Off-target analysis

Learn about how Ark Therapeutics ensured their lead molecule had no significant genomics off-targets.

ARK Therapeutics is concentrated to manufacturing of adenoviral vectors used in gene therapy, and to study and manufacturing of gene therapies. Ark Therapeutics has a gene therapy center and production facility is Kuopio, Finland.

Problem

Ark Therapeutics was developing a gene therapy for cancer. They had a lead molecule targeting a specific gene and hoped to find out if there were any unwanted off-target effects.

Solution

We developed a combined sequence analysis and gene expression pipeline that categorized genes into targets, secondary targets and off-targets.

Outcome

The categorization of differentially expressed genes showed how the gene regulation cascade was due to silencing of the primary target, and that the therapy had no significant off-target effects.

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