The Significance of Human Data in Evaluating a Targeting Ligand

Gene editing’s therapeutic application has transitioned from hypothetical to reality, marked by the recent approval of a CRISPR-based therapy for sickle cell and beta thalassemia. In the wake of these developments, new biotech companies are springing up spurred by advancements that redefine what conditions might soon become treatable.  

One contender in this rapidly changing landscape is Verve Therapeutics. This week on "The Top Line," Fierce Biotech’s Max Bayer sits down with the company’s CEO Sekar Kathiresan, M.D., to discuss how Verve intends to distinguish itself. They also chat about what drove Dr. Kathiresan to biotech after more than 20 years as a cardiologist and geneticist.  

To learn more about the topics in this episode: 

• Verve Therapeutics unveils its lead program—a one-and-done treatment for genetic high cholesterol
• Lilly beams up Verve gene therapy programs for $600M from deal-hungry Beam
• Verve shows base editing works in humans in first clinical data—and is punished by investors
• Verve's base editing hold lifted, starting a thaw on a regulatory blockade that sent peers ex-US

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