To create meaningful advances in rare disease research, it takes an innovative culture, strong collaboration, and a patient-centric vision for delivering transformational solutions. At Alexion, these pillars are important to successfully develop impactful medicines for patients. We spoke with Nick France, Vice President and Therapeutic Area Head for Neurology and Genomic Medicine, about his desire to make a difference for children with rare diseases and how we are at a nexus of incredible technologies with the potential to unlock transformative medicines for patients.
What is your role at Alexion?
As Therapeutic Area Head for Neurology and Genomic Medicine, I oversee our teams of physicians and scientists across multiple disease areas who work every day to move programs through all phases of clinical development to approval.
What led you to rare disease research?
My journey into rare diseases began as a pediatrician working in a critical care unit. I was astounded by the disparity between children who were admitted with chronic diseases, for which there were approved therapies, and children with rare diseases, for which there were no treatments.
I wanted to help solve this problem, so I considered academic research, but realized the biopharmaceutical ecosystem would present the best opportunities for me to make an impact on a global level. I’ve had roles at a large pharmaceutical company developing rare disease research programs, as well as at emerging biotechs building organizations from the ground up. I’m excited to be at Alexion where I continue to be part of this exciting global ecosystem where I believe we can have a big impact on patients' lives.
What’s ahead in rare disease research & development (R&D)?
We have this fantastic opportunity to use an expanding toolbox of therapeutic modalities to unlock transformative medicines for patients.
When I think about the future—and what could be a transformative therapy for patients, even potentially curative—I look at how far we've come as an industry in terms of gene editing, gene therapies, and advanced biologics. We have incredible technologies at our fingertips for doing things that we could only have dreamed of 20 years ago. I’m thrilled to see the potential impact for patients as we follow science and further develop and deploy these technologies with the aim to deliver the best possible results for them.
Why are you proud to work at Alexion?
What really motivates me every day is looking back on where I started as a physician and the rare disease patients that I saw in the clinic who had no treatment options at the time. I look at where we are now, how much progress has been made for some of those patients, and how much more work is left. It is incredibly motivating to come to work every day with the most innovative technologies, incredible scientists, and wonderful collaborators and coworkers. We come together on these projects with the patient in mind.
It has been inspiring to play a role in Alexion's patient-centric approach to drug development. We have a very robust framework in place to ensure that patient insights and touch points are incorporated at multiple points along the journey of developing a new medicine: from entering into the early science to understand the unmet need, to understanding the impact of being in a clinical trial. This approach is essential as we aim to deliver impactful medicines that are meaningful to patients.
What I'm even more proud of is our vision and ambition for the future. There are 10,000 known rare diseases, and over 90% of them don't have approved treatments. We are focused on leading the next wave of innovative medicines and bringing new transformative options to rare disease patients.
Learn more about our innovative culture in our Feature Stories and explore scientific career opportunities at Alexion.
Veeva ID: INT/NP/0036; US/NP/0062
Date of preparation: September 2024