Events

Many cell and gene therapy (CGT) developers use gene editing techniques to generate the therapy or produce the desired therapeutic outcome. However, standard quality control and analytical methods miss what happens within each cell potentially compromising its safety. Bulk NGS averages variant allele frequency across pooled cells, masking whether multiple edits (on-/off-target) landed in the same cell or bystanders. Droplet digital PCR (ddPCR) and quantitative PCR (qPCR) report an averaged copy number, hiding rare high-copy cells that carry genotoxicity risk. Flow cytometry (FACS) sorts cells by surface phenotype but cannot confirm that the same cell also carries the intended edits. Fluorescence in situ hybridization (FISH) and karyotyping catch large chromosomal changes but miss smaller structural changes at the edit site.

Single-cell analysis measures on-target and off-target edits, copy number, zygosity, co-occurring edits, gene expression, and phenotype in each individual cell. This shows which cells carry a specific genetic change and how that change relates to the cell's behavior. That per-cell link between genotype and phenotype is what cell and gene therapy safety assessments need.

Join CRISPR Medicine News for a panel on where single-cell analysis stands in CGT safety today, and how single-cell analysis can shape the future of these therapies.

What you will learn:

• From bench research to national measurement standards, where does cell and gene therapy safety evaluation stand today.
• Which analytical methods are commonly used to evaluate the safety of cell & gene therapies and where they fall short.
• Where single-cell approaches may fit into analytical and safety packages for cell and gene therapies.
• What NIST's Genome Editing Consortium and its cross-technology studies suggest about making single-cell data comparable and defensible across labs and platforms for cell & gene therapy development.
• Where the single-cell technology will take cell & gene therapy safety assessments over the next 5 years.

Panelists

Stefan Radtke, Ph.D.

• Senior Staff Scientist, Kiem Lab, Clinical Research Division
• Fred Hutch Cancer Center

Ayal Hendel, Ph.D.

• Professor, Principal Investigator, Genome Editing & Gene Therapy Laboratory, The Mina and Everard Goodman Faculty of Life Sciences
• Bar-Ilan University

Samantha Maragh, Ph.D.

• Leader, Genome Editing Program, Biomarker and Genomic Sciences Group, Material Measurement Laboratory
• National Institute of Standards and Technology (NIST)

Moderator: Karen O'Hanlon Cohrt, Ph.D.

• Intelligence Manager, CRISPR Medicine News