Alexion

Alexion Pharmaceuticals is a biopharmaceutical company that focuses on developing and commercializing therapies for serious diseases. Founded in 2007, the company is headquartered in Boston, Massachusetts. Alexion is known for its innovative approach to drug development, often leveraging its expertise in gene therapy and protein degradation technologies.

The company's portfolio includes a range of therapies for various conditions, such as Hunter syndrome, Fabry disease, and spinal muscular atrophy. Alexion's most notable product is Idursaglase, a gene therapy approved for the treatment of Hunter syndrome, a rare genetic disorder. The company has also developed therapies for other rare diseases, including Fabry disease and spinal muscular atrophy.

Alexion's business model is centered around its proprietary technologies, which enable the company to develop targeted therapies for rare and serious diseases. The company's gene therapy platform, known as "gene editing," involves modifying a patient's DNA to correct genetic defects. Additionally, Alexion's protein degradation technology, called "substrate reduction therapy," aims to reduce the levels of harmful proteins in the body.

In recent years, Alexion has faced challenges, including regulatory scrutiny and financial difficulties. The company has been the subject of investigations by the U.S. Food and Drug Administration (FDA) regarding its marketing practices and the approval process for its gene therapy products. Despite these challenges, Alexion continues to invest in research and development, aiming to expand its therapeutic offerings and address unmet medical needs.