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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Spotlights 2015/16

Translation and Application of Gene Editing

December 2015 The rapidly advancing developments in gene editing have numerous implications and applications for the cell and gene therapy field – from disease modelling through to the potential editing of patient-derived cells to modify/correct disorders. The Gene Editing spotlight features a lead review from Prof. Andrew Bassett, Oxford University, UK discussing the main gene-editing technologies in detail: Zinc Fingers, TALENs and CRISPR-Cas9, comparing mechanisms of action, limitations and opportunities for future development and applications. Building upon this, we present a series of Expert Insight articles discussing potential clinical applications in more detail including: Cystic fibrosis Duchenne Muscular Dystrophy Genetic blood disorders and hemoglobinopathies Alongside the discussion of the incredible advances in gene editing applications, an ongoing patent dispute plays out in the media over the CRISPR-Cas9 technology. Dr Beatriz San Martin, Partner, FieldFisher, UK helps demystify and explain the IP situation and talks about the possible implications of the outcome of this dispute. We also hear from gene editing industry leaders, Sangamo BioSciences, as Dr Fyodor Urnov discusses their exciting progress towards the clinic with zinc finger nucleases in our exclusive podcast interview.

The Cell & Gene Therapy Manufacturing Pathway: Part 1

Supported by GE Healthcare Innovation and Excellence in ATMP Manufacturing PART 1: Defining Commercial Attributes Early in Product Development The sector has recently seen an influx of investment and interest from Big Pharma and VCs, in part due to the promising early clinical data from immunotherapies such as CAR T cells and engineered T cells. However, many questions and challenges remain on the path to successfully translating a cell-based therapy into a commercially viable product. Our expert Guest Editors – Prof. Peter Zandstra, University of Toronto; Dr Stephen Ward, Cell Therapy Catapult; and Dr Greg Russotti, Celgene Cellular Therapeutics – have identified the critical issues along the manufacturing pathway and these will be discussed across a 4-part Spotlight Series.

The Cell & Gene Therapy Manufacturing Pathway: Part 2

Supported by GE Healthcare Innovation and Excellence in ATMP Manufacturing PART 2: Integration of manufacturing and delivery into healthcare systems In Part One of our Cell & Gene Therapy Manufacturing Pathway spotlight series we focused on the early stages of product development, examining the key attributes that will eventually impact commercial viability. Here Part Two takes us on a step to evaluate potential solutions to the core manufacturing and logistical challenges involved in cost-effectively delivering cell and gene therapies to patients. Working with our expert Guest Editors – Dr Gregory Russotti (Celgene Cellular Therapeutics, USA), Dr Stephen Ward (Cell Therapy Catapult, UK), and Dr Peter Zandstra (Univ. Toronto, Canada) – we have selected world-leading authors from multiple stakeholder groups: academic centers, translational institutions, cell and gene therapy manufacturers, and equipment and service providers; all of whom are invested in progressing the translation of this promising new technology.

Advanced Cell Therapies – Progress Towards the Clinic

GUEST EDITORS Dr Mahendra Rao, Founder and CSO at Mahendra Rao LLC, USA and Prof. Jun Takahashi, Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan. The discovery of pluripotent stem cells and their capacity for cell-type-specific differentiation have revolutionized our approach to cell therapy. This spotlight showcases the latest clinical advances in utilizing pluripotent cells and discusses potential solutions to the core challenges related to their manufacture, distribution and delivery.

The Cell & Gene Therapy Manufacturing Pathway: Part 3

Supported by GE Healthcare Innovation and Excellence in ATMP Manufacturing PART 3: Understanding your product and processes Guest editors: Greg Russotti, Stephen Ward & Peter Zandstra As more cell and gene therapies move towards the clinic, manufacturers are faced with a number of challenges pertaining to the move from clinical-stage bioprocesing to commercial-scale manufacturing. What are the implications of making process changes as you move towards commercialization and how can you control for these variables and the potential risks they introduce? This spotlight addresses these critical questions including.

Automation and single-use systems in cell & gene therapy manufacture: Realizing the potential

GUEST EDITOR Dr Qasim Rafiq, Assistant Professor in Bioprocess Engineering at Aston University, UK This Spotlight focuses on answering the key questions facing those grappling with how to use automation in the production of their cell and gene therapy candidates. At what stage in process development should I first consider automated single-use systems? What impact can I expect automation to have on cost, quality and reproducibility?

Gene Therapy Viral Vectors: Advances and Challenges

GUEST EDITOR Prof. Axel Schambach, Hannover Medical School, Germany As cell and gene therapies move towards the clinic, critical developments in the production of viral vectors are required to enable large-scale manufacturing and distribution. A balanced overview of the merits and challenges of the different viral vector types is provided; complemented by a discussion of the key manufacturing considerations to enable large-scale, quality-assured vectors for clinical use including culture systems and their impact on vector productivity.

The Cell & Gene Therapy Manufacturing Pathway: Part 4

Supported by GE Healthcare Innovation and Excellence in ATMP Manufacturing PART 4: Latest advances in the analytical toolkit GUEST EDITORS: Greg Russotti, Stephen Ward & Peter Zandstra The move towards commercialization of cell and gene therapies invariably requires changes and modifications across multiple steps in the clinical-stage manufacturing pathway, in particular with the increasing need to move from manual to closed-system and automated processes. It is critical then that a manufacturer is equipped with the analytical tools to enable product comparability, quality control and de-risking the process changes inherent in moving to commercial scale manufacturing.
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