Khondrion

We are a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease. Founded by Professor Jan Smeitink, a world-leader in mitochondrial medicine, we are advancing our proprietary science through a wholly-owned clinical and preclinical small molecule pipeline of potential medicines. Our priority is to rapidly develop our pipeline to deliver transformative medicines for patients living with mitochondrial disease. Our in-house scientists are driving innovative research projects and building a portfolio of promising compounds. We have active discovery programmes underway developing new therapies, biomarkers, diagnostic applications and new read-out technologies in the field of mitochondrial diseases. Our lead pipeline asset, KH176, is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases. It has been granted Orphan Drug Designation for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders and Leigh disease in Europe and for all inherited mitochondrial respiratory chain disorders in the US. We work in collaboration with patient organisations internationally as well as a global clinical and academic network to accelerate the discovery and development of our potential medicines for patients with mitochondrial diseases