gene therapy

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Using a matrix approach to address challenges for potency assay development for in vivo and ex vivo gene therapies.

The gene therapy field is advancing rapidly and though there has been significant progress, there are yet relatively few commercial products approved globally reflecting the infancy of this modality. Some therapies have suffered setbacks due to adverse side effects in patients and limited knowledge about product quality.   With more than 400 gene therapies currently under development, it is more important than ever to improve our...

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Challenges for potency assay development for in vivo and ex vivo gene therapies and the matrix approach

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.

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Would you like to influence plasmid release specifications for CGT?

Plasmids release specifications are critical to the manufacture of many cell and gene therapy products. These specifications exist, but there is unlikely to be alignment between different companies. What is needed is a common language and the ability to reference a shared platform that will increase the confidence, speed and efficacy of data gathering and collaborations. The platform should also help shape future guidelines and regulations...

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On the road to common solutions in cell and gene therapy

The emerging field of cell and gene therapy (CGT) has only recently started being commercialized and so standard methods for generating therapeutic products continue to change. Companies are developing their own procedures and many are following general biologics processes, which often do not apply to CGT. These divergent processes mean there is often confusion and inconsistency in the field – compounded by significant gaps in information...

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Commercialization: Gene therapy process map

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.

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