BRIEF—$100 million milestone for uniQure

The first sale of Hemgenix (etranacogene dezaparvovec) has triggered a $100 million milestone payment for the gene therapy’s original developer, uniQure.

The Dutch company has out-licensed global commercialization and further development of the product to Australian partner CSL Behring.

The one-time administered medicine is being marketed for the treatment of certain adults with hemophilia B.

Chief executive Matt Kapusta said the success “further validates our AAV platform, built on the back of 25 years of scientific leadership and innovation in the field of gene therapy.”

Hemgenix is currently approved in the USA and across Europe, including the UK.