The further advance of techniques for genetic modification also necessitates development of novel and more comprehensive genetic characterization tools to identify intended and unintended genetic changes in (subpopulations of) genetically modified cells. But the proliferation of these tools poses both opportunities and challenges for those developing cell and gene therapy products. The selection of global and/or targeted genomic analysis methods requires developers to consider the questions they wish to answer and the intended purposes behind the testing.

Is the analysis intended as a one-time highly-detailed survey of a gene-modified product, or will it be used for lot release testing? For viral vector products, what methods are best to confirm transgene identity and integrity? For gene-edited cells, how does one approach assessment of on-target versus off-target edits? For induced pluripotent stem cells (iPSCs), how does one detect genetic variants? What is the role for sequencing methods in testing of critical components and starting materials? How can bioinformatics tools be qualified in order to reliably use the large datasets generated by methods such as Next Generation Sequencing (NGS)? What are the strengths and limitations of the tools available to us, and how do more traditional methods such as karyotyping fit into the control strategy for cellular therapies?

In this session, we will explore the varied technologies available for genetic characterization and how they can be applied to ensure product quality and patient safety.

Session Speakers:

Characterization of VivoVec: A Surface-Engineered Lentiviral Vector Platform That Generates CAR T Cells in Vivo

Richard Rogers, Umoja Biopharma

Control of Chromosome Integrity in the Routine Manufacturing of Allogeneic CAR T Cell Therapies

Kimberly Davis, Sana Biotechnology

INDUCE-seq: Ensuring the Safe Development of Cell and Gene Therapies by Gene Editing

Simon Reed, Cardiff University

Additional Panelists:

Anna Kwilas, CBER, FDA

Marcel Hoefnagel

Appearances