Bowen’s FX Therapeutics is pioneering a transformative, systemically-delivered protein replacement therapy to restore FMRP function throughout the body, including the brain. Our innovative lead construct comprises the functional engineered version of FMRP  fused to a cell penetrating peptide for enhanced brain and cell penetration. This engineered protein is designed to cross the blood-brain barrier after intravenous administration, a breakthrough in CNS-targeted biologics, and to retain essential FMRP activity while minimizing toxicity and aggregation. 

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In preclinical models, engineered FMRP has demonstrated:

1) Effective brain penetration and neuronal uptake following systemic delivery;

2) Functional rescue in FXS cellular and mouse models;

3) Improved safety and stability profile compared to non-engineered FMRP.

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To optimize clinical translation, we are engineering next-generation variants with improved stability and bioavailability using rational design, 3D modelling and formulation testing, with an aim to select a clinical candidate for manufacturing and IND-enabling studies beginning in 2026.