On this episode of Emerging Biotech Leader, Kim Kushner sits down with Edward M Kaye, M.D., CEO and director of Stoke Therapeutics, to talk about his work in rare disease drug development.

Throughout their conversation, Ed describes the challenges of first-in-class drug development. He explains the importance of understanding diseases thoroughly and the value of natural history studies. He also discusses making the case for long-term research to investors and the lack of established roadmaps.

What stands out is Ed's emphasis on putting patients at the center of the drug development process - not just as a tagline, but in practical, impactful ways. He talks about how Stoke includes patients in their work, from getting input on trial designs to keeping patient groups informed about company updates. This approach, Ed argues, not only benefits the patients but also de-risks development by ensuring the end product truly meets patient needs.

When it comes to success in biotech, Ed takes a broad view. He explains that while drug approval matters, the real aim is ensuring patients can access and use the treatments. This involves working with various healthcare systems and considering patient costs.

For biotech leaders tuning in, Ed offers some straight talk on building company culture and the sometimes lonely reality of the CEO role. It's a conversation that gets into the weeds of biotech leadership while never losing sight of the ultimate goal: creating meaningful therapies for patients who need them.

This conversation with Ed Kaye offers a practical look at the realities of biotech leadership and rare disease drug development. Whether you're new to the field or an experienced executive, Ed's insights provide valuable perspective on balancing scientific innovation, business challenges, and patient needs in the pursuit of new therapies.

Are you leading a biotech and would like to share your experience with our podcast audience? We'd love to hear from you! Please reach out to us.