Program Cohort iPSCs Phenotype Assay Screen Hit Lead Opt. Preclinical
CDKL5
PTEN
EHMT1 (Kleefstra)
FOXG1
Chr21 / Down Syndrome
ANKRD17 (CAGS)
SHANK3 (Phelan-McDermid)
SETBP1
KCNQ2
TSC2
SCN1A (Dravet)*
Epilepsy & Pain (CBD)*
AP-4 HSP*
SSADH
SynGAP1
Complete    In Progress    * Patent application filed