Spotlights 2015/16

Welcome to the Cell and Gene Therapy Insights Spotlights - helping address the critical issues facing those in the field through peer reviewed content, interactive multimedia, interviews, webinars and more.

Navigating the Regulatory Landscape

Date: September 2015

As global Regulatory Agencies continue to evolve their policies to match the pace of development in cell and gene therapy, it’s essential for both academic and commercial scientists to understand the implications of these changes on their path to translation and product commercialisation.

We bring you regulatory updates and insight from three major bodies:
  • EMA
  • FDA
  • Japan
With Japan championing an innovative and radical approach to regulating cell therapies and regenerative medicines, we ask our industry experts to share their insight with us on the commercial impact of these changes – not only within Japan but for international companies looking to collaborate/partner and take advantage of this regulatory environment. The Spotlight also includes a series of interviews and webinars with commercial leaders who share with us their experience in navigating their cell and gene therapy products through the regulatory landscape.

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.

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?

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.