Following the Science, Every Day
We focus on pioneering science with transformative, and even curative, potential.
Research & Development
Our Approach
Building a better tomorrow, every day.
Delivering for Rare Disease Patients – Every Single Day
Pioneers in Rare Disease R&D
Alexion’s rich history of scientific firsts has accelerated the course of rare disease R&D and changed the trajectory of treatment for people living with certain rare conditions.
We are driven by a sense of urgency to push the boundaries of science to improve the lives of people living with rare diseases and their families.
Delivering for Rare Disease Patients – Every Single Day
We look to apply our rare disease expertise where it is most needed, and where we can make the biggest difference for patients and their families.
This often means we are exploring disease states where scientific progress has stalled for decades, and where patients have limited – or zero– treatment options.
Our Key Priorities
Our pipeline sits across multiple therapeutic areas, including haematology, nephrology, neurology, bone metabolism, cardiology, endocrinology and rare cancers. To make the greatest impact for patients, we are focused on three key priorities:
Sustaining Our Leadership in Complement
We are exploring rare, complement mediated diseases, developing next generation inhibitors, and researching new targets within the complement system.
Expanding Our Leadership in Rare Disease
We are advancing pioneering science to help address rare diseases with limited or no treatment options and with unmet medical needs.
Pushing the Boundaries of Science
We are investing in our research and new, potentially curative, modalities and collaborating across our global R&D network to accelerate the pace and impact of our work.
Leading in Rare Disease R&D: Patient Centricity in Action
A Patient-First Philosophy
Developing life changing medicines for rare diseases requires scientific expertise as well as deep knowledge of the patient experience. We put patients at the center of our work every day, through our people, processes and metrics. This approach ensures we hold ourselves accountable to the people who matter most.
Leading in Patient Centricity: Why it Matters
Continuous Innovation
Rare disease R&D presents unique challenges that require continuous innovation to deliver new treatments for patients.
Driven by our patient-first approach, we continue to tailor our R&D process and develop new tools and methods to gain a deeper understanding of rare conditions and the needs people living with them face.
Veeva ID: GL/ALL/0053