Sangho Park

Aavatar Therapeutics is a next-generation gene therapy delivery platform company that is developing safer and more effective gene therapies by overcoming the limitations of the current wild-type AAV.There are three key technologies that are essential for AAV delivery systems. The first technology is AAV-specific CMC, which allows for the production of AAV with high purity and at low costs.The second technology is capsid/promoter engineering. This technology allows for the delivery of genes to specific tissue cells and the expression of genes in the correct cells.The third technology is payload technology. This technology allows for the delivery of a therapeutic gene or the editing of genes.    Aavatar has the technology to manufacture difficult-to-produce AAV serotypes. This technology can produce full-capsid AAV with 90% or more purity at a low cost. Production yield was much higher than before and the full capsid AAV ratio was dramatically increased to allow the efficacy of AAV vectors to be increased by low dose administration. Aavatar has a platform technology that uses AI and big data to discover engineered AAV capsid/promoters. Our capsid engineering platform uses machine learning to derive the optimal hit from random mutation libraries using packaging and transduction data. Aavatar is introducing editing strategies other than gene augmentation by applying miniCRISPR. We are developing a pipeline of gene therapies for central nervous system (CNS) target diseases, hearing loss, and eye diseases.   My doctoral research program is in the areas of specific protein-RNA interactions which are a fundamental process in an infection cycle of RNA viruses. I was a post-doctoral research scientist in the lab of translational medicine at CHA Cancer Institute. I studied chronic inflammation and cancer with various murine models and cell lines. And I did the post-doctoral fellowship in Agriculture and Agri-Food Canada. I studied changes in the cells by viral infection using quantitative transcriptomics, quantitative proteomics and other microscopic and biochemical techniques.