Decoding neurons to unlock breakthrough cns therapeutics
Our Mission
Innovation
across axes
Our approach integrates cutting-edge scalable biology, state-of-the-art technology and engineering, and learning and surrogate models to identify novel therapeutic targets and the best candidate molecules to deliver new and meaningful therapeutics to patients.
03
insights
AI models mine our high-dimensional data for key disease phenotypes and higher in-vivo-potential candidates.
01
Models
Patient-derived stem-cell models enhance translatability by capturing disease phenotypes.
02
signals
High-throughput optogenetic screening collects high dimensional data across 100s of cells in parallel.
Innovation
across axes
Our approach integrates cutting-edge scalable biology, state-of-the-art technology and engineering, and learning and surrogate models to identify novel therapeutic targets and the best candidate molecules to deliver new and meaningful therapeutics to patients.
01
Models
Patient-derived stem-cell models enhance translatability by capturing disease phenotypes.
02
signals
High-throughput optogenetic screening collects high dimensional data across 100s of cells in parallel.
03
insights
AI models mine our high-dimensional data for key disease phenotypes and higher in-vivo-potential candidates.
Therapuetic pipeline
Disorder/Target
Discovery
Pre-clinical
Clinical
Cell Models
Cell Phenotypes
Human Model POC
Screening
Optimization
Mouse ICV Tolerability
In Vivo Efficacy
Rat IT Tolerability
NHP Tolerability (Dev Cand.)
IND Enabling
Phase 1
Chronic Pain Disorders
Nav1.7 (ASO)
Nav1.8 (ASO)
Dual Nav1.7 / 1.8 (ASO)
Nav1.7 (Small Molecule)
Seizure and Neurodevelopment Disorders
Dup15q Syndrome - UBE3A (ASO)
DEE13 - SCN8A (ASO)
DEE4 - STXBP1 (ASO)
SYNGAP (ASO)
Tuberous Sclerosis Complex - TSC2 (Small Molecule)
Fragile X Syndrome – FXS (Small Molecule / ASO)
We welcome partnership opportunities with other organizations seeking to develop novel therapeutics for serious diseases or with parties interested in leveraging our unique capabilities.
