|AUDENTES THERAPEUTICS, INC. filed this Form 10-K on 03/13/2017|
In a previously reported preclinical proof-of-concept study using AAV8-UGT1A1 in a murine model of Crigler-Najjar syndrome, a single administration of AAV8-UGT1A1 resulted in a rapid and significant reduction in total bilirubin levels of 12 mice as compared to 11 mice that received only phototherapy. The administration of AAV8-UGT1A1 also proved durable, lasting the entire 17-month duration of the study. Bilirubin levels at 17 months were over 50% lower in AAV8-UGT1A1 treated mice versus control mice that received only phototherapy. Furthermore, bilirubin levels remained below the level at which neurological damage is observed in this model for the duration of the study.