Matt Kapusta, uniQure CEO

UniQure halts high-dose treat­ments of Hunt­ing­ton's drug af­ter 3 pa­tients hos­pi­tal­ized — shares tank

UniQure has been work­ing on a gene ther­a­py for Hunt­ing­ton’s dis­ease for some time, and as the field has suf­fered through mul­ti­ple fail­ures, the com­pa­ny’s pro­gram has been set back.

The biotech an­nounced Mon­day morn­ing that back in Ju­ly, it had ob­served “sus­pect­ed, un­ex­pect­ed se­vere ad­verse re­ac­tions”, or SUSARs, in two pa­tients af­ter they were treat­ed with the “high­er dose” of a gene ther­a­py can­di­date, AMT-130, in a Eu­ro­pean Phase Ib/II clin­i­cal tri­al. A third pa­tient, who was treat­ed back in March in the US, had their side ef­fect deemed not re­lat­ed to the can­di­date, but it then was re­clas­si­fied as a se­vere ad­verse re­ac­tion af­ter re­view.

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