Addressing Safety Assessment in Next Generation Products
Time: 9:45 am
day: Day Two
Details:
• Autologous cellular therapies that incorporate gene editing require safety assays
to ensure absence of aberrant transformation in final drug product
• These assays include evaluating chromosomal abnormalities, IL-2 independent
growth as a surrogate for malignant transformation, and changes resulting from
on- or off-target editing
• Challenges with these assays such as determining acceptance criteria, qualifying
QC sites for assay implementation, and long turn-around-time can ultimately
impact needle-to-needle time
• Long-term goals are to build robust analytical tools, generate sufficient
characterization data, and continually engage with regulatory agencies to
mitigate challenges and ensure safety of final drug product
