Integrated Platform of siRNA Drug Discovery

RNA interference using small interfering RNA (siRNA) molecules has emerged as a promising therapeutic modality.  To accelerate the discovery of novel siRNAs, WuXi AppTec has established an integrated platform encompassing siRNA sequence design, chemical synthesis/modification, delivery ligand exploration, and activity/off-target assessment.

At the TIDES USA 2024 Conference, our scientists presented a poster showcasing the effectiveness of this platform in conducting a comprehensive study on complement component 5 (C5) siRNAs, identifying chemically modified C5-targeting siRNAs that exhibit potent inhibition of C5 mRNA with minimal off-target effects.

TIDES USA Poster_Integrated Platform of siRNA Drug Discovery

Download