Preparation and application of FXI targeted siRNA molecule and GalNAC conjugate thereof

By optimizing siRNA sequence design and chemical modification, and combining it with GalNAC conjugation technology, a TN7 sequence siRNA molecule was developed. This solved the problems of inaccurate design and unsystematic chemical modification of FXI-targeting siRNA molecules in existing technologies, achieving a highly efficient, stable, and specific FXI inhibition effect, and providing a new approach for the treatment of thrombotic diseases.

CN122256344APending Publication Date: 2026-06-23TIANJIN NORMAL UNIVERSITY
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Patent Information

Application Number
CN202610215694.3
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Filing Date
2026-02-14
Publication Date
2026-06-23

AI Technical Summary

Technical Problem

Existing FXI-targeting siRNA molecules are not precisely designed, lack systematic chemical modification strategies, and have delivery efficiency that needs improvement, resulting in poor efficacy in the treatment of thrombotic diseases.

Method used

By optimizing siRNA sequence design through bioinformatics analysis and molecular dynamics simulations, and combining precise chemical modification and GalNAC conjugation technology, a TN7 sequence siRNA molecule was developed to achieve efficient, stable, and highly specific targeted delivery.

Benefits of technology

It exhibits highly efficient gene silencing activity in vitro and a sustained FXI inhibitory effect in vivo. The drug concentration in the liver is more than 5 times higher than in other organs, significantly reducing off-target effects and significantly improving safety and efficacy.

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Abstract

The invention relates to preparation and application of an FXI targeted siRNA molecule and a GalNAC conjugate of the FXI targeted siRNA molecule. The siRNA molecule takes a TN7 sequence as a core, is obtained through precise bioinformatics design and high-throughput screening, and has excellent gene silencing activity. Through systematic chemical modification and optimization, the molecular stability is remarkably improved. More importantly, the TN7-GalNAC conjugate shows an unprecedented lasting inhibition effect in a mouse in-vivo experiment, and the FXI protein level is continuously maintained below 10% of the basic level within 28 days after single administration. The invention provides a brand new molecular tool for precise treatment of thrombotic diseases, and has important clinical application value.
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