First gene therapy for a rare disease

illustration-dna-thumbnail-portraitUniQure, a Dutch company, developed a gene therapy, Glybera to treat lipoprotein lipase deficiency. The European Medicines Agency has recommended approval of Glybera to the EC, which according the NY Times Health article says, the EC usually approves.

UniQure, which is privately held, was formed with new investment and took on the people and assets of Amsterdam Molecular Therapeutics. AMT took the toll of 3 rejections and ran out of money this year. This has obviously been reversed when the approved population had been narrowed to those with the most severe disease and that the company would be required to monitor the outcomes of patients treated with Glybera and provide that data to regulators.

Jorn Aldag, now CEO of uniQure, spoke at the World Orphan Drug USA in 2011. He was then CEO of AMT. Click here for a copy of Jorn's presentation.

World Orphan Drug Congress USA is an annual orphan drug stakeholder gathering to discuss global harmonization and collaboration involving not just industry, government, regulators and payers but also patient groups. For more information on the 2013 conference, click here.

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