Therapeutic Discovery Platform for Oligonucleotides Targeting Neurodegeneration & other CNS Disorders

Time: 11:30 am
day: Day One


  • Building a high throughput platform for functional characterization of neuronal activity in the context of genetically defined CNS diseases
  • Q-State uses this platform to inform design and development of antisense oligonucleotides targeting underlying disease mechanisms
  • Presenting examples from projects targeting Spinocerebellar Ataxia 13, Spastic Paraplegia Type 49 and KCNT1-related Developmental and Epileptic Encephalopathy