Gene Therapy Safety Testing

Literature review indicates three key areas to be evaluated for a safe Gene Therapy. 1. Immune response, 2. Vascular Injury, 3. Organ Failure. We have a simple but accurate plan to evaluate them.

These three-safety assessments could lead to an adverse event free Gene Therapy design.

Realize the full therapeutic potential of your drug by knowing accurate safety limits.

Leukocyte assessments in AAV treated group showing monocytes infiltrating a glomerular apparatus.

Hepatic Vascular Assessments in AAV Treated Groups. A Healthy Portal and Branching Vasculature. Severe Suppression of Adverse Effects such as plasma leakage may not be needed at all doses thereby increasing dose outcome.

Gastro-Intestinal infections can be prevented by identifying sloughing of epithelia. Organ profile assessments in AAV treated groups liver with sloughing epithelial cells. The activity of sequence specific AAV vectors environment will vary based ion the disease environment.