Our development programs are specifically targeted to help compensate impaired cellular metabolic pathways, whose malfunction leads to impaired quality of life, progressive loss of function and significantly reduced life expectancy.
We were established in 2017 based on an extremely promising scientific discovery that could effectively treat patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN). Our genesis follows the completion of a European Union-funded research consortium called Treat Iron-Related Childhood-Onset Neurodegeneration (TIRCON). It was set-up to improve the diagnosis and treatment of Neurodegeneration with Brain Iron Accumulation (NBIA) disorders, a subcategory of IEM’s.
We are backed by a consortium of top-tier international investors including Sofinnova Partners and INKEF Capital.