About

Guided by science and dedicated to patients

Our mission

is simple… bring innovative RNA modulating therapies to patients with genetic neurological diseases.

Our vision

is to become a recognized leader in developing antisense oligonucleotide RNA modulators for genetic neurological disorders and to establish an organization that fosters positive and collaborative relationships with patients, physicians, partners, community members and our fellow employees.

Our core values (TRAITS)

guide our culture and govern our performance.

Teamwork will drive our success
Respect each other
Accountable for ourselves and our teams
Integrity will build trust
Transparency
Serve patients and communities