Achillion Pharmaceuticals, Inc.
Alexion is dedicated to transforming the lives of people affected by rare diseases through innovative medicines, research, and patient-centric approaches. As part of AstraZeneca, the company emphasizes patients first, science and innovation, and health equity to develop breakthrough therapies and improve outcomes for the rare disease community.
Industries
Nr. of Employees
medium (51-250)
Achillion Pharmaceuticals, Inc.
Blue Bell, Pennsylvania, United States, North America
Products
Therapeutic pipeline for rare diseases
A diversified development portfolio and pipeline of therapeutic candidates across multiple modalities and therapeutic areas focused on rare diseases, used to support clinical development and regulatory submissions.
Therapeutic pipeline for rare diseases
A diversified development portfolio and pipeline of therapeutic candidates across multiple modalities and therapeutic areas focused on rare diseases, used to support clinical development and regulatory submissions.
Services
Discovery partnerships and collaboration programs
External partnership program to evaluate and collaborate on preclinical translation, target discovery, and technologies aligned to the company's therapeutic interests.
Externally Sponsored Research (ISR/ESC) support
Processes and guidance for investigator-sponsored research and externally sponsored collaborations, including submission pathways and investigator obligations.
Medical education and patient advocacy grants
Grant programs supporting independent medical education and patient advocacy initiatives, and centralized review for grant funding decisions.
Patient support programs
Operational patient support offerings including disease and treatment education, access support for insurance/navigation, ongoing support services, and community connection initiatives.
Clinical trial design and conduct services
Design and execution of clinical trials for rare diseases, emphasizing patient-informed endpoints, recruitment and retention strategies, and regulatory compliance.
Managed access program support
Assessment and administration of unsolicited requests for access to investigational medicines for patients who have exhausted other therapeutic options, subject to local law and supply considerations.
Discovery partnerships and collaboration programs
External partnership program to evaluate and collaborate on preclinical translation, target discovery, and technologies aligned to the company's therapeutic interests.
Externally Sponsored Research (ISR/ESC) support
Processes and guidance for investigator-sponsored research and externally sponsored collaborations, including submission pathways and investigator obligations.
Medical education and patient advocacy grants
Grant programs supporting independent medical education and patient advocacy initiatives, and centralized review for grant funding decisions.
Patient support programs
Operational patient support offerings including disease and treatment education, access support for insurance/navigation, ongoing support services, and community connection initiatives.
Clinical trial design and conduct services
Design and execution of clinical trials for rare diseases, emphasizing patient-informed endpoints, recruitment and retention strategies, and regulatory compliance.
Managed access program support
Assessment and administration of unsolicited requests for access to investigational medicines for patients who have exhausted other therapeutic options, subject to local law and supply considerations.
Expertise Areas
- Rare disease research and development
- Clinical trial management for rare diseases
- Patient-centric trial design and patient engagement
- Translational target identification and biomarker development
Key Technologies
- Data science and artificial intelligence
- Genomics and next-generation sequencing
- Diagnostics development
- Digital health technologies