UniQure/CSL Set To Launch First Hemophilia B Gene Therapy Next Year At Record Price
With $3.5m Price Tag
The firms’ gene therapy Hemgenix has won a US thumbs up for the treatment of hemophilia B and plans are underway for a US launch with a big price tag that experts agree should pay off in the long-term.
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The gene therapy firm announced that Bristol terminated a collaboration started in 2015. Nona follows its recent alliance with Moderna with a second collaboration, this time with Dragonfly.
In this week's podcast edition of Five Must-Know Things: a preview of the CTAD Alzheimer’s meeting; US myeloma drug withdrawal puts pressure on GSK; UniQure/CSL's gene therapy sets new US price record; new leadership at Teva; and Merck’s Frazier cautions against profit maximization.
Observers have been expecting uniQure to be sold off as a whole but the Netherlands-based firm has chosen to sell just one asset - EtranaDez - and focus on advancing its Huntington’s disease gene therapy.