This paper is the first in a series considering  real-world scenarios where the EHS companion risk assessment template has been completed.  This assessment focuses on cell expansion operations working with the cell line only in R&D labs to develop procedures and controls; it does not include production operations, such as transfection or viral infections.Â
The purpose of this article is to identify, share, and enhance a framework for master cell bank (MCB) testing and release. It is intended to increase the speed of learning and ultimately influence and/or aid regulatory agencies in developing guidance or policies around GT development and manufacturing.
Viral vector manufacturing is a relatively nascent field, with companies working in many different therapeutic areas, and employing a wide range of processes and procedures. Therefore, members of BioPhorum’s Cell & Gene Therapy Commercialization workstream has generated a set of gene therapy process maps that represent example processes, unit operations and platform technologies being used within in vivo gene therapy manufacturing. These gene therapy process maps are intended to be used as an initial introduction to the in vivo gene therapy manufacture processes and so act as an educational tool for people new to the field. They are also intended to form a general platform baseline for scientists and engineers working within the field to support collaboration enabling process improvements and accelerating commercialization of viral vector manufacturing.
Standard methodologies for generating therapeutic cell products (with or without gene modification) continue to change as the field matures. BioPhorum has identified significant gaps in publicly available information describing industrial practices for production of cell and gene-modified cell therapies. Therefore, members of BioPhorum’s Cell and Gene Therapy Commercialization workstream have generated a series of cell and gene-modified cell therapy process maps that represent example processes, unit operations and platform technologies being used in manufacturing today. These maps are intended to be used to form a common frame of reference for scientists and engineers
working within the field and support collaboration to enable process improvements benefitting future cell therapy patients by supporting acceleration of commercialization of novel modalities. They are also intended as an education tool for new members to the cell therapy field.
This paper and supporting template identifies, shares and enhances best practices and standards, improves risk controls and increases the speed of learning. It discusses a general process designed to provide a risk assessment framework and contains a ready-to-use template. This highlights the complexity of commercial-scale manufacturing as well as considering the areas to assess, potential questions to ask while assessing them, and the other parties who may have pertinent input to the overall risk assessment.
The template can form the basis for discussions between a contract manufacturer and client, or between production and development departments. The aim is to address the following questions when evaluating the CGT manufacturing process: What could go wrong? What controls are in place and are they enough? What did we learn from this?