
The Castle Delivery Meeting
Nucleic Acid Therapeutics, including ASOs, siRNAs, PMOs, mRNA, have enabled development of novel therapeutic options for disease targets previously considered un-druggable by conventional small molecule and biologics means. While major advances and recent regulatory drug approvals have been made with liver and CNS, with muscle closely following on, delivery outside of these tissues remains the technological problem to solve. Even for the most advanced clinical candidates currently less than 1% of the cargo is escaping from the endosome. It is widely recognized that there are significant knowledge gaps in our basic understanding of the mechanisms used for uptake and trafficking inside cells in vivo, and how-to best harness this knowledge to advance delivery for therapeutic use. This conference will provide a critical thinking forum for increasing our mechanistic understanding of the biological processes underpinning delivery, including molecular and cellular biology, in vivo pharmacology and toxicology, rational optimization strategies, targeting, formulation, bioengineering, preclinical and clinical programs. The conference will bring together biologists, chemists, pharmacologists, biophysicists, drug developers, nanotechnologists from academia and industry developing delivery strategies.
