Following a disappointing readout last year, uniQure posted promising Phase I/II data for its investigational gene therapy AMT-130 and nabbed the first-ever Regenerative Medicine Advanced Therapy designation from the FDA in Huntington’s disease.
Tag Archive for: uniQure
Today the Dutch drug developer said it would sell a part of the royalty rights for the world’s most expensive treatment Hemgenix to HealthCare Royalty and Sagard Healthcare for up to $400 million in cash.
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.
uniQure inked a deal with Apic Bio Tuesday to gain development and commercialization rights for APB-102, a therapeutic intended to treat a rare, genetic form of ALS.
End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.
U.S. drugmaker Pfizer Inc. said on Thursday its experimental gene therapy for the treatment of hemophilia B, a rare inherited blood disorder, met its main goal in a late-stage study.
If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said.
Bristol Myers Squibb is terminating a seven-year-old gene therapy collaboration to develop potential treatments for congestive heart failure with uniQure N.V. valued at $1 billion.
BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its Biologics License Application (BLA) for Roctavian (valoctocogene roxaparvovec), an AAV gene therapy for adults with severe hemophilia A.
Topline data from Lysogene’s Phase II/III AAVance study showed its investigational gene therapy, LYS-SAF302, failed to meet its primary and key secondary efficacy endpoints in mucopolysaccharidosis type IIIA.