Targeted MAPT Silencing via Brain Shuttle–Enabled siRNA Delivery in Alzheimer’s Disease

• Targeted MAPT Silencing via Brain Shuttle–Enabled siRNA Delivery in Alzheimer’s Disease
• Optimising CNS delivery of therapeutic payloads using TXP1, a novel second‑generation, brain‑selective shuttle engineered for enhanced transcytosis across the BBB
• Identifying a potent MAPT‑targeting oligonucleotide for tau silencing and optimising its site‑specific conjugation to TXP1 to enable efficient CNS delivery and robust knockdown
• Demonstrated in vivo efficacy, with potent MAPT knockdown observed in a PoC study in transgenic mice following intravenous administration of TXP1‑MAPT

Speakers:

Pawel Stocki

Vice President, Research

Ossianix