CRISPR-Cas9 gene editing technology is emerging as a next-generation drug development platform, expanding its application from the treatment of blood disorders to cardiovascular and metabolic diseases, as well as personalized therapies for ultra-rare disease patients. Following its commercialization, the world's first CRISPR therapeutic has seen expanded clinical application in pediatric patients, with a series of cardiovascular disease drug candidates based on in vivo direct gene editing and personalized gene editing treatment cases for individual patients. Recent announcements from the American Society of Hematology (ASH), major academic journals, and corporate disclosures indicate that CRISPR technology is moving beyond its initial application focused on rare blood disorders to become a versatile platform encompassing chronic diseases and precision medicine. Casgevy, the world's first CRISPR therapeutic, is signaling an expansion of its scope with positive clinical outcomes in pediatric patients, demonstrating safety and therapeutic efficacy similar to adults in children aged 5 to 11. Cardiovascular disease drug candidates based on in vivo direct gene editing are also gaining attention. While existing blood disorder treatments involved extracting cells, editing them ex vivo, and then re-injecting them, the in vivo direct editing method utilizes LNP (lipid nanoparticle) delivery technology to edit genes directly within the patient's body. This opens up possibilities for expansion into treatments for chronic conditions such as cardiovascular and metabolic diseases. N-of-1 personalized treatments have also emerged. CRISPR therapeutics custom-designed for a single patient with an ultra-rare disease are being developed, opening a new chapter in precision medicine. CRISPR technology is now establishing itself as a next-generation precision medicine platform, encompassing not only blood disorders but also cardiovascular and metabolic diseases, and personalized treatments for ultra-rare diseases.