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.
South San Francisco, California, United States, North America
Services
Patient support programs
Education, treatment access navigation, ongoing support and community connection services for people living with rare diseases.
Managed access (expanded access) program administration
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.
Clinical trial conduct and trial design support
Designing and running rare disease clinical trials with tailored recruitment, retention and patient‑informed endpoints; contact point for clinical trial enquiries.
Externally sponsored research support and collaboration
Processes and guidance for investigator‑initiated research and collaborative externally sponsored studies, with submission portals and investigator guides.
Discovery partnerships and preclinical collaboration
Collaborative opportunities to translate preclinical discoveries into therapeutic candidates, including target identification, biomarker work and novel delivery technologies.
Grant funding and charitable foundation programs
Grant and charitable funding for independent medical education, patient advocacy programs and community projects addressing health disparities in rare disease.
Patient support programs
Education, treatment access navigation, ongoing support and community connection services for people living with rare diseases.
Managed access (expanded access) program administration
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.
Clinical trial conduct and trial design support
Designing and running rare disease clinical trials with tailored recruitment, retention and patient‑informed endpoints; contact point for clinical trial enquiries.
Externally sponsored research support and collaboration
Processes and guidance for investigator‑initiated research and collaborative externally sponsored studies, with submission portals and investigator guides.
Discovery partnerships and preclinical collaboration
Collaborative opportunities to translate preclinical discoveries into therapeutic candidates, including target identification, biomarker work and novel delivery technologies.
Grant funding and charitable foundation programs
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)