Track 04 of 9
Drug Delivery Systems
Nanoparticles, microneedles, oral peptides, gene delivery.
Lipid nanoparticles transformed RNA medicine during the pandemic and now anchor an expanding pipeline of mRNA, siRNA, and CRISPR therapeutics including Casgevy, the first approved CRISPR-Cas9 product. The track will cover next-generation LNPs with tissue tropism beyond the liver, GalNAc-conjugated siRNA durability (inclisiran, vutrisiran, zilebesiran), and the engineering breakthroughs enabling oral peptides such as semaglutide and orforglipron. Sessions will also address microneedle patches, intratumoral and intrathecal delivery, AAV capsid engineering for in-vivo gene therapy, and the emerging non-viral platforms targeting hematopoietic stem cells in situ.
Focus areas
- LNP engineering: extrahepatic tropism and ionizable lipids
- GalNAc-siRNA platforms: inclisiran, vutrisiran, zilebesiran
- Oral peptide delivery: SNAC, permeation enhancers, orforglipron
- AAV capsid engineering and re-dosing strategies
- In-vivo CRISPR delivery and Casgevy ex-vivo manufacturing
- Microneedle patches and long-acting injectable depots
- Blood-brain barrier crossing: transferrin-receptor shuttles