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.

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

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.

Supported by GE Healthcare Innovation and Excellence in ATMP Manufacturing

PART 3: Understanding your product and processes

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.

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?

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.

Supported by GE Healthcare Innovation and Excellence in ATMP Manufacturing

PART 4: Latest advances in the analytical toolkit

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.