collaboration or divert funding from the best science. The move has alarmed some researchers who agree with the NIH’s stated aim of freeing up money for young scientists, who often struggle to obtain research grants. [Nature News] Editorial

NIH

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No:2017-08783) Notice

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No:2017-09289) Notice

NEW Gordon Research Conferences: Cell Growth & Proliferation
July 9-14, 2017
Mount Snow, United States

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

NEW Director – Quality Control (KBI Biopharma)

NEW Scientist – Analytical Development (KBI Biopharma)

Lecturer – Biomedical Sciences (University of Essex)

PhD Fellow – Experimental Genetic Cardiology (Oslo University Hospital)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Program Director – CAR-T Cell (Servier)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

Executive Director – Global CAR-T Supply (Celgene Corporation)

Postdoctoral Associate – Gene Therapy (University of Massachusetts Medical School)

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

Postdoctoral Fellow – Various Projects (University of Oklahoma)

Staff Research Associate III – Translational Research to Clinical Cell Therapies (University of California Davis Health System)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center)

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