Our Mission

We are pioneering new possibilities for the rare disease community

Driven by a Mission to Change What It Means to Live with a Rare Disease

Built on a legacy of turning pioneering science into transformative treatments, we listen to and partner with the rare disease ecosystem to help improve outcomes for more people impacted by rare diseases across the globe.

Making an Impact for the Rare Disease Community

Developing breakthrough therapies for rare diseases is only possible because of the tireless efforts of our global community of researchers and scientists, advocates and caregivers, and most important, patients.

Now, as part of AstraZeneca, we have the potential to impact the lives of even more people around the world.

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.

Trailblazers in Rare Disease

As leaders in rare disease, we draw inspiration from our history of delivering scientific firsts that have changed the course of rare disease R&D.

Our legacy in rare diseases is rooted in being the first to translate the complex biology into transformative medicines.

Today, we continue innovating with our pioneering spirit across diverse areas of unmet need — where scientific progress has been absent or limited — advancing first- and/or best-in-class medicines and new modalities.

Our Leadership in Complement

Building on a Legacy of Firsts

It takes collaboration with all of the rare disease community to create more life-changing therapies, a more equitable healthcare system and broader global awareness. It's why we work so closely with patients, advocacy groups and world-renowned research teams to ensure the greatest possible impact.

Our Approach to R&D Our Innovation

Our People

We are innovators, leaders and collaborators helping to change the world for people living with rare diseases.

Meet Our Leadership

Veeva ID: GL/ALL/0051

Research and Development

Innovation

Therapy Areas