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.
gene therapy
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Minimizing the impact of bioburden and sterility testing on gene therapy batch yield
Jun 2023 | Cell & Gene Therapy, Commercialization, Deliverable, Deliverables Report, POI - Cell and Gene Therapy, Publication
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.Â
CGT gene editing techniques URS
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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.
The challenges of using RNA as a therapeutic or a gene editing tool
Dec 2021 | Cell & Gene Therapy, POI - Cell and Gene Therapy, Publication, RNA as a Therapeutics and a Gene-Editing Tool
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
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Oct 2021 | Cell & Gene Therapy, Feedback to agency, Regulatory Strategy
BioPhorum member only summary document providing feedback and recommendations to FDA consultation Human gene therapy for neurodegenerative diseases: draft guidance for industry
Challenges for potency assay development for in vivo and ex vivo gene therapies and the matrix approach
Sep 2021 | Cell & Gene Therapy, High Level Analytics, POI - Cell and Gene Therapy, Publication, Regulatory Strategy
This paper highlights some of the challenges to develop potency assays for gene therapies and promotes a potential solution. It seeks to establish Industry alignment on the benefits of a matrix approach and provides high level guidance on how to adopt this strategy towards potency assay development and validation with examples for in vivo and ex vivo GT processes.
A discussion to help establish release specifications for master cell banks of parental host cells
Jul 2021 | Cell & Gene Therapy, Deliverable, EHS, POI - Cell and Gene Therapy, Publication
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.