laboratory specialized in the treatment of rare neurological diseases

« Rare neurological disorders, a major societal challenge and a market in crucial need of therapeutic innovation »

> 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. The company focuses on the role played by non-neuronal cells (otherwise known as "glial cells") in all brain processes, including pathological processes, and develops drug candidates targeting glial cells and neurons. 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 two 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 discovery platform, developed since 2013 and whose activities are being expanded within the framework of the NeuroLead project, in collaboration with the CEA and the Collège de France, with the help of PSPC funding obtained from Bpifrance, and aimed at the identification and characterization of new innovative therapy drug candidates in neurology