2022 was a big year for uniQure with the U.S. and European approval of world’s first gene therapy for hemophilia B. In its 2022 financial report Monday, the Lexington, Mass. and Amsterdam-based company provided updates on the progress of its Huntington’s disease and ALS programs.
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End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.
The Institute for Clinical and Economic Review (ICER) concluded Tuesday that gene therapies for hemophilia A and B are worth it even at a hefty lifetime cost of $2.5 million.
