Lysosomal enzyme can bypass the blood–brain barrier and reach the CNS following intranasal administration

Here we provide the first evidence that therapeutic levels of a lysosomal enzyme can bypass the blood–brain barrier following intranasal administration. α-l-iduronidase (IDUA) activity was detected throughout the brains of IDUA-deficient mice following a single intranasal treatment with concentrated Aldurazyme® (laronidase) and was also detected after intranasal treatment with an adeno-associated virus (AAV) vector expressing human IDUA. These results suggest that intranasal routes of delivery may be efficacious in the treatment of lysosomal storage disorders.

► A novel non-invasive route of delivery for lysosomal enzyme to the CNS.
► Delivery of IDUA to the brain by intranasal administration of laronidase.
► Delivery of IDUA to the brain by intranasal administration of an AAV vector.