No rare disease patient left behind.
30 million Americans suffer from a rare disease. 95% of rare diseases have no approved treatment.
Kalevala Therapeutics identifies overlooked therapeutic opportunities in rare diseases. We focus on conditions where promising clinical evidence exists, but regulatory and commercial barriers have previously prevented access in the United States.
Our internal AI discovery pipeline targets conditions with the most unmet need, and we partner with advocacy organizations, clinicians and pharmaceutical stakeholders via licensing and co-development arrangements to streamline timelines from discovery to approval.
In June 2025, Kalevala Therapeutics received FDA Orphan Drug Designation for its KV-3004M program in Gaucher Disease.