Induced pluripotent stem cell (iPSC) technology, first developed in 2006, has led to significant progress in stem cell biology and regenerative medicine. Human iPSCs are now widely used for disease modeling, drug discovery, and cell therapy. They enable the study of disease mechanisms, the development of new drugs, and the creation of personalized medicine. The combination of iPSCs with gene editing and 3D organoids enhances their utility in precision medicine. This review discusses the progress in iPSC applications relevant to drug discovery and regenerative medicine, highlighting challenges and opportunities. iPSC-based disease modeling allows the study of genetic disorders by generating patient-specific cells that mimic disease conditions. This approach helps identify disease mechanisms and test potential therapies. Gene editing technologies, such as CRISPR/Cas9, enable precise genetic modifications, improving the accuracy of disease models. However, off-target effects and line-to-line variations in iPSCs pose challenges. Isogenic iPSC lines, where only the disease-related mutation varies, are crucial for studying sporadic and polygenic diseases. iPSCs are also used in drug discovery, enabling the screening of compounds for efficacy and toxicity. Phenotypic screening, using iPSC-derived cells, is increasingly popular due to their ability to model human diseases accurately. iPSCs can be used to create disease-relevant cell types, such as neurons, for drug testing. Additionally, iPSCs facilitate drug repositioning, where existing drugs are tested for new applications. This approach has shown promise in identifying potential treatments for diseases like Alzheimer's and amyotrophic lateral sclerosis (ALS). iPSC-based drug screening has identified several clinical candidates, demonstrating the potential of this technology in drug development. However, challenges remain in terms of differentiation efficiency, cell purity, and the need for faster, more stable differentiation methods. The use of direct conversion techniques allows the generation of specific cell types without passing through the iPSC state, offering a potential solution for large-scale screening. In clinical applications, iPSC-derived cells are being tested for treating diseases such as macular degeneration and heart failure. However, challenges such as tumorigenicity, immune rejection, and the need for long-term immunosuppression must be addressed. Autologous iPSCs may reduce the risk of immune rejection, but their high cost and time requirements limit their use for common diseases. Allogeneic iPSCs, while more cost-effective, require improved immunosuppressive strategies to ensure safe transplantation. The integration of iPSCs with gene editing and 3D organoid technologies enhances their potential in regenerative medicine. These technologies allow the creation of more physiologically relevant models, improving the accuracy of drug testing and tissue replacement therapies. Despite challenges, the combination of iPSCs with advanced technologies offers a promising future for personalized medicine and regenerative therapies.Induced pluripotent stem cell (iPSC) technology, first developed in 2006, has led to significant progress in stem cell biology and regenerative medicine. Human iPSCs are now widely used for disease modeling, drug discovery, and cell therapy. They enable the study of disease mechanisms, the development of new drugs, and the creation of personalized medicine. The combination of iPSCs with gene editing and 3D organoids enhances their utility in precision medicine. This review discusses the progress in iPSC applications relevant to drug discovery and regenerative medicine, highlighting challenges and opportunities. iPSC-based disease modeling allows the study of genetic disorders by generating patient-specific cells that mimic disease conditions. This approach helps identify disease mechanisms and test potential therapies. Gene editing technologies, such as CRISPR/Cas9, enable precise genetic modifications, improving the accuracy of disease models. However, off-target effects and line-to-line variations in iPSCs pose challenges. Isogenic iPSC lines, where only the disease-related mutation varies, are crucial for studying sporadic and polygenic diseases. iPSCs are also used in drug discovery, enabling the screening of compounds for efficacy and toxicity. Phenotypic screening, using iPSC-derived cells, is increasingly popular due to their ability to model human diseases accurately. iPSCs can be used to create disease-relevant cell types, such as neurons, for drug testing. Additionally, iPSCs facilitate drug repositioning, where existing drugs are tested for new applications. This approach has shown promise in identifying potential treatments for diseases like Alzheimer's and amyotrophic lateral sclerosis (ALS). iPSC-based drug screening has identified several clinical candidates, demonstrating the potential of this technology in drug development. However, challenges remain in terms of differentiation efficiency, cell purity, and the need for faster, more stable differentiation methods. The use of direct conversion techniques allows the generation of specific cell types without passing through the iPSC state, offering a potential solution for large-scale screening. In clinical applications, iPSC-derived cells are being tested for treating diseases such as macular degeneration and heart failure. However, challenges such as tumorigenicity, immune rejection, and the need for long-term immunosuppression must be addressed. Autologous iPSCs may reduce the risk of immune rejection, but their high cost and time requirements limit their use for common diseases. Allogeneic iPSCs, while more cost-effective, require improved immunosuppressive strategies to ensure safe transplantation. The integration of iPSCs with gene editing and 3D organoid technologies enhances their potential in regenerative medicine. These technologies allow the creation of more physiologically relevant models, improving the accuracy of drug testing and tissue replacement therapies. Despite challenges, the combination of iPSCs with advanced technologies offers a promising future for personalized medicine and regenerative therapies.