
Advancing Therapeutic Innovation for Patients with Rare Diseases
We develop innovative therapies designed to address the significant needs of patients living with rare neurological conditions, especially when options are limited or inconvenient.
Grounded in scientific excellence and driven by innovation, we transform research into practical, patient-centered treatments –with the ambition to make a lasting difference in patients’ lives.
Therapeutic Focus
From Execution to Vision
Year of Creation
2013
Drug Candidates
in Development
4
Patent Families
3
Targeted Diseases
5
Our Research Department
Our Research efforts focus on unlocking the potential of antisense oligonucleotides (ASOs) to address the underlying causes of rare neurological diseases. Through a flexible and evolving platform, we design targeted therapies aimed at delivering meaningful clinical outcomes – bridging the gap between scientific innovation and real-world patient benefit.






