This paper outlines strategies for reducing the volumes required for stability studies with the goal of conserving product for patients, while remaining compliant and delivering data on critical quality attributes across the shelf life of gene therapy products. The recommendations provided include considerations for both drug substance and drug product.
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The purpose of this paper is to assess and provide recommendations for reducing volumes required for testing of bioburden and sterility of gene therapy products including considerations for clinical stage and process stage (i.e. in-process vs bulk drug substance (BDS)). This paper makes recommendations for reducing the volumes required for testing and therefore conserving product for patients, while remaining compliant and delivering assay and process information on the microbiological status of gene therapy products.
This paper reviews and identifies gaps in existing guidelines on APS for ATMPs; outlines technical limitations and challenges; and provides recommendations on design and how some of the existing challenges may be addressed through risk-based approaches.
A BioPhorum member survey examining approaches taken for labelling of allogeneic CGT clinical drug product.
A BioPhorum member only deliverable, the BioPhorum Commercialization workstream has developed a conceptual facility design that deliberately disrupts conventions for flow, segregation and cleanroom classification.
The scope of this paper focuses on CCIT of viral vector gene therapy drug product (DP). Cell therapies are out of scope. The strategy proposed here for CCIT is applicable to all advanced therapy medicinal product (ATMP) drug products that are manufactured in small lot sizes, typically <500 vials.
A BioPhorum member only survey to help gain further insight around how the industry is approaching scaling the production of AAV in suspension HEK- 293 cells from lab to commercial scale.
A focused short supplementary member only survey to help them resolve a few remaining questions for an article which is in the final stages of writing focusing on container closure integrity testing in CGT.
A BioPhorum member only survey, the goal of which is to understand what the current industry standard is for visual inspection of final drug product bag for Phase I/II autologous/allogeneic cell therapy programs.
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.