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CSL/UniQure’s Hemgenix To Enter Uncertain EU Market After Regulatory Nod

 
• By Ayisha Sharma

The firms’ one-time hemophilia B gene therapy has been approved in the EU just months after a US nod, but concerns remain about its prospects on a market that has seen several setbacks for other gene therapies.  

Removing part of a DNA helix, gene therapy concept
Hemgenix Delivers The Padua Gene Variant Of Factor IX To Patients • Source: Shutterstock

CSL Limited/uniQure N.V.’s hemophilia B gene therapy, Hemgenix, has been given a thumbs up from the European Commission but historical and recent mishaps faced by other gene therapies on the European market have not exactly paved the way for commercial success.

The EC granted Hemgenix conditional marketing authorization on 20 February for the treatment of severe and moderately severe hemophilia B...

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