The 146th Annual Meeting of the Pharmaceutical Society of Japan (Osaka)

Organizer: Takao Inoue (NIHS), Junji Kawakami (FIRST, Konan Univ.), Satoshi Obika (Grad. Sch. of Pharm. Sci., The Univ. of Osaka)

Oligonucleotide therapeutics, which act through a novel mechanism enabling the specific regulation of disease-related gene expression, have attracted significant attention as promising treatments for refractory diseases that have been difficult to manage with conventional drugs. Since the approval of the first oligonucleotide therapeutic in 1998, over twenty such agents have been approved worldwide.
Owing to their unique structural characteristics—relatively large molecular weight, polyanionic nature, and high degree of functionalization—as well as their production via solid-phase synthesis, oligonucleotide therapeutics require distinct approaches to quality control compared with traditional pharmaceuticals. Through close collaboration among industry, academia, and government, we have conducted research and development to address challenges in the manufacturing, purification, analysis, and regulation of oligonucleotide therapeutics. This symposium will outline these efforts, present concepts and strategies for quality control and assessment, and introduce advanced analytical and manufacturing technologies developed through our studies. Recent trends in regulatory perspectives on quality assessment will also be discussed.