Overcoming the Blood-Brain Barrier Challenge on the Trojan Horse
- Utilizing the advanced Trojan Horse formulations to enhance blood-brain barrier penetration, enabling systemic delivery of RNAi therapeutics for neurodegenerative diseases with gain in function
- Engineering receptor-mediated transcytosis to selectively transport siRNA and ASOs into the CNS, reducing off-target effects and improving therapeutic precision
- Demonstrating preclinical efficacy with a primary focus on Huntington’s disease, achieving significant knockdown of huntingtin protein and paving a clear path toward clinical development for severe neurological disorders