May 7, 2021

uniQure recently announced that their commercialization and license agreement with CSL Behring  for their investigational hemophilia B gene therapy was finally closed on May 5, 2021 – the agreement had been pending the completion of a series of antitrust reviews in the U.S., Australia, and the U.K.

Etranacogene dezaparvovec, which is currently being investigated in patients with severe and moderately severe hemophilia B, is developed to target the liver using adeno-associated virus serotype 5 (AAV5). AAV5 is a variant of the type of the adeno-associated virus (AAVs) vectors that are being evaluated in multiple gene therapy trials. Thus far AAVs have been demonstrated to be safe and well-tolerated modes of gene therapy delivery.

The licensing agreement, which came into effect on May 6th, provides CSL Behring with exclusive global rights to etranacogene dezaparvovec, for which uniQure will receive a $450 million cash payment from CSL.

“As a global leader in hematology and thrombosis, CSL Behring is an ideal commercial partner, and we are excited to embark on our relationship together with a shared goal of delivering this potentially transformative therapy to patients around the world living with hemophilia B,” stated Matt Kapusta, chief executive officer of uniQure. “This transaction positions etranacogene dezaparvovec to be made available to the largest number of hemophilia B patients as quickly as possible and provides uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates.”

According to a company press release, uniQure will be responsible for the completion of the HOPE-B trial program, manufacturing process validation, and the manufacturing supply of etranacogene dezaparvovec “until such time” that these capabilities are transferred to CSL.

Source: uniQure press release dated May 6, 2021

Stay connected with us! Subscribe to receive the latest news on treatments, clinical trials, advocacy issues, and NHF events.