Development of advanced biomedical technology for orphan diseases (KAP241791)

This study focuses on developing gene therapy and editing technologies and cell fate conversion technologies for the treatment of genetic diseases, as well as GMP production process technology, to advance the innovative source technology of advanced therapy medicinal products (ATMPs). Additionally, it conducts assessments of genetic and immune toxicity, patient-derived organoid-based evaluations, and animal model development to verify the efficacy and safety of the developed gene and cell conversion therapies.