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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Issue Vol 3 Issue 2

Editorial

Cell and gene therapy: scaling up and moving to mass production

Editorial

Nigel Whittle

There is tremendous clinical promise in the application of therapies based on administering cells to patients to treat a wide range of diseases. In order to fulfil this promise, it is important that manufacture of these therapies can be ‘industrialized’ through transfer into a commercial setting in a scalable and cost-effective manner. This editorial provides an overview of the key challenges and new opportunities for the successful commercialization of cell therapies.

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Recent advances in non-viral vectors for gene therapy & vaccination

Spotlight Article

Editorial

Next Generation Vectors

Next-Generation Vectors Clague P Hodgson, Aaron E Carnes & James A Williams While the majority of gene therapy vectors are derived from viruses, their use introduces the issues of immunogenicity, carcinogenicity, insertional mutagenesis and reversion to replication competency. In addition, viral vectors often come with manufacturing bottlenecks such as scalability and processing. In order to […]

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The road to gene therapy for recessive dystrophic epidermolysis bullosa

Editorial

Next Generation Vectors

M Peter Marinkovich, Ngon T Nguyen & Zurab Siprashvili The road towards development of an optimal gene therapy for recessive dystrophic epidermolysis bullosa has been long and many lessons have been learned along the way. This editorial highlights the key advances and challenges in the field as it progress towards clinical translation. DOI: 10.18609/cgti.2017.011 Citation: […]

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Commercial Insights

Cell & Gene Therapy Commercial Insight – February 2017

Commercial Insights

Next Generation Vectors

Mark Curtis & Richard Philipson Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors. CELL THERAPY: Kite Pharma delivered new pivotal data this month that showed that there was a limited drop in complete response rate between […]

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Interview

Advances in the development of parvovirus-based anti-cancer therapies

Spotlight Article

Interview

Next Generation Vectors

Next-Generation Vectors Dr. Antonio Marchini Antonio Marchini is a Principal Investigator at the German Cancer Research Center (DKFZ, Heidelberg, Germany) and Head of the Laboratory of Oncolytic Virus Immuno Therapeutics (LOVIT), a newly established joint research unit between the Luxembourg Institute of Health and DKFZ. His team focuses on the development of new anticancer strategies […]

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Expert Insight

Minicircles: next-generation gene vectors

Spotlight Article

Expert Insight

Next Generation Vectors

Ram Shankar, Marco Schmeer & Martin Schleef Next Generation Vectors Plasmid DNA is commonly used in vaccination, cell and gene therapy, and as a basic substance in viral vector and RNA production. Backbone sequences in a plasmid vector are only needed for amplification in bacterial cultures. Since the uncontrolled expression of these sequences may have […]

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Non-viral gene transfer with Sleeping Beauty system to engineer T cells for hematologic malignancies and solid tumors

Expert Insight

Hiroki Torikai, Judy S Moyes & Laurence JN Cooper

Next Generation Vectors

The manipulation of genes has progressed to human application thanks in part to viral-based vectors that transduce a therapeutic gene and sustain its long-term expression. The adoptive transfer of genetically modified T cells against cancer highlights the potential for gene therapy to cure human disease refractory to conventional therapy. The improvements in the genetic manipulation of clinical-grade cells has widened possible treatment options with the realization that personalized gene therapy will be needed in which the introduced gene matches the needs of a particular patient. To match this growing need for individualized therapeutic genes, we have developed a rapid, robust and low-cost approach for clinical translation. This is based on a DNA plasmid-based transposon/transposase, termed the Sleeping Beauty (SB) system. The SB system stably introduces therapeutic genes, such as chimeric antigen receptors (CARs), into clinical-grade T cells and allows for long-term expression of the introduced transgene. Our first-in-human clinical trial of SB-mediated CAR-expressing T cells targeting B-cell malignancies established the safety, feasibility and efficacy of the SB system. This clinical application of the SB system is the foundation for future use of this tool to personalize genetically modified T cells targeting hematologic malignancies and especially solid tumors.

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Next-generation gene therapy vectors: alternative approaches to vector design and application

Spotlight Article

Expert Insight

Next Generation Vectors

Next-Generation Vectors Richard P Harbottle This issue of Cell and Gene Therapy Insights will focus on alternative next-generation vectors for gene therapy and the genetic modification of cells. Rather than discussing the use of attenuated viruses, which are the most commonly used vector system, this issue comprises reviews and commentary on a range of alternative […]

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Sleeping Beauty transposon vectors for therapeutic applications: advances and challenges

Spotlight Article

Expert Insight

Next Generation Vectors

Suneel A Narayanavari & Zsuzsanna Izsvák Next-Generation Vectors Transposable elements are natural, non-viral gene delivery vehicles capable of mediating stable genomic integration. The Sleeping Beauty (SB) transposon has the ability to cut-and-paste the ‘gene of interest’ into the genome, providing the basis for long-term, permanent transgene expression in transgenic cells and organisms. The SB transposon […]

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Specifically integrating vectors for targeted gene delivery: progress and prospects

Spotlight Article

Expert Insight

Next Generation Vectors

Adrian Kovač & Zoltán Ivics Next-Generation Vectors Integrating vectors based on viruses or transposons are efficient gene delivery vehicles and promising tools for gene therapy. While different vector systems have different preferences and biases when it comes to target site selection, integration can always occur at vast numbers of potential sites throughout the human genome. […]

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