MARKETERCSL LIMITED

HEMGENIX® is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal. HEMGENIX was approved by the FDA in November 2022 and received conditional marketing authorization from the European Commission in February 2023.

Background

uniQure N.V. (Nasdaq: QURE) opted to monetize a portion of its royalty from CSL Behring on worldwide net sales of HEMGENIX to provide non-dilutive capital for continued investment in its AAV gene therapy pipeline and platform. By monetizing their HEMGENIX royalties, the company significantly extended its cash runway. The royalty sale was a joint purchase in conjunction with Sagard Healthcare, where HCRx and Sagard were each responsible for a portion of the up to $400 million transaction.

Note: This transaction also appears in the Corporate Financing section under uniQure.