Disease Areas

Focused on diseases where one gene copy isn't enough.

We target severe genetic conditions where restoring protein expression could change the course of the disease.

Dravet Syndrome

A severe, progressive genetic epilepsy caused by mutations in SCN1A. Beyond frequent, prolonged seizures, it drives intellectual disability and developmental delays.

Autosomal Dominant Optic Atrophy

The most common inherited optic nerve disorder, caused by mutations in OPA1 leading to progressive vision loss.

SYNGAP1-related Disorder

A rare neurodevelopmental condition characterized by intellectual disability, epilepsy, and autism spectrum features.