UniQure unveils mixed Huntington’s gene therapy data, shares tank

UniQure shared up­dat­ed da­ta from a Phase I/II study that showed a clin­i­cal ben­e­fit in a small group of pa­tients with Hunt­ing­ton’s dis­ease who re­ceived its gene ther­a­py, but con­flict­ing da­ta about the mu­tant Hunt­ingtin pro­tein sent shares $QURE down by about 40% on Wednes­day morn­ing.

The Dutch biotech on Wednes­day re­vealed new da­ta from the place­bo-con­trolled Phase I/II study with 26 pa­tients. Six­teen pa­tients were giv­en a low or high dose of AMT-130, with 10 pa­tients in the con­trol group.

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