Etranacogene dezaparvovec

Etranacogene dezaparvovec (also known as AMT-061) is a gene therapy medication developed by UniQure for the treatment of Hemophilia B. It is an investigational AAV5-based gene therapy that is administered through a single intravenous infusion.

Mechanism of Action

Etranacogene dezaparvovec works by delivering a functional copy of the human factor IX (FIX-Padua) gene to the patient's liver cells, using an AAV5 viral vector. This gene encodes a clotting factor (Factor IX) that is naturally deficient or dysfunctional in people with Hemophilia B. By delivering a functional copy of this gene, the therapy aims to enable the patient's own body to produce a continuous supply of the clotting factor, thereby reducing or eliminating the need for regular factor replacement therapy.

Clinical Trials

Etranacogene dezaparvovec has undergone Phase I/II and Phase III clinical trials. The Phase III trial, known as the HOPE-B study, demonstrated sustained increases in Factor IX activity levels, with a mean of 37.2% of normal at 26 weeks post-infusion. The therapy was generally well-tolerated, with no patients developing inhibitors to Factor IX and no patients requiring immunosuppressive therapy.

Regulatory Status

As of 2021, etranacogene dezaparvovec is not yet approved by any regulatory authority. However, it has been granted Breakthrough Therapy Designation by the FDA and PRIME (PRIority MEdicines) designation by the EMA.

See Also

• Gene Therapy
• Hemophilia B
• UniQure
• AAV5
• Factor IX