Bringing you the latest cutting-edge research and commentary in bioscience.

February 2017 GUEST EDITOR Prof. George M Church, Professor of Genetics at Harvard Medical School, USA 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 will feature updates from leading groups who are focused on utilizing gene editing technologies to advance cell and gene therapeutic approaches.

As the cell and gene therapy sector matures, the requirement for robust and high quality supply of GMP-compliant raw and ancillary materials becomes more and more of an imperative. This spotlight looks at the key current issues with a focus on real-world experiences: Donor screening and selection Optimizing raw materials at source: key considerations for the safe collection and processing of cell source material Characterization of raw materials: identity, purity and functionality testing Understanding the regulatory requirements

Guest Editor - Dr Richard Harbottle As cell and gene therapies move towards the clinic, critical developments in the production of alternatives to viral vectors are required to enable large-scale manufacturing and distribution. A overview of the latest developments in the design and advantages of next generation vectors will be provided, complemented by a discussion of the key manufacturing considerations to enable large-scale, quality-assured vectors for clinical use.

Guest Editor - Dr Isabelle Riviere The promising clinical results achieved in cancer patients treated with CAR-T cells has provided optimism and subsequent investment in the development of this technology. As we move towards the clinic, it is essential that reproducible manufacturing processes that generate high-quality, clinical-grade CAR-T cell products are developed and refined. This Spotlight discusses the challenges faced across the CAR-T manufacturing pathway, including standardization, automation, quality control, scaleability to meet patient demand, as well as the state of the art manufacturing platforms.

Examining the latest developments and thinking in preservation and packaging for cell and gene therapies, this spotlight looks at: The latest advances in cryopreservation technology Biopreservation Best Practices for regenerative medicine GMP manufacturing & focus on optimized biopreservation media When is the best time to start identifying your biopreservation and symbiotic supply chain strategy, and how to ensure a positive impact on Cost of Goods? Future goals, including alternative approaches to preservation without cryopreservation

Guest Editor - Dr Joanne Kurtzberg This timely spotlight brings you the latest advances and developments in cord blood manufacturing and clinical development. In collaboration with world renowned expert Dr Joanne Kurtzberg, this spotlight includes a selection of content from leading opinion leaders: Emerging use of cord blood in Regenerative medicine Advances in cord blood manufacturing Clinical translation of cord blood Use of cord blood in pediatric inherited metabolic diseases – update on clinical developments Unrelated cord blood transplantation in non malignant and malignant diseases Economics of cord blood banking

Ever more cell and gene therapies are entering clinical development, amplifying the requirement for novel purification systems designed specifically to meet regulatory requirements whilst increasing yield. This spotlight will provide a forum for discussing the current challenges and latest developments in delivering high purity cells and vectors including Harvesting, Purification, Enrichment and Fill-Finish strategies.

Guest Editor - Dr Qasim Rafiq, University College London, UK Our 2nd annual automation spotlight focuses on tackling the key questions facing those grappling with how to use automation, disposables and single-use technologies in the production of their cell and gene therapy candidates. The issue includes discussions on the: Question of when and what to automate Emerging business models for automation Potential benefits of automation for the production of cell therapies Application of automation in process monitoring and control in cell therapy manufacture Progress and Prospects of automation in CAR T manufacturing

Efficient and effective scale-up of production for viral vectors and allogeneic cell therapies and scale-out of production for autologous cell therapies; everyone agrees that these are absolutely key to the development of commercially viable products. But how can CoGs be controlled in practice? And what’s the optimal timing to start working on scale-up or scale-out? This spotlight includes the latest thinking from those leading the field, to help you define the right strategy for your product.

From the point of collection through to point of care delivery of advanced therapies to patients, there are myriad challenges that must be overcome to enable product commercialization. From sourcing clinical grade cells/tissue, training requirements through cost implications to regulatory compliance, this spotlight discusses in detail pressing issues such as: Key practical considerations across the whole supply chain - from vein-to-vein - including the procurement and handling of your soure material to ensure quality, safety and compliance with regulatory requirements Storage infrastructure requirements at Point of Care: Are we suitably geared up for cell and gene therapies becoming routine clinical practice The impact of different thawing techniques on quality and viability of cell and gene products What are the implications of the current regulatory guidance on PoC handling, storage and thawing? End to end tracking of your product; in-flight monitoring of product to ensure quality at the bed side Big data management across the supply chain Utilizing advanced, cloud-ready software systems to effectively and efficiently manage data