In early Dec 22, I wrote about uniQure’s latest development on their AMT-061 gene therapy treatment. They have made headlines as the world’s most expensive drug. For this post, I take a more in-depth look into this company to see what are the current developments on AMT-061 and their research pipeline.

Company Overview

uniqure NV is a biotechnology company focused on developing gene therapies for rare diseases. The company was founded in 1998 and is headquartered in Amsterdam, Netherlands. uniQure uses an Adeno-associated (AAV) virus delivery system for the foundation for all their therapeutic genes. They use the AAV5 variant, or serotype, and have exclusive, worldwide rights to its use in therapeutic products delivered to the brain or the liver. https://www.uniqure.com/science-innovation/aav-platform You can visit the link above to get a complete understanding of how the system works. There...