Pawel Stocki

• 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

• Explore the search for CNS-targeted oligonucleotide ligands and shuttle strategies that could match GalNAc’s efficiency in the liver
• Evaluate emerging chemistry, conjugation, and receptor-mediated approaches, including methods to validate and optimize chemical modifications for CNS delivery
• Discuss translational opportunities and challenges in creating next-generation CNS delivery platforms that enable differentiated therapeutic strategies

NEW DATA