Presenter: Dr. Annabelle Biscans (AstraZeneca)
Date: January 27th, 2021
Small interfering RNAs (siRNAs) have the potential to revolutionize medicine due to their potency, duration of effect, and ability to target previously “undruggable” disease genes. The clinical success of siRNAs is dependent on their efficient delivery to disease tissues. However, as of today, clinically efficient siRNAs are limited to treat liver related diseases only. Robust and safe siRNA delivery in tissues beyond the liver remains a challenge and represents currently unmet technological needs. Thus, developing platforms that enable efficient and safe siRNA delivery to tissues beyond liver is the next milestone for expanding the utility of RNAi technology.
One of the most promising approach is to modulate oligonucleotide delivery through direct chemical modification and conjugation. Full chemical stabilization, identification of optimal conjugates and optimization of siRNA structure and modification content are essential to enabling sustained and robust efficacy following a single administration.