Bowen’s FX Therapeutics is developing a new therapeutic drug designed to replace FMRP - the protein that is missing in the body and brain of people with Fragile X Syndrome and is the root cause of Fragile X. This protein is critical for learning, memory, and healthy brain development, but people with Fragile X are unable to make enough of it. Our approach delivers a specially engineered version of the Fragile X protein through a simple intravenous infusion. The protein has been designed to cross into the brain, which many medicines struggle to do, and to work safely inside cells without clumping or causing harm.

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In early lab studies, this therapy has shown promising results:
It gets into the brain and is taken up by neurons (brain cells) that need it.
It restores key functions in experimental models of Fragile X. 
It is safer and more stable than earlier versions of the protein.

We are now working on next-generation versions to make the treatment suitable for large-scale manufacturing, and for storage and dosing in humans. Our goal is to select the best candidate to move into manufacturing the drug by 2026, then clinical testing one to two years afterwards.