We focus on pioneering science with transformative, and even curative, potential.
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 Therapy Areas
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.
- Neurology: Striving to advance scientific understanding in rare neurology and to accelerate the development of potential life-changing treatments. Our innovation in rare neurology spans more than a decade.
- Nephrology: Enhancing the understanding of rare renal diseases and accelerating the development of potential therapies. We are building on our expertise in rare nephrology to study, develop and advance a robust pipeline of investigational therapies across chronic and acute kidney diseases where the complement system may play a role.
- Endocrinology: Identifying potential new opportunities to advance care for those impacted by rare endocrine diseases. Our work in rare endocrinology builds upon our strong foundation in bone metabolism, positioning us to build on established expertise to advance science for a community with significant unmet medical needs.
- Bone Metabolism: Researching and developing innovative treatments that aim to target the underlying causes of rare bone disorders. We are researching and developing innovative treatments that aim to target the underlying causes of rare bone disorders and address critical unmet medical needs.
- Cardiology: Following the science to develop and evaluate potential innovative treatment options to help people living with rare cardiology conditions. We are following the science to develop and evaluate innovative treatment options to help address the unmet need of people living with rare cardiology conditions.
- Haematology: Working to improve potential outcomes for more people living with rare haematological diseases worldwide. Our innovations in rare disease medicines have positively transformed the trajectory of the patient journey for people living with certain rare haematological diseases-establishing and advancing the treatment landscape.
- Rare Cancers: Striving to advance scientific understanding of rare cancers to bring new solutions. Leveraging AstraZeneca’s oncology expertise and established global footprint, we are exploring new molecular targets and accelerating our pipeline to address unmet medical needs for people living with rare cancers.
Our Approach - Following the Science, Every Day
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 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.
