Insertional Mutagenesis Risk

Understand where integrations occur, how many copies are present, and what they do to the genome.

Get My Free Risk Assessment

Cancer Formation

Uncontrolled or off-target integration can disrupt tumor suppressor genes or activate proto-oncogenes.
Teams are working to avoid:
Tumorigenesis

Loss of function in key regulatory genes

Upregulation of oncogenic pathways

Failure of cell-cycle checkpoints
Large-Scale Genomic Structural Changes

Insertion events and the editing processes used to create them can introduce unintended structural rearrangements,

Teams are working to avoid:

Any structural change that compromises therapeutic cell quality, safety, or long-term stability.
Regulatory Delays and Setbacks

Regulators expect clear evidence that insertional mutagenesis risk is understood, measured, and controlled.
Teams are working to avoid:
FDA requests for additional genomic safety studies

Delays to IND or CTA submissions

Questions around vector safety profiles
Loss of Therapeutic Function

Unintended integrations may alter genes or signaling pathways essential to therapeutic efficacy.
Teams are working to avoid:
Reduced efficacy of engineered cells

Loss of phenotype or potency

Unexpected biological effects
Unpredictable Cell Populations

Random or poorly controlled integration leads to heterogeneous products that vary from batch to batch.
Teams are working to avoid:
Variable expression levels

Unstable clones

Poor reproducibility in manufacturing
Process Instability

Random integration creates manufacturing processes that are difficult to control and scale.
Teams are working to avoid:
Costly investigations and rework

QC failures

Inconsistent pre-clinical performance that delays clinical entry