Pioneering CNS therapeutics
We reach neurological disease at its genetic source
Precisely targeting genetic disease drivers across the nervous system
Our Science
Broad. Potent. Durable.
Three convergent mechanisms, engineered to precisely target the drivers of disease across the CNS.
Broad and Deep Distribution
Deep reach across the central nervous system.
Potent and Selective Knockdown
Robust reduction of therapeutic targets.
Durable effect
Designed for longer duration.

Our Commitment
Every program begins with the people it’s meant to reach
Lead program
We begin with Huntington’s
A genetic neurodegenerative disease with known targets and a profound unmet need

Team