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Theranexus, treatment for rare neurological diseases

Published on 15 December 2022
​Innovative drug candidates for devastating and currently-untreatable neurological diseases.

Theranexus is an innovative biopharmaceutical company born from the CEA. It is developing what is currently the most advanced drug candidate in the world to slow the progression of Juvenile Batten disease, a neurodegenerative disorder that affects children starting at the age of four. The drug could be made available in 2026.

The Beyond Batten Disease Foundation (BBDF), which is collaborating with Theranexus to develop Batten-1, was created by Charlotte and Craig Benson in response to their daughter Christiane’s childhood Batten disease diagnosis.

Batten disease is a genetic disorder that affects children as young as four. It results in loss of vision, motor and cognitive impairment, and seizures. There is no treatment available, and the disease is fatal after 20–25 years on average.

Batten-1, the drug candidate developed by Theranexus in partnership with the US-based Beyond Batten Disease Foundation, addresses this condition by targeting both neurons and astrocytes, non-neuronal brain cells. It acts on the process that recycles toxic molecules from cells, which the disease disrupts. Theranexus will begin a pivotal clinical trial in 2023, with the hope of getting the drug onto the market in 2026.
Beyond this flagship project, Theranexus, a startup created by two CEA researchers, is working collaboratively with the CEA to discover other innovative therapy drugs. Possible future avenues include targeting one or more of the 40 rare neurological conditions close to Batten disease.

Key figure: 350 million

Rare neurological diseases for which no treatment exists affect 350 million people worldwide

Prescribed for:

  • Treatment of Batten disease


  • Proprietary pharmaceutical specialty protected by X patents
  • Action mechanism: prevents brain cell death by blocking glycosphingolipid accumulation and neuroinflammation
  • Method of administration: oral solution

Year founded: 2013

CEA institute: CEA Jacob