Women Leaders Rewriting the Protocols of Rare Disease Care
November 20, 2025 | Women in Leadership, Women in healthcare, podcast, rare disease
The true measure of a medical leader is the courage to act when existing systems fail the patient. This is the professional and personal ethos of clinical immunologist Dr. Hilary Longhurst and clinical scientist Mrinal Shah. These women are not only advancing the science of gene editing for rare diseases like Hereditary Angioedema (HAE), but are defining a new standard for female leadership in a high-stakes field.
Listen to the full episode here.
Overcoming Challenges in Female Leadership
Dr. Longhurst spoke about navigating medicine as a woman, recalling moments when her voice was challenged and she felt underestimated. She learned a profound lesson from experience: her greatest professional fears were often “sins of omission”—the failure to act out of caution.
- This self-correction fueled her conviction: “The worst thing that could happen is to become one of those people who doesn’t do what’s right.”
- This philosophy led her to take an unconventional path for an HAE patient whose life was transformed from decades of unemployment to a thriving career.
Listen to the full episode here.
The Power of Patient Advocacy and Transformation
This patient’s story demonstrates that an individual’s brave decision can drive systemic change in rare disease care. For Dr. Longhurst, success means stepping outside rigid guidelines and being brave enough to “represent your patient.”
Responsible Innovation and Protecting Curiosity
For Mrinal Shah, Director of Clinical Development at Intellia Therapeutics, responsible innovation requires protecting creativity and curiosity, especially in a large organization. She emphasizes the importance of the patient voice and transparency.
- Mrinal champions a critical skill for the next generation of women scientists: being unafraid to ask fundamental questions.
- She noted that by simply asking, “somebody inevitably will be like, oh, I’m so glad you asked, because I didn’t know what the answer was either.”
- This openness is foundational to her team’s work in advancing novel science, specifically the one-time, curative potential of gene editing for HAE.
Listen to the full episode here.
A Vision for the Future
The impact of their work confirms that breakthroughs are not isolated events. They are a powerful fusion of diverse expertise: courageous clinicians, scientific rigor, and unwavering patient advocacy.
Their collective message to aspiring women leaders in science and medicine is a powerful call to action: “Take your opportunities, and dream, because you can be part of making that thing happen.” Through this blend of passion and pragmatic innovation, these women are fundamentally redefining the future of medicine.
Listen to the full episode here.
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