uniQure N.V. has monetized a portion of its HEMGENIX royalties to secure non-dilutive capital for further investment in its AAV gene therapy pipeline.

Target Information

MARKETER CSL LIMITED is associated with HEMGENIX®, an innovative gene therapy designed to address hemophilia B. This treatment works by enabling patients to produce factor IX, the protein that is deficient in individuals suffering from this condition, thereby significantly reducing the occurrence of abnormal bleeding episodes. HEMGENIX utilizes an adeno-associated virus (AAV) vector, specifically AAV5, to deliver the Padua gene variant of Factor IX (FIX-Padua) to liver cells, leading to the production of factor IX proteins that are 5 to 8 times more active than their normal counterparts. Approved by the FDA in November 2022 and granted conditional marketing authorization by the European Commission in February 2023, HEMGENIX represents a significant advancement in the treatment of hemophilia B.

Industry Overview

The global gene therapy market is experiencing rapid growth, driven by advancements in biotechnology and a surge in investments aimed at developing innovative therapies for previously untreatable diseases. Within this context, hemophilia treatment on a global scale has witnessed exceptional progress due to new therapeutic techniques, including gene therapy. The focus on hemophilia B, a less common form of the disease, has particularly intensified with therapies like HEMGENIX launching onto the market, promising improved patient outcomes.

In the United States, the hemophilia treatment market has seen increasing competition and innovation. The costs associated with treating hemophilia can be substantial, making it essential for new treatment