Rewriting the Code of Disease with Kiran Musunuru_e.091

In this episode of Lab Rats to Unicorns, John Flavin sits down with Dr. Kiran Musunuru, a cardiologist, geneticist, professor at the University of Pennsylvania, and leading expert in gene editing. Kiran’s research spans cardiovascular, metabolic, and rare genetic diseases, with a focus on turning advances in human genetics and CRISPR into therapies that address the underlying causes of disease. He is also a co-founder of Verve Therapeutics and helped lead the development of a personalized gene-editing treatment for infant KJ Muldoon. Kiran traces the path that brought him to gene editing, from growing up with a cardiologist father and pursuing combined medical and scientific training to recognizing the potential of human genetics, stem cells, and eventually CRISPR. He explains how his willingness to adopt new technologies led his lab from early tools like zinc finger nucleases and TALENs to CRISPR, base editing, and prime editing. That progression ultimately opened the possibility of not simply treating symptoms, but making precise changes to DNA that could turn off disease-causing genes or repair genetic mutations at their source. Throughout the conversation, Kiran shares how experiments targeting the cholesterol gene PCSK9 led to the idea of a one-time treatment for cardiovascular disease and ultimately to the creation of Verve Therapeutics. He discusses his unexpected journey into entrepreneurship, Verve’s progression into human trials and acquisition by Eli Lilly, and his return to academia to apply the same gene-editing platform to rare diseases. The episode culminates with the remarkable story of KJ, whose personalized base-editing therapy was designed and produced in roughly six months, as well as Kiran’s larger vision for transforming one-off treatments into a scalable platform capable of reaching patients around the world.