Addressing Safety Assessment in Next Generation Products

Time: 11:30 am
day: Day 2 Track 1 AM


• 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