Gene Therapy for severe Hemophilia has new Phase I/II Results

29/07/2016 - 3 minutes

uniQure has new data from the Phase I/II trial of its gene therapy for Hemophilia B, a rare genetic disorder. After 9 months, the therapy maintains 75% reduction of transfusions. A good sign for the therapy’s durability.


uniqure fix gene therapy amt060Although quite rare (1 in 20,000 male births), Hemophilia B is famous for being spread in European royal families.

In the present day, patients with severe Hemophilia B need regular transfusions of a type of clotting factor, factor IX (FIX). With its FIX blood level below 1%, they often suffer from spontaneous bleeding into joints and muscles.

Sobi (Sweden) recently received EU market authorization for its long-lasting FIX replacement, which brings down the necessary transfusions to just once per week. Still, the gene therapy pioneer uniQure is aiming higher (or longer, in this case) with one of its clinical candidates – AMT-060.

AMT-060 contains a gene for wild-type FIX, modified for being more easily expressed in human cells (codon optimization).

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