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Design of efficient delivery carrier for CRISPR-Cas9 system

 In this era, the concept of pharmaceuticals has expanded from small molecule drugs to macromolecule drugs, leading to novel therapeutic strategies such as gene therapy and regenerative medicine. Especially, nucleic acid-based therapeutics have begun to be approved in Japan such as Spinraza®, Onpattro®, and Collategen® in recent years, and further development as a new therapeutic method for incurable diseases is expected. The key factor determining the therapeutic effect of nucleic acid-based therapeutics is how to deliver them to the site of action of target tissues/cells. Due to their low membrane permeability and difficulty in controlling their biodistribution, nucleic acid-based therapeutics is the field where DDS technology is most needed. In recent decades, various nucleic acid and gene delivery technologies such as lipid nanoparticles, micelles, and conjugates have been developed and studied. In this symposium, we will learn how these technologies can overcome the barriers that exist in the process of nucleic acid delivery in vivo, and discuss methodologies for controlling the biodistribution and intracellular trafficking of them. 

Development of DDS technology for nucleic acid drugs and gene therapeutics --From control of pharmacokinetics and intracellular trafficking to therapeutic application--

The 141st Annual Meeting of the Pharmaceutical Society of Japan (Hiroshima)

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[GS02-5]Design of efficient delivery carrier for CRISPR-Cas9 system

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