The BioPhorum Gene-Editing subteam have produced a member only tool enabling the end-user to determine comparisons between different gene-editing techniques and delivery methods which can be used in cell and gene therapy drug product development and manufacture. The tool allows the user to form individual conclusions based on the users' requirements.
Cell and Gene Therapy
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Cell and gene therapies (CGTs) and their patient-specific delivery of therapeutics are often seen as the future of medicines. However, CGT manufacture is complex and typically involves multiple organizations delivering a treatment. In such complex ecosystems of delivery partners, it is vital to define who does what, and how it is all orchestrated. A new BioPhorum document contains a set of reference models explaining who needs to be involved in...
This document contains a set of reference models explaining who needs to be involved in the supply of different types of Cell and Gene Therapies (CGTs) and, at a very high level, what they do. It can be used by anyone who wishes to better understand the manufacture and delivery of CGTs, and in
particular how IT systems can support that.
A member only survey to benchmark industry experience with replication competent AAV testing. The outcome of the survey will help stimulate further discussion of the topic within the workstream.
In its current state, cell therapy manufacturing comprises several open and manual operations which increase the risk of contamination. Members of BioPhorum’s Cell & Gene Therapy Commercialization workstream have generated a ‘mock’ process detailing the manufacture of a generic autologous CAR-T product operated through a series of process steps combining some open and some closed system elements. The mock process has undergone an end-to-end closure analysis to identify those operations that pose the highest contamination risks, and to provide suggested mitigation solutions to minimize such risks. Suggestions include options that are currently available, as well as potential future ‘desired state’ options. This paper details the full process and summarizes potential risks and possible mitigations. The primary aim of this paper is to demonstrate systematic application of a closure analysis method on an ex vivo gene therapy (also known as gene-modified cell therapy) process so that organizations can apply similar analyses to their own processes. The secondary aim is to propose process closure solutions that companies could implement in their own operations.
While previously only found in academic, pre-clinical and early clinical stages, RNA technologies, including mRNA therapeutics, have emerged as a promising drug modality due to their essential biological role in protein expression and their potential versatility in manufacturing. RNA is in the global spotlight and is being
prioritized due to the COVID-19 pandemic. However, to maintain the current momentum as industry and agencies return to a ‘normal’ cycle of development for a wider range of drugs and therapies, experts in this field need to come together to identify and address challenges unique to the RNA modality. BioPhorum has started this by bringing together industry leaders to identify RNA-focused manufacturing challenges, and is working toward solutions to common challenges and improving current processes. The purpose of this article is to introduce this critical area and to show the unique RNA challenges, not only limited to mRNA therapeutics already identified by the cross-industry team, but also to include how these challenges could potentially negatively impact companies and the RNA industry. It sets out what the team is hoping to achieve via this collaboration, both in terms of outputs and benefits to the industry.
EHS: Environmental health and biosafety risk assessment framework for commercial scale cell and gene therapy manufacturing and template
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?
Cell and gene therapies (CGT) are changing the treatment landscape for many diseases and, with each new headline, patients are putting their hope in the promises that these novel therapies may bring. However, these new approaches come with many challenges that the industry needs to address – from delivery paradigms, to regulatory and supply chain issues. BioPhorum Cell and Gene Therapy has worked hard for two years and is beginning to deliver...
Cell and gene therapy (CGT) is a diverse field, with products and processes as varied as the patients it is trying to treat. As a relatively new group, the Commercialization Workstream in the CGT Phorum began by creating maps of the manufacturing processes for CGT; and what originally started as an internal exercise has grown into a valuable project that could benefit not only member companies but the industry as a whole. Creating the maps...