Human Brain Biodistribution After Intrathecal Administration of Tominersen

Time: 8:30 am
day: Conference Day Two

Details:

  • Human data on brain exposure of tominersen and HTT mRNA after IT administration of tominersen will be discussed
  • The comparison of this data to preclinical model predicted biodistribution exemplifies the value of translational PK models
  • In addition, comparison to control brains provides critical direct insight into the relationship between tominersen IT dosing, brain biodistribution, and pharmacodynamic effect of the ASO in an individual treated with Tominersen

Speakers: