2019 Volume 34 Issue 2 Pages 124-130
The gene and nucleic acid medicines have attracted attention as novel drugs by advance of molecular biology and genetics. The gene and nucleic acid medicines are used to age-related macular degeneration and spinal muscular atrophy in clinical practice. However, it is not enough for gene and nucleic acid medicines to integrate the information on quality and safety. Their target diseases and administration routes are limited by low bioavailability because of low stability and poor membrane permeability. Clinical development of gene and nucleic acid medicines need to integrate non-clinical and clinical information and create adequate regulation. It is also necessary to develop chemical modification and DDS for the gene and nucleic acid medicines to increase the stability and organ delivery. Many studies have not produced practical DDS for gene and nucleic acid medicines. Safety and biocompatibility of DDS are very important for clinical use. Most DDS has been developed by the seeds, not healthcare needs. So, we have developed the multi-functional and effective DDSs (Nano-ball) constructed with safe materials of medicines and foods. The spleen targeting Nano-ball have been developed to show its high efficiency as cancer vaccine and infectious vaccine after extensive screenings. siRNA Nano-ball showed therapeutic effect against metastasis of melanoma and peritoneal dissemination of colon cancer in mice. The lung targeting Nano-ball showed a relationship between Nano-ball contents and organ distribution. Nano-ball was able to be prepared by sterilized mass production. Early clinical use of Nano-ball is expected because of their efficacy and safety. We would like to discuss advancement of the gene and nucleic acid medicines from a standpoint of healthcare needs.
