As part of animal toxicity studies prior to the launch of a gene therapy clinical trial for Pompe disease, researchers at the University of Pennsylvania have reported safety problems.
- Rhesus macaque primates were given a gene therapy product developed by the Amicus laboratory in increasing doses by the systemic route.
- The construct included the human GAA gene encoding acid maltase and a tracer gene, contained in an AAV9-like AAV.
- Cardiac toxicity, probably of immune origin, was noted in 5 of the 11 monkeys injected.
These findings call for a degree of vigilance in the context of clinical development, even though this type of complication has not been reported in other gene therapy trials currently underway in Pompe disease.
