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Leading the Charge in Rare Disease Research

At Alexion, AstraZeneca Rare Disease, you grow and innovate in a dynamic, entrepreneurial environment. We deeply connect with patients to create transformational medicines. Here, every story matters, we are patient-driven, united as a team to make a meaningful impact.

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About Alexion

We are pioneering new possibilities for the rare disease community.

With more than 400 million people worldwide living with a rare disease, and 10,000 rare diseases known to exist today, rare diseases are not as rare as we may think. Our expertise in rare disease R&D allows us to diversify our pipeline and portfolio to make an impact across disease areas with significant unmet need, using an array of innovative modalities. With a diverse late-stage development portfolio and a robust pipeline spanning multiple therapeutic areas and modalities, we continue to do more and better for patients.

Our Philosophy

Every day, we're inspired to think differently and follow the science to transform the lives of people around the world living with a rare disease. These five pillars continue to guide our decisions.

  • Pioneers in Science: Advancing the next wave of rare disease innovation to revolutionize care and explore potential cures for patients.
  • Patient-Focused Innovators: Cultivating deep connections with rare disease patients and caregivers to integrate their insights and lived experience into everything we do.
  • Advancing our Global Footprint: Expanding the reach of rare disease research, knowledge and access to treatments around the globe.
  • Championing Health Equity: Taking bold steps to speed access to diagnosis and equitable care for the benefit of patients and society.
  • Purpose-Driven People: Uniting our teams, driven by our commitment to pioneer life-changing outcomes for the rare disease community and for the health systems we operate in.

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.

Voices of Alexion: Testimonials from Our Team

Our Disease Area Group

We look to apply our rare disease expertise where it's needed most, and where we can make the biggest impact for people living with rare diseases. 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 Rare Disease Area Groups include:

  • Rare Haematology, Nephrology & Transplant
  • Rare Neurology
  • Rare Bone & Endocrine Disorders
  • Rare Cardiology & Amyloidosis
  • Rare Tumours

Our Approach - Following the Science, Every Day

We focus on pioneering science with transformative, and even curative, potential.

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.

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.

Our R&D capabilities   

Our R&D philosophy listen, understand and respond to the communities we serve. We have proven what's possible in a variety of rare diseases and we continue to advance our science to help patients and families fully live their best lives. Rooted in deep rare disease subject matter expertise, our unique R&D capabilities and approach include:  

  • Designing Research Frameworks for Rare Diseases.
  • Shortening the Diagnostic Journey.
  • Conducting Clinical Trials in Rare Diseases.
  • Infusing Patient Centricity at Every Stage.
  • Collaborating and Working Together.
  • Establishing Regulatory Pathways.

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