Bowen’s FX Therapeutics is pioneering a transformative, systemically delivered protein replacement therapy for Fragile X syndrome, a genetic neurodevelopmental disorder, 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.

​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.

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.