Oligonucleotide Delivery II : Biology, Engineering and Development an ECI Conference series

Memo

Nucleic acid therapeutics (NATs), including ASOs, siRNAs, mRNA, Gene Therapy and Genome Editing, can precisely regulate, and potentially correct, gene expression in disease. As such, they can enable the development of novel therapeutic options for disease targets previously considered un-druggable by conventional small molecule and biologics means. The NAT therapeutic revolution is ALMOST upon us. While major advances and recent regulatory drug approvals have been made with liver, CNS and eye delivery, delivery outside of these tissues remains the bioengineering technological problem to solve. Even for the most advanced clinical candidates using lipid nanoparticles, currently less than 1% of the NAT cargo is escaping from the endosome. For viral delivery, novel vectors with specific tropism, improved transduction efficiency and safety remain a major need. It is widely recognized that there are significant gaps in our basic understanding of the mechanisms used by non-viral and viral delivery vehicles for tropism, uptake and trafficking in vivo, and how to best harness this knowledge to advance delivery for therapeutic use. The ECI “Delivery of Nucleic Acid Therapeutics: Biology, Engineering and Development” conference will provide a forum for increasing our mechanistic understanding of the biological processes underpinning non-viral and viral delivery, including molecular and cellular biology, virology, in vivo pharmacology and toxicology, rational optimization strategies, targeting, formulation, bioengineering and process development solutions to scale-up and manufacturing, regulatory considerations and preclinical and clinical NAT programs. The conference will bring together biologists, pharmacologists, chemists, biophysicists, drug developers, nanotechnologists, process engineers from academia and industry developing viral and non viral delivery strategies for the antisense, RNA interference, mRNA, Gene Therapy and Genome Editing, ex vivo and in vivo.