Portola Pharmaceuticals Inc.
Alexion Pharmaceuticals, Inc., part of AstraZeneca, is dedicated to transforming the lives of people affected by rare diseases through continuous innovation and meaningful value creation. The company focuses on pioneering science, patient-centric research, and addressing unmet medical needs in rare disease communities worldwide.
Industries
Nr. of Employees
medium (51-250)
Portola Pharmaceuticals Inc.
Services
Education, treatment access navigation, ongoing support and community connection services for people living with rare diseases.
Evaluation and case‑by‑case provision of investigational medicines when trial participation or other options are not available, subject to local law and supply constraints.
Designing and running rare disease clinical trials with tailored recruitment, retention and patient‑informed endpoints; contact point for clinical trial enquiries.
Processes and guidance for investigator‑initiated research and collaborative externally sponsored studies, with submission portals and investigator guides.
Collaborative opportunities to translate preclinical discoveries into therapeutic candidates, including target identification, biomarker work and novel delivery technologies.
Grant and charitable funding for independent medical education, patient advocacy programs and community projects addressing health disparities in rare disease.
Education, treatment access navigation, ongoing support and community connection services for people living with rare diseases.
Evaluation and case‑by‑case provision of investigational medicines when trial participation or other options are not available, subject to local law and supply constraints.
Designing and running rare disease clinical trials with tailored recruitment, retention and patient‑informed endpoints; contact point for clinical trial enquiries.
Processes and guidance for investigator‑initiated research and collaborative externally sponsored studies, with submission portals and investigator guides.
Collaborative opportunities to translate preclinical discoveries into therapeutic candidates, including target identification, biomarker work and novel delivery technologies.
Grant and charitable funding for independent medical education, patient advocacy programs and community projects addressing health disparities in rare disease.
Expertise Areas
- Rare disease clinical trial management
- Patient‑centric research and outcomes
- Regulatory strategy and regulatory pathways for rare diseases
- Diagnostics and genomic medicine
Key Technologies
- Next‑generation sequencing
- Genomic analysis
- Digital health technologies
- Data science and artificial intelligence (AI)
News & Updates
An interview with Chathuri Daluwatte, highlighting her role in Alexion's R&D.
Exploring the medical needs within the amyloidosis community and Alexion's efforts.
Efforts to improve and shorten the diagnostic journey for rare disease patients.
Profile of Nick France and his contributions to Alexion's R&D.
Profile of Seng Cheng and his role in Alexion's R&D.
Alexion's initiatives to improve health equity for people living with rare diseases.
An interview with Chathuri Daluwatte, highlighting her role in Alexion's R&D.
Exploring the medical needs within the amyloidosis community and Alexion's efforts.
Efforts to improve and shorten the diagnostic journey for rare disease patients.
Profile of Nick France and his contributions to Alexion's R&D.
Profile of Seng Cheng and his role in Alexion's R&D.
Alexion's initiatives to improve health equity for people living with rare diseases.