> Mission

Founded in 2013 by two CEA researchers, Franck Mouthon and Mathieu Charveriat, Theranexus is a biopharmaceutical company developing drug candidates for the treatment of rare neurological diseases. Theranexus has chosen to deploy its resources in the research and development of treatments dedicated to rare neurological diseases because of a very strong medical need in these indications.

There are more than 5,000 rare neurological diseases affecting nearly 350 million people worldwide. 70% of these diseases occur in childhood and the majority have no treatment. The global market for the treatment of rare neurological diseases will double in size by 2027. Products in this type of indication benefit from an incentive-based regulatory framework for development and operation, and accelerated market access. In this targeting logic, the company has  innovative assets to achieve its ambitions in rare neurological diseases:

• A drug candidate, Batten-1, for Batten disease, licensed to an American foundation (the BBDF Foundation), which is close to entering the pivotal phase 2/3 clinic.
• A drug candidate TX01, licensed to the company Insud Pharma / Exeltis, in the treatment of Niemann-Pick disease type C and Gaucher disease.
• A drug candidate, an antisense oligonucleotide, targeting the TFEB protein.
• A new drug discovery platform aimed at the identification and characterization of new drug candidates for innovative therapy in rare diseases.