Precision siRNA Delivery to the Proximal Tubule via Endogenous Receptor Targeting
- Leverage endogenous receptor-mediated uptake (via megalin) to deliver ligand-siRNA conjugates directly into proximal tubule cells for high kidney exposure and gene silencing
- Achieve durable target gene knockdown (50–70%) in the kidney lasting up to four weeks from a single dose— demonstrating effectiveness of the receptor-mediated delivery strategy
- Overcome a longstanding delivery challenge by increasing siRNA accumulation in the kidney 5–30-fold compared to naked siRNA, enabling new therapeutic targets to be addressed