Portola Pharmaceuticals
Alexion Pharmaceuticals, Inc. is dedicated to transforming the lives of people affected by rare diseases through continuous innovation and meaningful value creation. As part of AstraZeneca, they focus on developing breakthrough therapies, advancing science, and improving health equity for the rare disease community worldwide.
Industries
Nr. of Employees
Very Large (1000+)
Portola Pharmaceuticals
Patents
Methods of treating neurofibromatosis type 1 and related conditions with alkaline phosphatase
US-12268733-B2
View DetailsSubcutaneous dosage and administration of anti-C5 antibodies for treatment of paroxysmal nocturnal hemoglobinuria (PNH)
US-12240893-B2
View DetailsPolypeptides that bind complement component C5 or serum albumin and fusion proteins thereof
US-12221473-B2
View Details
Methods of treating neurofibromatosis type 1 and related conditions with alkaline phosphatase
US-12268733-B2
View DetailsSubcutaneous dosage and administration of anti-C5 antibodies for treatment of paroxysmal nocturnal hemoglobinuria (PNH)
US-12240893-B2
View DetailsPolypeptides that bind complement component C5 or serum albumin and fusion proteins thereof
US-12221473-B2
View DetailsProducts
Commercial and investigational medicines for rare diseases
Prescription medicines and investigational therapeutic candidates developed to treat rare diseases across hematology, nephrology, neurology, metabolic disorders, endocrinology, cardiology and bone metabolism.
Commercial and investigational medicines for rare diseases
Prescription medicines and investigational therapeutic candidates developed to treat rare diseases across hematology, nephrology, neurology, metabolic disorders, endocrinology, cardiology and bone metabolism.
Services
Design and conduct of clinical studies that integrate patient engagement, patient-focused metrics and strategies to improve accessibility and scientific rigor.
Assessment and processing of unsolicited physician requests for access to investigational medicines under country-specific regulatory frameworks.
Guidance and administrative support for investigator-sponsored research and externally sponsored collaborations, including submission pathways and evaluation criteria.
Partnerships with academia, research hospitals and biotech to support preclinical translation, target identification, biomarker work and novel delivery method evaluation.
Application of algorithms and analytics to accelerate discovery, analyze clinical and diagnostic data, and support diagnostic pathway improvements.
Provision of medical information responses and formal channels for adverse event reporting and product issue submission.
Design and conduct of clinical studies that integrate patient engagement, patient-focused metrics and strategies to improve accessibility and scientific rigor.
Assessment and processing of unsolicited physician requests for access to investigational medicines under country-specific regulatory frameworks.
Guidance and administrative support for investigator-sponsored research and externally sponsored collaborations, including submission pathways and evaluation criteria.
Partnerships with academia, research hospitals and biotech to support preclinical translation, target identification, biomarker work and novel delivery method evaluation.
Application of algorithms and analytics to accelerate discovery, analyze clinical and diagnostic data, and support diagnostic pathway improvements.
Provision of medical information responses and formal channels for adverse event reporting and product issue submission.
Expertise Areas
- Rare disease research and development
- Clinical trial management for rare diseases
- Genomics and genetic diagnostics
- Data science and AI for drug discovery and diagnostics
Key Technologies
- Artificial intelligence and machine learning
- Next-generation sequencing
- Digital health technologies
- Real-world evidence analytics
News & Updates
Coverage of Rare Disease Day highlighting Alexion’s efforts in rare disease community.
Highlighting the global impact of rare diseases and Alexion’s role.
Showcasing Alexion’s leadership in rare disease research and development.
Profile of Seng Cheng, a key figure in Alexion’s R&D.
Discussing Alexion’s efforts in amyloidosis.
Highlighting innovations in diagnosis facilitated by Alexion.
Coverage of Rare Disease Day highlighting Alexion’s efforts in rare disease community.
Highlighting the global impact of rare diseases and Alexion’s role.
Showcasing Alexion’s leadership in rare disease research and development.
Profile of Seng Cheng, a key figure in Alexion’s R&D.
Discussing Alexion’s efforts in amyloidosis.
Highlighting innovations in diagnosis facilitated by Alexion.