NBC News : declared in Firm Pulls World's First Gene Therapy Treatment: No One Wants It

The biotech company behind the Western world's first gene Therapy and the most expensive prescription medicine in history is giving up on the product because of lack of demand. Related: Gene Therapy Offers Hope to Cancer PatientsGlybera is given as a series of injections to fight lipoprotein lipase deficiency (LPLD), a disabling condition that clogs the blood with fat. In fact, the move will save some $2 million in annual costs and help UniQure focus on other gene medicines. Only one patient has been treated commercially since the $1 million treatment was first approved in Europe nearly five years ago, a spokeswoman for Dutch-based UniQure said on Thursday. The commercial flop is a reminder of the economic challenges facing the emerging field of gene therapy, which seeks to cure rare genetic diseases by offering a one-time fix of a faulty DNA but inevitably comes at a very high price.

Goodbye Glybera! The World's First Gene Therapy will be Withdrawn

Since Glybera missed approval in the United States, this announcement marks the end of the road for the world's first gene therapy as it will be withdrawn in October. Gene therapy isn't over — as Kapusta told us, it's just "in the very early innings."Images from gabriel12, tickcharoen04 / shutterstock.com The excitement about the therapy was undercut when it became clear Glybera would be a commercial failure. While the withdrawal of Glybera is the end of a chapter, uniQure's development of gene therapies is accelerating. Now that uniQure is recognised as having proved it concept through Glybera, the company is expanding its technology into broader indications like congestive heart failure.


UniQure to Yank Pioneering Gene Therapy From Market in EuropeXconomy Boston —[Updated, 9:33 a.m. ET, see below] It took decades to get the first gene therapy in the Western world to market. Said Van Deventer in 2016, many of the gene therapy companies "I'm pretty sure wouldn't have been there if there were not Glybera." UniQure (NASDAQ: QURE), with operations in Amsterdam and Lexington, MA, said this morning that it will not seek to renew marketing authorization of alipogene tiparvovec (Glybera), the Western world's first approved gene therapy, in Europe. Gene therapy has since pushed forward more rapidly.


collected by :Lucy William

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