Cell Therapy News Volume 18.27 | Aug 14 2017

TOP STORY

Inducible Activation of MyD88 and CD40 in CAR T-Cells Results in Controllable and Potent Antitumor Activity in Preclinical Solid Tumor Models

T-cells expressing HER2-CARζ and a MyD88/CD40-based inducible co-stimulatory (iCO) molecule (HER2ζ.iCO) T-cells with chemical inducer of dimerization (CID) significantly improved survival in vivo in two xenograft models. Repeat injections of CID were able to further increase the antitumor activity of HER2ζ.iCO T-cells in vivo. [Cancer Discov] Abstract

(Ranked by impact factor of the journal)

Cardiac and Systemic Rejuvenation after Cardiosphere-Derived Cell Therapy in Senescent Rats

Cardiosphere-derived cell (CDC) therapy has exhibited several favorable effects on heart structure and function in humans and in preclinical models; however, the effects of CDCs on aging have not been evaluated. Researchers compared intra-cardiac injections of neonatal rat CDCs to vehicle in 21.8 ± 1.6 month-old rats. [Eur Heart J] Abstract | Press Release

Immune Tolerance Induced by Platelet-Targeted Factor VIII Gene Therapy in Hemophilia A Mice Is CD4 T Cell-Mediated

The authors investigated whether non-selectable platelet-targeted FVIII (2bf8) lentiviral vector (LV) for the induction of platelet-FVIII expression is sufficient to induce immune tolerance and how immune tolerance is induced after 2bF8LV gene therapy. [J Thromb Haemost] Abstract

Human Adipose-Derived Mesenchymal Stem Cells Seeded into a Collagen-Hydroxyapatite Scaffold Promote Bone Augmentation after Implantation in the Mouse

Human adipose-derived stem cells are considered as the better candidate for autologous cell transplantation. In order to evaluate the regenerative potential of these cells along with an osteoinductive biomaterial, investigators used collagen/hydroxyapatite scaffolds to test ectopic bone formation after subcutaneous implantation in mice. [Sci Rep] Full Article

Clinical and Radiological Regeneration of Large and Deep Osteochondral Defects of the Knee by Bone Augmentation Combined with Matrix-Guided Autologous Chondrocyte Transplantation

The authors evaluated one- to three-year clinical outcomes and radiological results on magnetic resonance imaging after the treatment of large osteochondral defects of the knee with bone augmentation and matrix-guided autologous chondrocyte transplantation. [Am J Sports Med] Abstract

Alginate-Gelatin Encapsulation of Human Endothelial Cells Promoted Angiogenesis in In Vivo and In Vitro Milieu

The in vivo angiogenic potential of encapsulated HUVECs was analyzed in immune-compromized mouse implant model during seven days post-transplantation. An enhanced in vivo angiogenic response of encapsulated HUVECs was evident as compared to non-capsulated cells. [Biotechnol Bioeng] Abstract

Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model

Scientists packaged minigene dual vectors in Y731F tyrosine-modified adeno-associated virus (AAV)-9 and delivered to the extensor carpi ulnaris muscle of a 12-month-old affected dog at the dose of 2×1013 viral genome particles/vector/muscle. [Hum Gene Ther] Abstract

Bifidobacterium breve as a Delivery Vector of IL-24 Gene Therapy for Head and Neck Squamous Cell Carcinoma In Vivo

The authors investigated the therapeutic effect of new recombinant B. breve strain expressing IL-24 gene on head and neck tumor xenograft in mice. [Gene Ther] Abstract

AAV5-miHTT Gene Therapy Demonstrated Suppression of Mutant Huntingtin Aggregation and Neuronal Dysfunction in a Rat Model of Huntington’s Disease

The mechanistic efficacy and gene specificity of selected constructs delivered by an adeno-associated viral serotype 05 (AAV5) vector was addressed using an acute Huntington’s disease rat model. Researchers demonstrated suppression of mutant huntingtin (HTT) mRNA, which almost completely prevented mutant HTT aggregate formation, and ultimately resulted in suppression of DARPP-32-associated neuronal dysfunction. [Gene Ther] Abstract

CRISPR Editing in Biological and Biomedical Investigation

The authors outline different applications and current limitations of CRISPR use in biological and biomedical investigation. They provide a perspective for future development and potential risks of this multifunctional technology. [J Cell Physiol] Abstract

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

Benitec Biopharma Advances OPMD Orphan Disease Program

Benitec Biopharma Limited announced it has developed a new single vector system that uses DNA directed RNAi to silence expression of the mutant gene associated with oculopharyngeal muscular dystrophy (OPMD), while simultaneously adding back a copy of the normal version of the same gene to restore gene function. [Press release from Benitec Biopharma Limited PR Newswire Association LLC.) discussing research to be presented at the 2017 Cell & Gene Meeting on the Mesa Conference, La Jolla] Press Release

Celyad Announces New Agreements with Celdara Medical and Dartmouth College

Celyad and its fully-owned subsidiary, OnCyte LLC, announced revised terms to their agreements with Celdara Medical LLC and Dartmouth College. [Celyad] Press Release

Agilis Biotherapeutics and Gene Therapy Research Institution Enter into Strategic Partnership

Agilis Biotherapeutics, Inc. and Gene Therapy Research Institution Co, Ltd. announced that the companies have completed a manufacturing and collaboration partnership joint venture to advance adeno-associated virus gene therapies. [Agilis Biotherapeutics, Inc.] Press Release

CARsgen Launches First-in-Class Anti-Claudin18.2 CAR-T Clinical Trial

CARsgen Therapeutics announced its first-in-class anti-Claudin18.2-CAR-T cell therapy for gastric and pancreatic cancers has entered into Phase-I clinical trial and starts patients recruitment at Changhai Hospital of Shanghai. [CARsgen Therapeutics (PR Newswire Association LLC.)] Press Release

BioTime Announces $2 Million Grant for Further Development of OpRegen^® for Dry-AMD

BioTime, Inc. has been awarded a new grant for 2017 of up to 7.2 million Israeli New Shekels from the Israel Innovation Authority (IIA). The grant provides funding for the continued development of OpRegen^®, and to date the IIA has provided annual grants totaling approximately $12 million. [BioTime, Inc.] Press Release

BioMarin Announces Plans to Progress Both the 6e13 vg/kg and 4e13 vg/kg Doses of BMN 270, Its Investigational Gene Therapy for Hemophilia A, into Phase III Studies

BioMarin Pharmaceutical Inc. announced that it will expand its development plan for BMN 270, its investigational gene therapy for hemophilia A, to include an additional Phase III study of the 4e13 vg/kg dose based on updated data from its ongoing open-label Phase I/II study of BMN 270. [BioMarin Pharmaceutical Inc.] Press Release

RXi Pharmaceuticals Advances Immuno-Oncology Pipeline by Selecting Two sd-rxRNA Compounds for Preclinical Development and Sourcing cGMP Manufacturing

RXi Pharmaceuticals Corporation announced that it has selected two self-delivering RNAi compounds from its immuno-oncology pipeline for preclinical development. [RXi Pharmaceuticals Corporation] Press Release

ViaCyte Announces First Patients Implanted with PEC-Direct Islet Cell Replacement Therapy in International Clinical Trial

ViaCyte, Inc. announced that the first patients have been implanted with the PEC-Direct™ product candidate, a novel islet cell replacement therapy in development as a functional cure for patients with type 1 diabetes who are at high risk for acute life-threatening complications. [ViaCyte, Inc.] Press Release

Kite Submits Investigational New Drug (IND) Application for KITE-585, Anti-BCMA CAR-T Therapy Candidate for Multiple Myeloma

Kite Pharma, Inc. announced the submission of an IND application with the FDA to initiate a Phase I, first-in-human trial of KITE-585, a CAR-T cell therapy engineered to target B-cell maturation antigen (BCMA) in patients with relapsed/refractory multiple myeloma. [Kite Pharma, Inc.] Press Release

Kite Announces Initiation of Axicabtagene Ciloleucel CAR-T Clinical Program in the European Union

Kite Pharma, Inc. announced that patients in the European Union are now being treated with its lead investigational candidate, axicabtagene ciloleucel, in the safety expansion cohort of ZUMA-1. [Kite Pharma, Inc.] Press Release

US Biomedical-Research Facilities Unprepared for Attacks and Natural Disasters

When Hurricane Sandy hit New York City in 2012, the storm destroyed scientific equipment worth more than US$20 million at the New York University Langone Medical Center. Tropical Storm Allison hit the University of Texas Health Science Center in Houston in 2001, and caused so much damage that some researchers had to restart their careers elsewhere. Despite such catastrophes, a report found that many research institutions in the United States are still unprepared for disasters. [Nature News] Editorial

Thousands across India March in Support of Science

Thousands of scientists, university students and science enthusiasts gathered in dozens of Indian cities to march in support of science — lamenting their country’s low levels of funding for research, and complaining about government promotion of ‘unscientific ideas’. [Nature News] Editorial

New FDA Security Rules Will Bar Agency from Hiring Some Foreign Nationals

The FDA is moving to adopt a new policy on security background checks that is sowing confusion and alarm among some of its scientists. The change, described in a “communications plan” shared with the agency’s senior staff in recent weeks, appears to effectively bar the agency from hiring—as employees or contractors—foreign nationals who have not lived in the United States for a total of three out of the last five years. [ScienceInsider] Editorial

FDA

Prospective Grant of Exclusive Patent License: MicroRNA Therapeutics for Treating Squamous Cell Carcinomas (FR Doc. No:2017-16524) Notice

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; De Novo Classification Process (Evaluation of Automatic Class III Designation) (FR Doc. No:2017-16230) Notice

NIH

Scientific Advisory Committee on Alternative Toxicological Methods; Announcement of Meeting; Request for Comments (FR Doc. No:2017-17036) Notice

Prospective Grant of Exclusive Patent License: The Development of a Bispecific, Biparatopic Antibody-Drug Conjugate to GPC3 for the Treatment of Human Liver Cancers (FR Doc. No:2017-16525) Notice

NEW 5^th Annual European Congress on Clinical & Translational Sciences (EUSTM-2017)
October 20-22, 2017
Berlin, Germany

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

NEW Postdoctoral Associate – Enhancer RNA Therapy (Sylvester Comprehensive Cancer Center)

Postdoctoral Fellow – Cell and Gene Therapy Studies Targeting Heart Failure (Icahn School of Medicine at Mount Sinai)

Research Group Leaders (Centro de Investigación Príncipe Felipe)

Postdoctoral Research Associate – Molecular Mechanisms of Stem Cells in the Heart (University of Louisville)

Research Associate – Cell Engineering (Caribou Biosciences, Inc.)

Quality Improvement Coordinator – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Scientist – Analytical Development (KBI Biopharma)

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

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

Postdoctoral Fellow – Various Projects (University of Oklahoma)

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