In Feb and March, the PK Methodologies for Biologics, Cell, and Gene working group hosted two discussions on BA support for gene and cell therapies.
In the first meeting, Tong-Yuan Yang presented the current considerations and practices regarding bioanalytical support for gene therapies. The second meeting comprised a group discussion that focused on several topics, including:
• Development and validation of analytical methods: The bioanalytical methods used for gene/cell therapies must be highly sensitive, specific, and accurate to detect the small quantities of gene/cell therapy products in biological matrices.
• Detection of vector integration: Gene therapies use viral vectors to deliver therapeutic genes to target cells. Detecting the presence and integration of these vectors into the genome of host cells can be challenging due to low levels of vector integration and the potential for false-positive results.
• Immunogenicity assessments: Gene/cell therapies can elicit immune responses in patients, leading to adverse reactions. Therefore, assessing the immunogenicity of gene/cell therapy products is critical for patient safety.
• Comparability: Any changes to a gene therapy product's manufacturing process or formulation can impact its safety and efficacy. Therefore, it is crucial to provide data on the comparability of the modified product to the original product, including any differences between the two.
• Software/LIMS for qPCR data regression and analysis: Having suitable software or laboratory information management systems (LIMS) to handle and analyze qPCR data is essential for efficient bioanalytical work.
Bioanalytical work for the support of gene and cell therapies
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Mark Ma Ph.D.
Exe. Director
Alexion Pharma
New Haven CT
[email protected]Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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