Alexion Pharmaceuticals, Inc.

Alexion is a global biopharmaceutical company focused on rare diseases, and it now operates as AstraZeneca Rare Disease. It develops and delivers therapies for people living with rare diseases, investing in new targets, modalities and technologies, including genomic medicine and gene therapy. More than 400 million people worldwide live with a rare disease, and its work spans conditions such as hypophosphatasia, or HPP, an inherited metabolic disease that affects how bones and teeth harden.

Alexion moved its headquarters from New Haven, Connecticut, to Boston starting in 2018, taking a third of an office tower in the Seaport on floors three through eight. AstraZeneca acquired the company in 2021. Beyond the lab, Alexion sponsors patient registries such as the Global HPP Registry, a decade-long international effort that pools data from patient volunteers, and it publishes thought leadership on health equity, data science and clinical trials.

What to Know

• About 80 percent of rare diseases are believed to have a genetic cause, a focus of its genomic research.
• The Global HPP Registry was the first international effort dedicated to studying HPP.
• The company publishes articles on Rare Disease Day and related community efforts.
• It is a member of the Greater Boston Chamber of Commerce.