Applied Genetic Technologies Corporation has run into safety problems after escalating the dose in its paediatric gene therapy trial. Sixty percent of subjects in the small high-dose cohort suffered suspected unexpected serious adverse reactions (SUSARs) after being treated for a rare eye disease.
The study followed a dose escalation and age de-escalation model, giving progressively higher doses first to adults and then to children. AGTC gave the second highest dose to children without incident. After safely dosing adults with the highest dose, which was around threefold higher than the next rung down, AGTC enrolled five kids with total colour blindness to receive the gene therapy.
Three of the five paediatric patients who received the highest dose developed severe inflammation around one month after dosing. Investigators saw inflammation in both segments of the eye and needed to perform a second procedure to diagnose or treat the condition with medications delivered to the jellylike fluid inside the eye. A fourth patient developed significant inflammation but didn’t meet the SUSAR definition.
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